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About the NATPARA Recall

What You Need to Know

After discussions with the FDA, Takeda issued a US recall on September 5, 2019, for all doses of NATPARA® (parathyroid hormone) for Injection (25 mcg, 50 mcg, 75 mcg, and 100 mcg).

NATPARA is a parathyroid hormone currently approved in the US as the only adjunctive treatment for adult patients with chronic hypoparathyroidism who cannot be adequately controlled with standard therapy alone (calcium and vitamin D).

With patient safety as the company's main priority, Takeda is communicating directly with US healthcare professionals, who have already received this information, as well as with patients and specialty pharmacies. Discontinuing NATPARA abruptly can cause a decrease in blood calcium levels (severe hypocalcemia) which can result in serious health consequences. It is critically important that patients contact their prescribing healthcare provider to discuss their individual treatment plan and ensure close supervision, including frequent monitoring of blood calcium levels and close titration of active vitamin D and calcium supplements upon stopping NATPARA to avoid low blood calcium (hypocalcemia).

All of us at Takeda understand the impact that this recall has on patients, and we are continuing to work closely with the FDA until we are able to resolve the issue. Takeda values the US FDA’s collaboration and feedback as we work together to resupply NATPARA to patients who need it.

If you have questions or concerns about this recall, browse the information on this page, and talk with your doctor. For more details, read our PDF. Be sure to bookmark this page and check back often for the latest updates.

Watch: The Path to Resupply

When a prescription treatment or medical device is recalled, it can take time to make it available for patients again. Watch this video to learn about the path back to resupply.

Listen: Patient Community Calls

Takeda hosted a call on April 26, 2021, to update the hypoparathyroidism patient community on the NATPARA recall. Listen to the complete recording, or read the full transcript, below.

This is an audio recording of a call intended to update the hypopara patient community regarding the ongoing NATPARA recall.

NATPARA is a prescription parathyroid hormone used with calcium and vitamin D to control low blood calcium (hypocalcemia) in people with low parathyroid hormone blood levels (hypoparathyroidism). It is only for people who do not respond well to treatment with calcium and active forms of vitamin D alone, because it may increase the possible risk of bone cancer (osteosarcoma). NATPARA was not studied in people with hypoparathyroidism caused by calcium-sensing receptor mutations. It was not studied in people who get sudden hypoparathyroidism after surgery. It is not known if NATPARA is safe and effective for children 18 years of age and younger. NATPARA should not be used in children and young adults whose bones are still growing.

During animal drug testing, NATPARA caused some rats to develop a bone cancer called osteosarcoma. It is not known if people who take NATPARA will have a higher chance of getting osteosarcoma. Tell your doctor right away if you have pain in any areas of your body that does not go away, or any new or unusual lumps or swelling under your skin that is tender to touch.

Please listen to this audio in full for additional Important Safety Information about NATPARA. Please also see the Prescribing Information, Medication Guide, and Instructions for Use available on NATPARA.com.

Operator:

My name is Matthew and I will be your event specialist today. At this time, I would like to welcome everyone to today's web conference. All lines have been placed on mute to prevent any background noise. Please note that today's web conference is being recorded.

Today's presenters will appear on webcam on your screen. You can change the layout of your screen at any time in WebEx. Locate the layout option and select great or stage view on the top right side of your screen to toggle between views as needed.

It is now my pleasure to turn today's program over to your moderator Cheryl Schwartz, Head of the Takeda U.S. Rare Disease Business Unit. Cheryl, the floor is yours.

Cheryl Schwartz:

Thank you Matthew and good evening everyone. As Matthew said, my name is Cheryl Schwartz and I lead Takeda's U.S. Rare Disease Business Unit.

Thank you all so much for joining this evening.

Before we get started I just wanted to take a moment to acknowledge your feedback that the written communication we provided last month was surprising for many of you and even upsetting.

Our intent was to provide you with the latest timing estimates for NATPARA so that you and your physicians could make the most informed healthcare decisions, but I do want to personally apologize if that communication came across in any way as either insensitive or incomplete.

I also want you to know that we absolutely hear your frustration. We have read the letters and the e-mails that you sent us and I've heard that many of you have said that you felt hopeless and confused and angry that the issues that have led to the NATPARA recall have taken so much longer to address than really any of us had initially anticipated.

You've also clearly asked us for more information about the specific challenges that we're facing in bringing NATPARA back and why it's taking so much time. And some of you have also had questions about the cause of the ongoing shortage notifications for some of the patients in the Special Use Program and whether those two issues are somehow related.

And then lastly over the last couple of weeks we have been collecting your questions with the objective of sharing more information today on the multiple challenges that we're facing as we continue working towards bringing NATPARA back to patients in the U.S.

So, I want to introduce a few of my colleagues that are joining me here today who will help to address some of the questions that you have asked. First, is Tom Koutsavlis who many of you met along with me at some of our previous community calls. Tom is our Head of Medical Affairs and our Chief Medical Officer for Takeda's U.S. Business Unit. And Tom is also an emergency medicine physician and has treated patients with hypoparathyroidism.

So Tom is here really primarily here to address some of the questions that we've received regarding the Special Use Program and our recent notifications about some possible supply interruption.

So Tom and I are also joined today by Stefan Wildt our Research and Development Chief Technology Officer and Head of our Pharmaceutical Sciences Group.

So, we've asked Stefan to join us here today because a large group of people on his team in our research and development organization are working towards the goal of resupplying NATPARA to all U.S. patients. On today's call he'll explain the work that's being done by our device team as well as our regulatory, manufacturing and quality teams.

And then Stefan will then walk through two separate issues. We'll talk about quite a bit today that are contributing to the overall timelines of NATPARA's return. The first is, as you all know, the device component changes that we have been talking about since the beginning that are needed to fix the rubber particulate issue that originally led to the U.S. recall. And then equally important are some new issues that are impacting our ability to really ensure a stable and reliable supply of NATPARA and that's compounding our timeline.

And then finally I wanted to introduce you all to Tony Frangie who will be joining us just a little bit later. Tony is Vice President and Head of our Endocrinology business. And Tony and his team work every day on coordinating our efforts on NATPARA. He also works really closely with our patient services and our medical team on our Special Use Program support. And so, Tony has been really behind the scenes here also collecting and organizing all of the questions that you have been sending to us.

We've gotten some from the HypoPARA Association, by other groups that have submitted questions and by individuals through the e-mail address that we have provided. So our intent today is try to answer as many of those questions as possible kind of in the upfront comments because there was a lot of consistency in the questions that we received. But we're also going to make sure that we leave some time at the end, and Tony will help us with this, to address some additional very specific questions that you've asked in the recent days.

So with that I'm going to turn it over first to Tom, as I mentioned, to clarify the intent and the purpose of the SUP.

Tom Koutsavlis:

Thank you very much Cheryl. I appreciate that. I did want to say hello and welcome everyone. Unfortunately, I wish we could be doing this more in person. I know many of us have kept in touch over e-mail and text over the – over the past year. And again, looking forward to a chance when we do get together again and say hello.

I did want to cover some key points around the Special Use program and where we are, just to give you a little bit of background and walk you through some of the future-looking directions that we're taking as well.

So we've received several questions about the program itself, including why we can't open the program up to all patients who were prescribed NATPARA at the time of the recall. And I thought it might be helpful to remind everyone why the SUP was put in place, share some of the regulatory requirements around the program itself and give you some insights into the size and scope of the SUP to help give you a sense of the numbers and demands that it places on our manufacturing supply.

So, as probably many of you remember back in September 5th of 2019, Takeda issued a recall for all doses of NATPARA. So, that includes the 25, 50, 75 and 100 microgram doses. The recall occurred after discussions with the – with the FDA due to the potential risk of some rubber particles from the rubber stopper, the component that you know as the septum, entering into the cartridge itself at the time when it's punctured by the needle.

So during that 14-day NATPARA treatment period you could potentially have some rubber particles coming in through the septum when it gets punctured and that – those rubber particles may end up in the cartridge.

So, I wanted to pause and mention here that we've received many questions about the concept of the words voluntary recall. I think many of you may have read the words voluntary recall on the websites and how the FDA has described the recall that we have in play.

So just to clarify a voluntary recall is a regulatory definition for initiating a recall without the FDA requiring a filing of legal action or court proceedings. So, most drug recalls in the U.S. are labeled as voluntary recalls.

And even though NATPARA was classified as a voluntary recall, it was still an action Takeda was required to take as per the FDA code of regulations. And that's code of Federal regulations and there's a lot of detail that comes with that. So again, although voluntary is a term that's used in the statement itself, it is a recall that's required by the FDA.

So, at the time of the recall we focused on informing patients as quickly as possible that we were not longer authorized to commercialize NATPARA and that patients should seek their physicians immediately so they could be safely discontinued from the product.

We also reached out to physicians so patients like yourselves and providers could work together so that they can discontinue NATPARA as safely as possible considering the circumstances.

Now during the communication period that we had early on we started receiving concerns from healthcare providers that for a very few specific patients abrupt discontinuation due to the recall itself could lead to life threatening complications and that was definitely a concern that we heard from several healthcare – several of your healthcare practitioners.

And when the product itself is subject to a Class I recall federal law requires that all of the product be withdrawn from the market and the manufacturer can no longer make the product available to patients.

So, because of the concerns that we had from the healthcare community, healthcare practitioners, we reached out to the FDA and we worked on a solution of quickly putting together a very unprecedented program.

So, it's the first time this type of a program has been put into play. Where with the U.S. Regulatory Oversight we would give some healthcare practitioners the ability to request NATPARA for extraordinary cases where patients were facing life threatening consequences as a direct result of the discontinuation.

And that became known as the NATPARA Special Use Program, which you know today and which we sometimes refer to as the SUP. So this part, the part about the complications being a direct result of NATPARA discontinuation is particularly important because the SUP was intended to address only life threatening complications directly resulting from not having access to the product after the recall came in play.

So, in order to apply for the SUP your physician must initially assess whether you meet the appropriate criteria and then if so submit the applications to the Adjudication Committee with information evidencing the patient life threatening condition.

And again, the program was implemented with specific access and use restrictions to mitigate the risks associate with the recall and obviously to follow all of the particular requirement environment and the regulatory partnership that we had with the FDA.

So, it's important to note that as part of our agreement with the FDA to put the program in place a medical adjudication committee assessed and continues to assess to this day all SUP applications to ensure that they meet and continue – that they met and continue to meet the strict requirements of the program. The committee itself consists of both Takeda medical professionals and outside medical experts to ensure that we maintain the utmost of objectivity as we assess each one of these cases.

Now we originally estimated the program would be small and relatively short term while we were working on getting NATPARA back. But, of course, as you know and unfortunately the time to bring the product back has taken much longer than expected and today we actually have about 430 patients enrolled in the SUP in the U.S. So it's a fairly significant number.

Now according to the SUP requirements and remember the program is very regulated, strictly regulated with the FDA, NATPARA must be administered as single-use. So only one dose per cartridge and this is due to the potential risks of the device and the risk of the rubber particulate being pushed into the medication vial if the stopper is punctured repeated times with the needle itself. And that was of course the initial reason for the recall.

So what this means is that the SUP program while only meeting the needs today of about 430 patients requires twice as much supply of NATPARA as the entire patient population required prior to the recall. So two times as much supply. I know the numbers may not make intuitive sense but let me maybe walk you through just briefly some of the math that goes into how much product is needed at this point when we have to use a single cartridge as opposed to using a single cartridge 14 times to using a single cartridge only once.

So if I look at some of the numbers, if we think of before the recall itself there were about 2,800 patients using NATPARA and that equals about 72,000 cartridges per year. And if you look now at the little over 400 patients that we have that's about 150,000 cartridges per year through the SUP program. So we've doubled the demand of the supply in this smaller group of patients. And that's the unfortunate reality of only being able to use the cartridge once as opposed to 14 times.

So mention the significant supply demand of the SUP because any manufacturing delay we have can lead to a potential supply disruption for SUP enrolled patients. So this is why and you've seen in communications with you in the past, we monitor the SUP supply extremely closely and obviously the main goal is that we need to maintain the continuity of that supply for all of our SUP enrolled patients. So very critical that we keep a close eye.

So again a very unprecedented program and obviously there to help our most severe life threatening circumstances that some of you have faced personally. And I'm pleased that we're able to continue that with our FDA colleagues as we work closely with them.

So now I'm going to pass it over to Stefan who will be able to walk you through some of the work we're doing on remediating our device and other work on the manufacturing supply. So, Stefan, I hand it over to you.

Stefan Wildt:

Thank you, Tom. This is my first time meeting this patient community. I would like to thank you for giving me the opportunity to describe to you all our efforts. I represent an interdisciplinary team of dedicated Takeda colleagues and I want to assure you that we are working tirelessly to find a way to bring NATPARA back to you as patients.

So who is this team and how are we working? The team consists of colleagues from all parts of Takeda. Colleagues in development who work on the device and the drug product itself. Experts in manufacturing, quality and regulatory who are dedicated to the effort. In addition, we have specialists from distribution, medical and patient services. All of us are and have been focused on that NATPARA during this time and we remain highly committed to you, the patient community.

As you would expect, Takeda has dedicated significant resources across the organization to find a way to get that NATPARA back to patients. However, the journey has been much more difficult than we could have anticipated.

So what then is our approach? Shortly after the start of the U.S. recall our cross functional team went to work to first understand the specific regulatory concerns underlying the recall. Second, develop tests to understand the cause of the problem. And third, make a plan to evaluate remediation approaches to fix device components and address the concerns surrounding the recall.

Following this three-step process we tested a number of approaches that we hoped would allow us to quickly fix the device and return the product to patients. For example, Tom mentioned the rubber septum, we examined the rubber septum itself, here we focused on the possibility of fewer than 14 punctures. We also looked at the needle and just to give a sense for the level detail, we studied the way the needle twists through that rubber septum. We examined the rotation during needle insertion as well as several other variables.

This kind of testing takes time. It can involve designing studies that will appropriately address the questions from regulators. Conducting the studies, generating and analyzing sufficient data and compiling the reports and drawing conclusions. Then, if necessary, it can also include redesigning the studies and going back to the very first step.

When our early tests failed to identify a short-term fix to the device we had to pivot to our disappointment to work on more complex, robust and reliable solutions. It was at that time that we communicated the delay of at least one year in January 2020.

As we moved to other ideas to solve the problem we started all over again with the process I just described. We designed and conducted new tests. Tested the hypotheses followed by generating and analyzing data reports.

So where are we today in April 2021? At this time we believe we have developed an approach that will address the rubber particulate issue. Specifically, after a significant amount of testing we have changed two components of the device. We have introduced a new rubber septum and a new needle. We met with the FDA earlier this year to discuss the approach and work is ongoing now to generate data that the FDA has requested for a review and potential approval.

It might sound simple to replace a few components of the device however I'm hopeful that I have been able to give you a sense for the complexity of the task at hand that Takeda teams are dealing with here. We are encouraged by the progress we have made to date. At this point we're still a few months away from having generated all the data required for submission to the FDA.

Our latest assumption is that the data will be submitted in the third quarter of this year likely during late Summer. So, let's look ahead for a moment. What will happen next? Once our data and reports will be submitted the typical regulatory review timeline for this kind of submission can be four to six months. After that there are a few potential regulatory outcomes such as; one, regulatory approval; two, regulatory feedback that may have required minor or moderate changes to our approach or three, significant regulatory feedback that may lead to starting the process all over again.

So while we are optimistic that we have a potential solution, the timeline and outcome is unfortunately still far from certain. In the best case this process and current timeline would put an approval the fix of the device issue and return to the U.S. market at about 10 to 12 months from now. So this is important let me just restate that. In the best case we're looking at a return to the U.S. market with a fix of the device issue in 10 to 12 months from now. You have our commitment that we keep the community updated on our submission progress, timing and of course, the outcome.

At this point let me turn to a third topic, which is an emerging issue that arose while we were making progress with how to fix the device. This new issue has had an impact on SUP supply and also has the potential to impact our ability to provide consistent, reliable and stable supply in the future. To explain the issue I would like to take a step back and provide some information about NATPARA's manufacturing process.

First off, NATPARA is produced in what we refer to as batches which are based on dose strength, for example, 25 micrograms, 50, 75 and 100 microgram doses. During and after the manufacturing process every batch must meet predefined rigorous manufacturing and quality standards. These standards have been agreed upon with the FDA. Standards like these are put in place for approved medicines to ensure they're producible, reliable and safe manufacturing.

If a batch does not meet those rigorous standards for any reason, it cannot be released for patient use and is destroyed. One important part of the inspection and product release process is the visual inspection of the product; I'll explain that in a second. For NATPARA, the regulatory release requirements include that the drug product solution is clear and essentially free from particles.

The issue that occurred recently, which is impacting SUP supply for specific doses of NATPARA, is due to a higher percentage of batches that have not met the visual inspection requirement. So now let me move on to what we know. We know that the manufacturing process for NATPARA is particularly complex because it is a molecule that is extremely sensitive to manufacturing stresses, which can result in some product lots not meeting the quality standard established with the FDA.

We are currently actively working to understand one – why we are seeing an increase in the number of batches that do not meet our visual release criteria and why that's happening at a much higher rate than ever before, and two, why the manufacturing stress appears to be impacting the higher concentration doses, the 100 microgram dose at higher rates than the other doses?

In those batches that failed the visual inspection, we've seen clumping or cloudiness that shows the presence of protein particles. If we observe protein particles in the drug solution, the product may not meet the release specifications. And again, if it doesn't meet the regulated specifications for release, we cannot and would not supply it to patients.

Before I go further, it is important to note that all batches that have been released for any patient use have met quality standards agreed upon with the FDA, and are considered safe for use. I would like to repeat that. All batches that have been released for any patient use have met quality standards agreed upon with the FDA, and are considered safe for use.

Currently, our Takeda teams are working very hard to understand the root causes and identify potential solutions. This issue has led to the unpredictable supply interruptions for the SUP that we have been communicating over the past several months. We have already communicated the impact to the 100 microgram dose in the SUP, and we're watching other doses closely while we continue to work to address this issue.

One of the things we do to alleviate the risk of disruptions to any given dose for the SUP is generate a back-up or alternate prescription. We have done this for all patients on the 100 microgram dose already, and we're keeping an active watch out for our 75 microgram patients as well.

We will keep impacted patients and their healthcare providers informed. You may reach out to impacted patients with additional information as soon as this week. The upgrading challenges to SUP supply, these issues are also contributing to our latest projection that NATPARA's resupply timeline in the U.S. is at least 10 to 12 months from now, and Tom walked you through the stresses on our supply chain here.

Because even if we receive regulatory approval to move forward with fixing the device components, we have to ensure we have stable, consistent, and reliable supply for patients in order to bring the product back into U.S..

This brings me to the end of my section. I covered a lot of ground. I would like to thank you, also, on behalf of my Takeda colleagues, which I am representing here, for giving me this opportunity to describe the breadth of expertise of our dedicated team, share our efforts of expertise of our dedicated team with you, and our efforts today on overcoming the rubber particulate issues, and provide you with new information on the emerging Special Use Program supply challenge.

It is my sincere hope that I was able to clarify some of the complexities we face and address your questions. Thank you, again. Back to you, Cheryl.

Cheryl Schwartz:

Thank you, Stefan. So I know that was a lot of information, and I think, as you can imagine, it was really difficult to communicate some of this in just a letter, so I'm glad we have an opportunity to be able to explain it to you all in a little bit more detail. I'm just going to recap a couple of things because I know there was a lot thrown at you there, but Stefan's outlined really well that we're experiencing two separate but interdependent issues, right.

So we have the device component issue, for which we think we have a reasonable path forward, but then we also have the recent increase in those batches that are not meeting the criteria to release them for use, particularly the 100 microgram strengths which we're seeing more frequently.

And that issue is impacting our U.S. supply for both the SUP and for future resupply efforts. So some of the questions that we received about that is yes, they are connected and related to that underlying supply reliability issue. So what this means is that we have much to do. We still have to complete our regulatory submission with that proposed fix that Stefan just walked you through for the device components in the coming month.

And then we also need to get FDA's approval to our submission, and as you mentioned that is a roughly four to six month period, following that submission, that they take to review all of the data and information that we generate and come back with an opinion, and Stefan walked you through some of the potential outcomes of that review period.

And then lastly, we need to understand and address the recent manufacturing challenges we've experienced. So we can ensure we have a really robust device, as well as sufficient reliable supply continuity to come back to the market. I think that last part's really important because the last thing anyone of us wants to do is to come back prematurely and not have sufficient supply, and then cause more disruption for patients, especially given the risks that Tom walked you through that are associated with coming off treatments, and we know that many of you have experience that in the past.

So it's because of these three inter-related complexities that we're projecting the timeline that we shared with you of, at least, another 10 to 12 months from now before we think we can get back to the U.S. markets. And I hope that helps to explain a little bit of the why behind some of the timelines that we communicated last month.

So I know that there were also a variety of other questions that we also want to make sure we have time to get to. So I'm going to invite Tony Frangie to come back on here, and I'm going to turn it back over to Tony who's going to lead us through our Q&A session, and Tom and Stefan and I will kind of tag team some of the different questions that we have received, as I mentioned, through a variety of different channels. So Tony, I'm going to turn it over to you.

Tony Frangie:

Thanks so much, Cheryl, and thank you everyone for submitting your questions, either to us directly or through others. The first question we have is going to be for Cheryl. Cheryl, can you help us understand why you haven't shared more information about the extended timeline to resupply until now? To paraphrase, why hasn't Takeda been more transparent?

Cheryl Schwartz:

Thanks, Tony. So, where I started earlier this evening was that from the really – from the start, our main goal here with our communications has really been to make sure that you have all of the information that you need to make the best treatment decisions with your healthcare providers because we know you're dealing with a complex disease.

And so when we release information about timelines, it's because we want you all to have realistic expectations about the potential timing of the return of NATPARA to really guide all of those healthcare decisions. I will say that now I think we certainly realized that we missed the mark on striking the right balance in some of those communications, but I want to say again we're very sorry if this has led to additional confusion or concern.

That certainly was never our intent. Our intent is always to try to be as transparent as possible, but to be completely frank, sharing this kind of concrete information sometimes has been challenging, and that's for a few reasons. I think first, a number of issues that we've described to you today have really come up kind of real-time.

And often we've been in the midst of problem-solving mode, and it's really challenging to try to provide updates when we don't have enough information to not just give you updates but really clear solutions. And we didn't want to cause more confusion as we were working through the details and addressing the challenges that have arisen.

And then second, as you've heard so far this evening, the issues that we're talking you through have been pretty complex, both from a technical standpoint and also because of the interdependency issue and the way they affect one another. And so, we were really working through those interdependencies, again in real-time.

Every time that we were communicating with you all, we really believed that we were doing the right thing by prioritizing the information that you needed to inform decisions about your healthcare, but we do recognize the fact that you have requested and that you deserve greater transparency.

We hope that this session today is a step in the right direction to try to do better going forward and of course as we go through this process and we continue to kind of provide you with updates, we absolutely welcome your continued feedback on how we're doing and if there are areas that we need to do more, please do let us know.

Tony Frangie:

Thanks, Cheryl. The next question will be for Tom. Tom, this one relates to concerns around the recent supply issues. So the question is why can't Takeda just produce more NATPARA to ensure that patients enrolled in the Special Use Program don't experience supply interruptions?

Tom Koutsavlis:

Thank you for the question, Tony. And thank you – thank you to the community for the question and a very reasonable question as well. And indeed we have significantly increased supply. So in that sense we have actually increased supply to try and of course do exactly as you've described.

Unfortunately, despite maximizing our manufacturing capacity and producing a lot more product, we still find ourselves watching the SUP supply closely. Precisely with the reasons that Stefan mentioned. So I think he outlined why we're still struggling on the supply front.

So I know we've already communicated to you that we're projecting a supply shortage for the hundred microgram dosage strength and we're also keeping a very close watch on the 75 micrograms as well.

And it's possible we may be communicating with you later this week. So something we're keeping a very, very close eye on. I think until we identify the cause and can fix the manufacturing issues that we spoke about and that Stefan walked us through, producing more unfortunately doesn't adequately address the problem.

So as much as we've made more we haven't been able to address the issues that we're trying to manage. So we're committed to continuing to investigate the cause as we mentioned so we can address it as quickly as possible and obviously restore that product continuity for SUP patients.

I think one key that Cheryl mentioned and Stefan you mentioned as well, is that we absolutely want to make certain that we wouldn't bring product back to market until we know we can actually have product available for all of you.

And the priority obviously announced to make certain for those critical SUP patients yourselves that we absolutely are able to continue to provide product as much as possible. So that's our challenge and our push that the entire team is working on. So Tony, I hope that helped answer that question.

Tony Frangie:

No, thank you very much. And there is a broad team that's working on the issue for sure. Maybe Cheryl I can come back to you with the next question.

Cheryl Schwartz:

Sure.

Tony Frangie:

This is a more business and investment related question I think. Has the delay to NATPARA been caused by the financial liability of the overall program? For example, does the company benefit more from tax write-offs related to the SUP versus bringing the product back commercially?

Cheryl Schwartz:

Thanks, Tony. And while I appreciate where the question is coming from, I just want to be super clear that the answer to that question is no. The extended timeline to the U.S. resupply is related to the – all of these interrelated complex issues that we just shared with you tonight, it is unequivocally – it's not a question of profitability or priority focus, it's not a question of not supporting this product.

We absolutely understand the importance of this product and support its return to all of the patients who need it. I also just want to be super clear that there is not – there's no incremental tax benefit associated with operating the SUP. And operating the program is actually not treated as a charitable donation.

And ultimately our goal as a company is to deliver medicines to patients who need them. I mean especially patients like you who are living with rare diseases who have such high unmet need and who we've heard from all of you the impact that NATPARA has to your lives and the impact that not having NATPARA has to your lives.

So I just want to be super clear, there is no other kind of motive here. We have no intent of abandoning our efforts to understand and to address the issues that are impacting the timelines here. And we'll continue to do the work that's necessary, the work that Stefan and Tom and I have kind of talk to you through to do just that.

Tony Frangie:

Thanks, Cheryl. I'll ask this follow-on question to Tom. Tom, is there truth to the circulating rumor that Takeda is stalling with NATPARA until a competitor product such as TransCon PTH comes to market?

Tom Koutsavlis:d

Again, thanks for the question, Tony. And thanks to the community for sending in that question. So I think – let me start right off the bat and say unequivocally there is no truth whatsoever to any of these rumors.

I think many of you now I've gotten to know more personally and many of you have gotten to know me and I appreciate having had the opportunity to work with you for such a long time.

I can tell you that everything in our power, everything in our being has been to try and fix all of these problems and get the product back to you as quickly as possible. So rest assured that this is a full speed endeavor for many, many people.

You see the four of us on screen right now and I can you there are hundreds behind us that are helping to try and move this forward. So that way I can – I can assure you with certainty that there's absolutely no truth to any of those rumors. Our goal is to absolutely help the community as much as possible and I think I shared with you my own personal connection to hypopara.

Look, we absolutely know that there are other products in clinical development to treat patients for chronic hypopara and I think that's a wonderful thing. We very much support innovation and availability of treatment alternatives, especially in the space of rare and serious diseases like hypopara where there are limited options and there are not a lot of other things for patients and their families.

So as a physician myself I'm encouraged to see other things being developed and R&D really pushing in that direction. We've also been very privileged to share some calls and meetings and congresses with you with other companies as well who are working forward – looking forward to working on other treatment alternatives.

And again, it's a very exciting time for us in the community. So that piece is very critical. Now at the same time we also understand the risk of drug development and commercialization well enough to know that we cannot depend on all of those other options as potential solutions.

So we've seen what we've gone through obviously with NATPARA and we know that until a product is approved and on the market, we don't quite know where some of the other R&D will head to. So critically important that regardless of anything else that's happening with other products and other R&D that's coming, we have to keep our focus on addressing the challenges of NATPARA and making sure we get NATPARA back to you. I think that's critically important.

Tony Frangie:

Thanks, Tom.

Tom Koutsavlis:

Thanks, Tony.

Tony Frangie:

All right. Cheryl, I'm going to come back to you again. We've received several questions about the difference between the U.S. and markets outside of the U.S. For example, some patients have asked why the product is still available for commercial use in Europe while others have asked if Europe has the same supply issue that we are currently experiencing with the U.S. Special Use Program.

Cheryl Schwartz:

Yes, we have definitely heard lots of variations on the question and I think first-off we definitely want to acknowledge that we understand that it definitely can seem confusing or frustrating that this product is available in Europe. It's called NATPAR in Europe and that it's still available for patients in Europe.

The thing – the first thing to note is that the drug product is the same. And we did provide the same information to all the regulatory agencies where NATPARA or NATPAR as it's called in ex U.S. are available.

In the case of NATPARA, the U.S. regulatory authority, the FDA did request a voluntary recall, which we talked about earlier. While that option was not requested, I think as you all know by other regulatory authorities, including the European Medicine Agency.

So but in terms of the current supply interruptions, the interruptions for U.S. patients that are receiving NATPARA through the SUP are really influenced by the high number of cartridges that are required in the U.S. because of the single use requirements.

So that was the map that Tom shared with you a little bit earlier. And when you compare that to Europe the number of cartridges that are required for that population is actually much lower since we can use all 14 of those does in each cartridge for patients.

So just to give you a little bit of again, kind of an order of magnitude of what we're talking about here in Europe is there is just about as many patients outside of the U.S. on NATPARA as in the current U.S. SUP program.

So I think that's a little bit of a – kind of a misperception that exists was that – is that Europe population is the same – was the same as the pre-recall U.S. population. So it's a much smaller number. And so therefore one single release batch, we talked about batches earlier of product in Europe basically supplies enough cartridges with 14-day use to last a full year.

So one batch in Europe lasts a year for the number of patients that are currently taking it there. But by contrast with the single use requirements of the cartridges that are used in the SUP, that same one batch basically with the same number of cartridges because we're only using it one time, only provides about three to five weeks of supply coverage for SUP patients.

So that's why even though the underlying issues are the same, that's why we don't have the same consideration both on the regulatory front and on the supply front. And I know that's definitely something that we've received a lot of questions about and it can be confusing.

But I do think it just helps to understand the order of magnitude and really to just kind of dimensionalize what it means when you're using that one versus that 14-dose, and it's certainly a huge impact in terms of the supply implications.

Tony Frangie:

Thanks, Cheryl. Stefan, I didn't forget you – that you're on the line, so the next one is for you. This one is another question we received a few different variations of, so hopefully I've captured it correctly. Why haven't we offered patients a different device, or an alternative delivery option while we're working to fix the current device components of NATPARA?

Stefan Wildt:

Yes, so thank you for the question. Well, we want to assure you that since the beginning of the recall we've had a large cross functional team that spans regulatory, manufacturing, quality device, medical, and many other areas of expertise dedicated to trying to understand and address these issues.

So this team, as I described before has explored many different options, and just to be safe we also have engaged external consultants to pressure test and validate our findings.

And some of the options we explored were not technically feasible, while others are possible but would require extensive clinical or as we refer to them, human factor trials which are a regulatory requirement for medical devices to demonstrate that they are effective with a specific drug and in a specific patient population.

So you can see how all of this then can add also to – and result in even longer regulatory review timelines than the plan we outlined today. So we believe that the plan we've been working on offers the greatest likelihood of success and the fastest timeline, otherwise we would have picked something else.

That does not mean that we do not have other options that we are working on in parallel, but at this time we are focused on the approach we shared tonight which involves a new rubber stopper, and a new needle.

Thank you for that question.

Tony Frangie:

No, thank you, Stefan. We received it in a lot of different ways, whether it was can you mix NATPARA, draw out the doses into separate vials? Could you use some kind of pump to replace the device? But I think your answer is really appreciated there. So all of these things have been considered.

So Cheryl, I think this is our last question. So I'd like to come back to you with it. We received

...

Cheryl Schwartz:

Thanks.

Tony Frangie:

... several questions here as well in a few different formats – or forms. Really about our level of commitment to hypoparathyroidism as a category, to the patient community, and to bringing NATPARA back in the U.S.

Cheryl Schwartz:

Thanks, Tony. Again, I totally understand why we keep getting this question because I think that at the end of the day the result isn't yet where you all need it to be, right? And so it's a natural question, I think, to ask.

I want to assure you first and foremost that our commitment of Takeda to bringing NATPARA back, and to all of you – to the patient community, to your families remains really, really strong. I want to make sure that you understand that there is not a day that goes by that we don't talk about this program, that we don't work on this program.

The team has really kind of looked under every stone and I just want to reinforce kind of one other point which is that – that hasn't come up tonight, which is about Takeda itself as an organization.

And you know, we have something that we use as our kind of, our guiding principles, our core set of values, basically that we use to drive decision making at our company, we call it PTRB and it stands for patient trust reputation and business.

And really, that's how we make decisions, we start with patients, we think about trust, we think about reputation and then and only then do we think about business. And so the P in PTRB comes first really for a reason, and that's because all of us at Takeda are really committed to putting our patients first.

And so, really whenever we talk about NATPARA we always start with a discussion of all of you and the impacts of what's happening to you as we go through this process.

For example, Tom shared earlier some of the background that led us to propose the special use program to the FDA in the first place. When we set up the SUP and we continue to provide NATPARA to the enrolled patients in that program, which I said, there's now 430 patients that are enrolled that – who come with a direct request from healthcare providers to really meet critical patient needs.

And while that program was originally intended to address a short-term need, that would provide a bridge until we had NATPARA back, we've definitely remained committed to continuing the SUP as long as we have that supply, and that program is under very strict regulatory oversight.

So if obviously we can't predict exactly what the future holds, but it's certainly something that we talk about – again, every single day about the importance of that program, and we monitor it incredibly closely. It's also with the patient community in mind that we continue to commit the necessary – the time, the money, the resources, to bring NATPARA back to the U.S.

And so, I just want to assure you that that is happening on an ongoing basis. One other thing that we've heard quite a bit in terms of questions that's kind of related to the question you're asking Tony, and I know you've heard this one as well.

If Takeda is planning to sell NATPARA, and the answer to that is again, no. When Takeda acquired Shire and NATPARA came with that, we made the decision to continue to commercialize NATPARA as Takeda. And that decision was based on patient need.

We absolutely understand the critical nature of NATPARA to those who are suffering in this community, and we don't intend to sell it off now, or abandon our ongoing efforts that we've been committed to.

So I think that we can certainly all agree that we had hoped that the road to resupply in the U.S. would have been much shorter, and with far fewer curves and bumps. But we have stayed the course because we are committed to working to get NATPARA back to all of you.

So I hope that does help to address it. I think ultimately, it's going to come down to us continuing to prove to you that we're working on this, but it is something – as I said, we are very, very committed as an organization.

So maybe with that, Tony, thank you for the questions. I hope that we've been able to address many of the questions that you sent us. Before we close the call today, I do want to take time to thank you again for joining today, and let you know how much we appreciate all of your feedback. Sometimes it's hard to hear, but it's important to hear – and so we really, really do appreciate that.

And although, I hope that we've answered a lot of the questions that you sent us. I know that this new information might help you to understand why the delays are happening, but it certainly does not alleviate the suffering of the community, or help those of you who really continue to be so impacted by this terrible disease.

We said a few times tonight, we talked about all the different teams that are working on fixing this issue that are impacting our resupply timelines. And I know when you see our communications, and you see these letters that come out, or you see those of us on the screen today – you might just see like big corporation.

But I want you to know that Takeda is made up of all of these people. You know, the people that you see here today, the people on Tom, and Stefan, and Tony's team whose job it is to develop and bring medicines to patients who need it.

And you know, most of us got into this work because we're either scientists, or physicians, or actually a lot of us who work in this area were drawn to the field because we have direct personal experiences with rare diseases ourselves in our families. And so we do have a personal investment and commitment of people as well – above and beyond Takeda as a company.

So, kind of in closing today, above everything else that we've talked about today, we want you to know that we understand what this medicine means to you and to your families, and again, I want to just let you know that we remain committed to you, and to continuing to do everything in our power to bring NATPARA back.

So with that I'll thank Tom, Stefan, and Tony for joining me here today and for helping to answer, hopefully some or many of your questions. And a huge thanks to all of you for joining us this evening. We commit to staying in touch, we hope that you'll commit to keeping on giving us the feedback that we need to do better, and we hope to be able to provide you with additional updates soon.

So, thank you very much and hope you have a good evening.

Tom Koutsavlis:

Thank you.

Stefan Wildt:

Thank you.

Operator:

Thank you again for joining us today. This concludes today's web conference, you may now disconnect. Have a good day.

Thank you for listening to the recording of this call.  Please stay on to hear Important Safety Information about NATPARA.

Important Safety Information

What is the most important information I should know about NATPARA?

Warning: Possible bone cancer (osteosarcoma).

  • During animal drug testing, NATPARA caused some rats to develop a bone cancer called osteosarcoma. It is not known if people who take NATPARA will have a higher chance of getting osteosarcoma. Tell your doctor right away if you have pain in any areas of your body that does not go away, or any new or unusual lumps or swelling under your skin that is tender to touch.
  • NATPARA is only available through the NATPARA Risk Evaluation and Mitigation Strategy (REMS) Program. The purpose of the NATPARA REMS program is to inform patients about the potential risk of osteosarcoma associated with the use of NATPARA. For more information about this REMS program, call 1-855-NATPARA (628-7272) or go to www.NATPARAREMS.com.

NATPARA may cause other serious side effects, including:

High blood calcium (hypercalcemia)

  • NATPARA can cause some people to have a higher blood calcium level than normal.
    • Your doctor should check your blood calcium before you start and during your treatment with NATPARA.
    • Tell your doctor if you have nausea, vomiting, constipation, low energy, or muscle weakness. These may be signs that you have too much calcium in your blood.

Low blood calcium (hypocalcemia)

  • People who stop using or miss a dose of NATPARA may have an increased risk of severe low blood calcium levels.
  • Tell your doctor if you have tingling of your lips, tongue, fingers and feet, twitching of face muscles, cramping of feet and hands, seizures, depression, or have problems thinking or remembering.

Tell your doctor right away if you have any of these signs and symptoms of high or low blood calcium levels.

Who should not use NATPARA?

Do not use NATPARA if you are allergic to parathyroid hormone or any of the ingredients in NATPARA.

What should I tell my healthcare provider before using NATPARA?

Before you start using NATPARA, tell your doctor about all of your medical conditions. Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of NATPARA?

NATPARA may cause serious side effects like allergic (hypersensitivity) reaction, including anaphylaxis. Tell your healthcare provider or get emergency medical help right away if you have any of the following symptoms of an allergic reaction:

  • swelling of your face, lips, mouth, or tongue
  • breathing problems
  • fainting, dizziness, feeling lightheaded (low blood pressure)
  • fast heartbeat
  • itching
  • rash
  • hives

The most common side effects of NATPARA include: tingling, tickling, or burning feeling of the skin, low or high blood calcium, headache, nausea, reduced sense of touch or sensation, diarrhea, vomiting, pain in joints, too much calcium in urine, and pain in limbs.

These are not all the possible side effects of NATPARA. For more information, talk with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see Prescribing Information, Medication Guide, and Instructions for Use available on NATPARA.COM.

Thank you!

On April 29, 2020, Takeda conducted a call with the Hypoparathyroidism Association to update the hypoparathyroidism patient community on the NATPARA recall. Listen to the complete recording, or read the full transcript, below.

This is an audio recording of a call conducted on April 29, 2020 between Takeda, the makers of NATPARA (parathyroid hormone), and the Hypoparathyroidism Association, intended to update the hypopara patient community regarding the ongoing NATPARA recall.  Please note the recording has been edited for clarity and length.

NATPARA is a prescription parathyroid hormone used with calcium and vitamin D to control low blood calcium (hypocalcemia) in people with low parathyroid hormone blood levels (hypoparathyroidism). It is only for people who do not respond well to treatment with calcium and active forms of vitamin D alone, because it may increase the possible risk of bone cancer (osteosarcoma). NATPARA was not studied in people with hypoparathyroidism caused by calcium-sensing receptor mutations. It was not studied in people who get sudden hypoparathyroidism after surgery. It is not known if NATPARA is safe and effective for children 18 years of age and younger. NATPARA should not be used in children and young adults whose bones are still growing.

During animal drug testing, NATPARA caused some rats to develop a bone cancer called osteosarcoma. It is not known if people who take NATPARA will have a higher chance of getting osteosarcoma. Tell your doctor right away if you have pain in any areas of your body that does not go away, or any new or unusual lumps or swelling under your skin that is tender to touch.

Please listen to this audio in full for additional Important Safety Information about NATPARA. Please also see the Prescribing Information, Medication Guide, and Instructions for Use available on NATPARA.com.

Operator:

At this time, I'd like to introduce Bob Sanders, chairman of the HypoPARA Association.

Bob Sanders:

Hey, everybody, I just wanted to again, thank everyone for calling in. What's up?

I'm sorry. Anyways, yes, it's Bob Sanders. I just wanted to thank everyone for calling in and thank Takeda for setting this up and giving them an opportunity to – giving all of us an opportunity to ask them some questions and hopefully get some good information.

We got Dave Murdoch, he's going to be kind of running the communication side of this. I'll let him take over and introduce himself and kind of go through the schedule.

Dave Murdoch:

Thanks, Bob. My name is Dave Murdoch. I lead Corporate Communications for Takeda's Rare Business Unit in the U.S. Thank you all for joining and Bob for joining as well. Danette Astolfi is also here from the HypoPARA Association.

I'm also joined by my Takeda colleague Cheryl Schwartz who is Senior Vice President and head of the U.S. Rare Business Unit, as well as Tom Koutsavlis, head of U.S. Medical Affairs. And obviously we'd all like to be with you in person like we were in October but COVID has taken care of that but glad that we could be with you virtually.

Just a couple of housekeeping items before we get started. You'll be hearing some brief updates on Natpara from Cheryl and Tom, and they'll respond to a number of questions that have been pre-submitted through the HypoPARA Association Facebook page.

And lastly, I do want to advise our speakers that this call is being recorded. At this point, I'll hand it back to Bob for – if you have a few more brief words of introduction.

Bob Sanders:

Yes, I mean, I – sure. I guess.

Dave Murdoch:

You don't have to.

Bob Sanders:

No, yes, I pretty much said it. Like you said, I was the chairman of the board of the association. And Danette – I think Deb's in the group as well listening on. So, again, if you guys have any questions, e-mail them to Danette and she'll kick them over to us. And hopefully we can get them answered today.

Dave Murdoch:

All right. Thanks, Bob. At this point, I'll turn over to Cheryl to say hello, give a welcome.

Cheryl Schwartz:

Great. Thanks, Bob and thanks, Dave. Good afternoon, everyone. As Dave mentioned, my name is Cheryl Schwartz. I head up Takeda's U.S. Rare Disease Business Unit and that includes our endocrinology, therapeutic area and Natpara specifically. So I had the pleasure of speaking with some of you earlier in October and I look forward to being able to speak with you a little bit more today.

First off, I just like to thank the HypoPARAthyroidism Association for inviting us to participate in this call. We certainly know that, you know, during this very unusual time when so many people are self-isolating due to concerns about COVID-19., you know, we especially appreciate the chance to connect with you all and hear how you are all doing and be able to address some of your questions that we've heard.

We definitely recognize that the news that we shared earlier this year that Natpara was not returned to the market for at least a year was very, very difficult for the community to hear. I want to reiterate that we deeply regret that we're not able to bring Natpara back as soon as we initially hoped. And while unfortunately, we still don't have a definitive timeline to share with you today. We did want to try to do three things today in response to some of the questions that we've heard.

The first is we – we've heard a lot of questions from the community about with everything going on in the world with COVID-19, has this impacted any ongoing work to bring Natpara back? And are efforts still ongoing? So first and foremost, I want to reassure you all that this remains a top priority for us. There's a very large team of people at Takeda who continue to work on this every day. And we'll share a bit more on some of those details with you all of that ongoing work.

Second, we've heard some concerns about continuation of supply with regard to the Special Use Program also in the light – in light of COVID-19. I wanted to let you know, and Tom can speak more about this in a moment, that based on our current assessments of the global supply chain, we do not anticipate any potential supply disruption due to the coronavirus outbreak. The program remains open for continued applications as needed. And we are tracking the situation as it evolves.

We'll make – take any necessary measures to continue supplying our therapies to the patients that we serve. And of course, we will keep your healthcare providers and the patient advocacy organizations informed of any material changes in status, including of course, the HypoPARAthyroidism Association. I also wanted to mention that we had previously communicated to patients enrolled in the Special Use Program, that shipments of Natpara will now include two weeks of single-use vials instead of the original one week of treatment to just ensure greater efficiency and access to medicine.

And then, finally, in addition to the Natpara recall, we also know that people who are living with chronic diseases have very specific concerns in these unprecedented times. So we wanted to take this chance to hear from you all directly and really better understand how we can help to support you. While we continue to work on bringing back Natpara.

So following this call, in addition to getting feedback from you on, thank you for submitting your questions in advance of the call as well. We'll be working with an outside organization to reach out to some of you to help us better understand your needs during this time. So there will be some follow-up communications that will occur.

So thank you again, so much for the opportunity to speak with you all and for sending in your questions for today's call. We look forward to answering as many of those as we can. But before we jump right into that, I'll just turn it over to Tom for a few opening comments as well.

Tom Koutsavlis:

Well, thank you very much, Cheryl, appreciate that. Thank you, Bob, as well. For those of you who don't know me, I'm Tom Koutsavlis, I'm the head of U.S. Medical Affairs for Takeda. I think I've met many of you over the past many months and years and I certainly enjoyed time together in Denver as well back at the end last year. I'm sorry, we can't be together in person. And unfortunately, that is the reality we're all living with today. But I do very much look forward to the next opportunity when we get a chance to connect in person again, too.

So a big thank you to the HypoPARA Association. Like Cheryl mentioned, certainly a very appreciative for you asking us to be on this call with you today. Hopefully, we'll be able to share some thoughts and ideas and get your thoughts and ideas as well.

So now, I think – I very much wanted to reiterate what Cheryl mentioned. Our entire organization recognizes how important Natpara is to all of you, how critical it is in this patient community. And I can assure you that our commitment remains unwavering. As strong as it always has been, we will not give up until we work what we need to do with Natpara to make sure that you have the availability of that product for the entire community.

Patient safety is so critically important. It's our highest priority. And we've done a lot of work on the medical side in terms of what next steps we can do to continue to help you.

One of the big pieces, as you know, has been the special use program. It's been a critical opportunity to make certain that those patients, those of you who have had very critical life-threatening complications as a result of the discontinuation still have access. The program, as you know, is certainly challenging in some respects, because it's very restrictive. We have had to work very closely with the FDA to make certain that we are allowed to progress with what we are able to offer within the limits of the program itself.

And we had actually originally anticipated that it would only be for a very extremely limited number of patients about 30 or 40 patients, which translates to about 1 percent of the total number of U.S. patients on therapy. But actually, over the last many months, we've been able to accommodate now more than 400 patients. So we have over 400 patients at the moment who've met that stringent criteria that was set by the FDA. That is a critical requirement for the program. So we're very pleased that we really are able to help as many people as we can.

We're also looking at cases daily and new cases that are coming in during the week knowing that obviously now with COVID-19, this is a very stressful time for patients, certainly patients with pre-existing health conditions. And so we're keeping a very close eye on that evolution of how you are coming to us with questions and requests and what needs you may have. What I want to make certain to relay to you is that please absolutely keep your endocrinologist informed of any changes to your condition. Anything that worsens or anything that differs, especially as we go through COVID-19 together now.

And then if there are any changes or any concerns, your endo can absolutely submit or resubmit your file to Takeda for reconsideration as part of the Special Use Program. So please don't hesitate. If you do see changes in your condition to reach out to your physician so that that can circle back through the program for us.

We understand obviously that the program is limited, and it won't address the needs of the majority of you. But it's something that we want to make sure we continue to absolutely provide so that – for those critical patients that continues to exist. We're continuing to work with the FDA. I think I mentioned this back in October when we spoke, and we're staying very close with our communications with the FDA to work on bringing Natpara back more permanently to the community. And this remains a very high priority. So certainly that has not changed, and we continue very active communication and conversations with the FDA.

So there's a lot of moving parts, as you know, and I appreciate Cheryl giving you the update in terms of where we are right now. And certainly with COVID, specifically, ensuring that we can continue the Special Use Program. So that does not change and also make certain that as things may change with you going through COVID that you're able to circle back into the program as quickly as possible with any new or reconsideration of applications.

And I think I'd mentioned to you – to all of you back in Denver and I appreciated you spending some time with me then that hypoparathyroidism is very near and dear to my heart. And I mentioned that actually during my residency, one of the first patients I ever saw and treated was a hypopara patient. And it continues to be an area of importance for me, both professionally and personally. I very much appreciate hearing from many of you and certainly some of the positives and how you've been able to manage through hypopara but also a lot of the negatives and challenges that you face.

And it's important for all of us to really be able to understand and hear that and really be able to recognize how critical it is for us to continue to do the work that we do to be able to keep Natpara available to certainly the most critical groups and then to get it back as soon as possible. So, again, a big thank you, for you being available and spending time with us today. And again, my apology that we can't be together in person but look forward to doing that soon.

And then perhaps I can pass it back to Bob, and Bob, you can help us walk through the next steps in our call.

Bob Sanders:

OK, so from here on out, what we'll do is, like, so we got a list of questions that we collected off our Facebook page. And we'll kind of go through them. And I'll ask them one by one and get Cheryl and Tom an opportunity to address those questions before we move on to the next. And again, I'd like to encourage you, if you have any other questions or sparks of conversation, just email those to Danette and she will get them over to us and then we can hopefully address those at the end of this call.

So question one is, why can't we use a syringe to draw up the medicine and administer or use single-use cartridges like special use?

Tom Koutsavlis:

Thanks, Bob. So maybe I can jump in and take that question. This is Tom again. So at this time, unfortunately, because of the regulatory restrictions and limitations of the Special Use Program, we aren't able to make any single product available for syringe drawing or using it in that manner. So the program is it's quite restrictive, it's very clear on what we can and cannot do within that program, obviously with conversations with the FDA. So we need to be very cautious that we can only proceed as the current program is designed.

If we were to modify, and that's part of the process, we're looking at enhancing the longer term solutions, anything we do in terms of modifying the device or the product itself requires many regulatory steps in terms of getting all of the appropriate testing done and then the appropriate approvals with the regulatory agency. So unfortunately at this time, we just can't use a new device or syringe with the existing Natpara products, so that is not an option.

Bob Sanders:

OK. And then the second one kind of ties into that a little bit. It says, what if you use a different supplier for the cartridges? And then could that get Natpara back on the market sooner?

Tom Koutsavlis:

So that's an excellent question, and we've received a lot of suggestions and insights for new suppliers and manufacturing partners that we can connect with for this work. And what I can confirm is that we've actually identified those vendors and partners to be able to do that work in terms of new supply. And what we need now is to move forward once that FDA conversation has happened, and the FDA provides feedback to our proposals for the work that those vendors would do.

So thank you for your suggestions and partnerships that we can put in place, which we have. And so now we're awaiting that FDA feedback before we can go ahead and head to the next step. So again, very appreciated, and great question.

Bob Sanders:

OK. You have anything to add, Cheryl, to any of those questions?

Cheryl Schwartz:

No. I don't think so.

Bob Sanders:

OK. All right. So moving on is, what is the current status of the return of Natpara? And is the plan for Natpara to return to the U.S. market in early 2021?

Cheryl Schwartz:

Thanks, Bob. I'll take that one.

So, as I mentioned earlier, you know, we communicated to everyone back in January that Natpara would not return to the market for at least a year. And I know we have discussed previously, some of the reasons why and specifically the fact that changing a component or different components of a device can be a very long and complex process.

As Tom mentioned, we've been in very close communication with the FDA on all of the different aspects and concerns that they have specifically regarding the safety for the product. And I will share that we have made very important progress since the September recall on looking at all of the different device remediation options, some of which Tom just kind of ran you through. So we have, over the last several months, explored in consultation with FDA, multiple options to correct the Natpara device. We have conducted really thorough testing and data analysis of each one of those approaches. You know, and at this point, we believe we have a path forward. But that's still requires additional discussions with the FDA, while we simultaneously continue to advance our testing and address ongoing FDA questions.

So as we said previously, you know, this is really largely going to be in the hands of the FDA's level of response to the data that we've generated. And, of course, it's the goal of both the agency and Takeda to safely bring Natpara back to the community as quickly as possible. So we're very hopeful that we'll continue to have good collaboration and discussions with them moving forward.

Bob Sanders:

OK. So is there a plan in place with the FDA?

Tom Koutsavlis:

Maybe I can jump in, Cheryl. So, yes. Thanks, Bob. And indeed, as you've heard from Cheryl, we've had a lot of conversations with the FDA over the last many months and we continue to be very close contact with the agency at times, it really is sort of daily/weekly contact, which is great to have that connection. And as Cheryl mentioned, obviously, the agency as well as Takeda are very, very keen to make certain that we can move this forward as quickly as we can.

So we do have a path forward, I think we've done a lot of that testing that Cheryl mentioned, and a lot of the work that's been done now to give us a good understanding of what we need and what will be required. And so the really – the important next step now is to get that agreement from the FDA on all of those requirements and do whatever additional testing and data they may ask for as we walk through that. So the plans are there. And I think once we get to that next step of getting an agreement with the FDA on what that looks like in more detail, then we'll be able to get to that next phase. So, well on its way and I think, hopefully, that'll continue to be that close commitment that we have with them.

Bob Sanders:

Awesome. So, will there be any changes to the processes for receiving Natpara once it returns to the U.S. market?

Cheryl Schwartz:

Yes, thanks, Bob. I'll take that one. I think, you know, since we are still in discussions with FDA on the path forward, it's still basically too early to know whether or not there's going to be any changes to the process for receiving Natpara, when it returns to the U.S. market. We'll, of course, continue to keep you all and, you know, your prescribing physicians informed along the way as we go. But we don't have any definitive answers on that yet. But we will of course, work through any additional elements to make sure that any product that we're bringing back to patients is done so in a way that we could maximize access for all of you as well.

Bob Sanders:

OK. And so the other rumors that have been floating around is on to the next question, I guess is, is it possible that Takeda may discontinue Natpara?

Cheryl Schwartz:

Yes, I can take that one too. Yes. So we've definitely heard the rumors. And we've seen, you know, on certainly discussions on, you know, had questions that have come to us directly around this. And, you know, we've definitely heard people's concerns. I think the one that kind of comes up the most is that, you know, will Natpara fall through the cracks in a big company like Takeda? Is it something that is being prioritized?

And, you know, I want to just mainly assure you that our commitment to patients like you is at the center of everything that we do at Takeda. We have kind of an internal philosophy around patient-centric decision making. So the relative size of one patient community versus another or one product or another doesn't really have any bearing on how we make decisions internally or on that ongoing commitment.

In fact, the team that continues to work on this issue daily, as Tom mentioned, you know, includes there's more than 30 people that are working on this kind of internally on a daily basis. And many, many more who are working to test potential solutions in our lab, in our manufacturing organization that are looking at all of these different scenarios. So we have, you know, a very large effort that's ongoing internally to ensure that we're really looking for every possible solution to expedite return of Natpara to the patients who need it.

We also know that the teams within the FDA are equally dedicated to working with us. As Tom mentioned, we've had really robust discussions with them. And so I think that we're really aligned in our goal to bring this very critical medication back to you all as quickly and safely as possible. So I really wanted to take a moment to reassure people that this does remain a very high priority for Takeda as an organization. But more importantly, for all of us who work on this program, Tom and myself and all the other members of the team are very incredibly committed to finding a path forward. So hopefully that helps address some of those rumors that have been swirling around.

Bob Sanders:

OK.

Tom Koutsavlis:

And Bob, if you permit me, maybe if I can just also – this is Tom, again.

Bob Sanders:

Yes.

Tom Koutsavlis:

If I can just echo Cheryl's comments because I know you get to see and hear from us, from the two of us, Cheryl and myself, on a few occasions along the way. But there is an army of folks behind us that are working so diligently to get Natpara back and are so committed and I just want, on their behalf because they can't all be on the call with us. I just wanted to maybe express their amazing dedication and their passion to make certain that they can help you.

And, you know, I wish I could kind of put a picture or video up of the group and the whole team. And there's so many different folks that are working on a daily basis, whether it be in our manufacturing sites, in our labs, in our medical groups, in our regulatory teams, in our commercial group. So, rest assured that each and every one of those folks is absolutely 100 percent committed. And that they – you know, we meet daily and so there's no question that that absolute, you know, it's heartwarming to see. And I wish I could share it more with you. But I just wanted to reiterate that I know it's not easy going through this time, and it's not easy hearing rumors that come out. And I just wanted to help dispel some of that because there's a great group of people behind us that you see more regularly that are really committed to you.

Bob Sanders:

OK. Just moving on is, how is the risk of Natpara withdrawal being documented and analyzed? And how should patients document adverse events associated with the discontinuation of Natpara?

Tom Koutsavlis:

So great question. Sorry, Tom, again, I'll take this one. So all of you, all patients should be communicating with your healthcare practitioners to provide any complications, any concerns any adverse events, all of that should be reported almost as quickly as possible following the discontinuation. And then obviously, any information that comes to us within Takeda, we also share with the FDA. We have regulatory obligation to do that, which we do.

And you yourselves also can report directly into the FDA's MedWatch on their website. What's called MedWatch is the system that they use to monitor adverse events. So all of that data is all constantly being fed into the regulatory agency. So that the agency can be aware of what's happening in terms of post discontinuation, what some of the issues have been with respect to adverse events.

So please, I encourage you to continue to do that. When you do have any concerns and issues, do not just keep it to yourself, but please do share it with your healthcare practitioners. And if they're not reporting them to the FDA or if they haven't had the opportunity to do that, either share it with us and we will report it in or directly to the FDA themselves.

Dave Murdoch:

Tom, this is Dave Murdoch. Sorry to interrupt here. We did just get a question in about whether it would be possible to – for Takeda to track adverse events and present them periodically to the community. Is that – I know the FDA – is the keeper of the adverse events is that something that that we would be able to do?

Tom Koutsavlis:

So a great question. Thanks, Dave for jumping in. So we collect what we receive. But bear in mind, as you can imagine, it's only going to be a small portion of what comes in because many either go directly to the FDA, or through the healthcare practitioner approach. And so what we have is a piece but not the entire picture. And so we will also look at that, we will analyze that data and we do analyze that data on a regular basis to understand what has been happening.

So there's nothing I would be able to share with you of value now. But other than just simply qualitatively, saying, obviously, what we see as the adverse events are those that you're experiencing with a discontinuation. So, the key is to keep bringing them in so that the entire data set will be held with the FDA and then more analysis can be done there.

Bob Sanders:

All right.

Tom Koutsavlis:

OK.

Bob Sanders:

So the next one we have, is there a way to notify endocrinologists about Natpara? Some patients are still having or see physicians who have not heard of it yet?

Tom Koutsavlis:

That's a good question, Bob. And indeed, it – obviously, it's a unique product, but also a fairly rare disease as all of you know. And so not everyone has heard of – first of all, not everyone has heard of hypopara and many have not heard of Natpara either.

So, unfortunately, because Natpara itself is under a recall now in the U.S., we're not permitted to proactively contact physicians to educate or increase awareness for them of the product. Having said that, though, our patient services team continues to communicate with the Natpara prescribers and when we have important information to share including changes to the Special Use Program, or any updates about the recall, status of the recall itself. All of that will be communicated to those prescribers.

So, unfortunately, we can't be more proactive to folks who have not prescribed or are not aware of the product right now because of the recall. But anyone who has become aware or is a prescriber, definitely has access to all information from us. So we will certainly continue to do that.

Bob Sanders:

OK, so if the Coronavirus outbreak causes shipping delays, how will these be mitigated for the Special Use Program?

Tom Koutsavlis:

Another good question, Bob, and maybe I can take that as well if that's OK, Cheryl. So first off, obviously, just a brief note on coronavirus and COVID, in general, and I just wanted to extend a heartfelt wish that everyone is healthy and you and your families. I know this is not an easy time. And certainly, with patients having underlying hypopara, that's always a concern.

So we've kept a close eye specifically on COVID. And the endocrinology community as a whole to see what we can do and how we can support and as a company, we're wanting to continue to do that, specifically for the Special Use Program and shipping and supply. So based on all of our current assessments of our global supply chain, we don't anticipate any potential supply disruption because of COVID. So we're tracking the situation very closely. We're tracking it at every step as it evolves. And if there’s anything that we – if we need to take any additional measures or whatever we need to do to make certain, we continue to supply. That is our priority to continue that supply for all of the patients that we serve.

And that's not obviously only for Natpara, that is for all products. Natpara is in the unique Special Use Program so it has an even more hyper vigilance on it. And obviously we'll keep your healthcare providers and patient advocacy groups and HypoPARA Association informed of any changes that may happen. So we're keeping an eye on supply. It's a daily exercise for us and so far, so good. So that's important to keep in mind.

If you're enrolled in the Special Use Program, you will continue to receive your Natpara directly from TheraCom. And as we've previously communicated to patients that are enrolled in SUP, each shipment will now include two weeks of a single use vials, just as Cheryl mentioned instead of the one week previously. Just to continue to ensure efficiency and a better guarantee that you have supply in hand.

And then all of the other program elements, nothing changes. So everything stays exactly the same, including retrieving the used product. I hope that's helpful, Bob.

Bob Sanders:

OK. So if there is a delay in the Special Use Program shipment, would patients be allowed to use the cartridge twice?

Tom Koutsavlis:

That's a great question, but because we're under very clear regulatory guidance and scrutiny, so at this time, patients receiving Natpara under Special Use are prohibited from using more than one dose per cartridge. They're very stringent requirements that we have from the FDA and we kindly ask that patients absolutely abide by those requirements.

And it's important to mention that if you fail to be able to comply with those requirements, it could put enrolled patients at risk of being discontinued. So it's critically important that we stay within the boundaries and the guidelines of the FDA and not deviate from those. But please do follow the guidelines of the program as diligently as possible.

Dave Murdoch:

Tom and Cheryl, it's Dave again, apologies. We just did just get a question, if there is a delay in an SUP shipment, who do patients contact?

Cheryl Schwartz:

Yes, I can answer that. I mean, so the patient should work through their patient services contacts the same way that they do for any other considerations. If they have any other issues and they'll be able to advise them on next steps.

Bob Sanders:

All right, thank you. What is the limit to how many people can be accepted to the Special Use Program with the current manufacturing? And can or will the Special Use Program be expanded to more people?

Tom Koutsavlis:

That's a great question. Thanks, Bob. So I'll take that, it's Tom again. So as you know, the program itself is focused on supporting patients with extreme risk of life-threatening complications. So it's a very specific and narrow focus and we've been thankful to be able to get to that 400 patient group from the initial 30 or 40 we were thinking of. The challenge is you can imagine is that because we're prohibited from using more than one dose per cartridge, instead of getting the 14 doses from each cartridge shipments, patients just administer one and the other 13 get discarded.

So you can imagine, you know, a patient taking Natpara would normally would use about 26 cartridges per year and suddenly they're using all of that up in about a month. So the challenge is the supply with that. So we're still doing our best to make certain that anyone who needs the product and is applying into the program and follows – falls under the strict life-threatening complication guidance that has been given, and the restrictions have been given with the FDA, will have access. So that's still a key priority.

Unfortunately, we just simply don't have enough supply to open it beyond that at the moment. And so that's been a challenge from day one. But we will continue for those patients who need it and will fall into the criteria to make it available.

Bob Sanders:

All right. Will the increased risk associated with the emergency room visits be considered in assessing patients for the Special Use Program?

Tom Koutsavlis:

Thanks, Bob. That's a very important question. So obviously, a lot has been happening with COVID now and what that means in terms of going into emergency or going into other settings. And so the big key here is that if you have any change in your condition, and either you've applied or have not yet applied into the Special Use Program, please absolutely speak to your healthcare professional so that they put in an application accordingly. If there's anything that's changed in your condition, if anything that requires emergency room visits, et cetera, that's different from what you've been accustomed to previously, something that has changed, we want to see those applications coming in.

So please do that if you have not. And obviously, maybe many of you on this call have participated in the SUP. For those who haven't or those that are not on this call, if you can share that with your colleagues and then make certain that healthcare practitioners are doing that when there's a change in the patient's condition to please do that, that's critically important. And obviously, there's no mechanism for us to know that other than your healthcare practitioner advising Takeda of that. So, we need that information. So thank you for that.

Bob Sanders:

OK. So if a patient has the history of hypocalcemia with infections, could their contract – could their contracting a virus make it eligible for Special Use Program? Can a patient be allowed to participate in a case like this?

Tom Koutsavlis:

So again, great question, very similar to the previous question as well. If there's any change in your condition, whether it be because of an infection, because you see differences in the hypocalecemia worsening, you absolutely please do reach out to your healthcare practitioner so they can reach back out to us. So, again, yes, absolutely. We want to make certain that you have access to sending an applications accordingly for the Special Use Program. Even if you've previously not been accepted, if there has been a change, we want to see that.

So Bob, are there still other questions maybe. Yes.

Dave Murdoch:

We might love …

Bob Sanders:

OK. Sorry, I had you on mute. My bad, my bad. Sorry about that.

Dave Murdoch:

That happens. No worries.

Bob Sanders:

Yes. So who is on the medical review committee and can I contact them with questions?

Tom Koutsavlis:

I'll take that Bob, if you don't mind. So the committee consists of several board certified endocrinologists both internal to Takeda, but as well as external endocrinologists who have a lot of experience, significant experience treating patients with hypopara. But in order to make – to enable this committee to stay objective and to work and do the work that they do, we can't identify them by name or make them available to patients and/or caregivers. They're very isolated groups that are assessing these cases and adjudicating them with their expertise in an isolated manner. So they can say stay distant and so that – I think it's important to keep them independent.

Bob Sanders:

OK. And so why has there been so little information?

Tom Koutsavlis:

Sorry, Bob, specifically to the committee or is that a general question?

Bob Sanders:

I'm guessing it's referring to the recall in general.

Danette Astolfi:

I can step in, this is Danette. I think that question was specifically related to – around the plan, that there's little public information around what exactly is happening so that patients can just have some comfort level around what was going to happen and what the next steps are for Natpara to return to the U.S. market.

Cheryl Schwartz:

Yes. I mean, I can address that a little bit Danette. I think, you know, I know it's super frustrating that it seems like we're not sharing more information. I think that there's a couple of reasons for that. One is I think a lot of what is happening right now behind the scenes is it's really highly technical and addressing the concerns that FDA has raised. And it's really about doing the due diligence on the testing and generating additional data to be able to share with the agency.

I think the other issue is that, you know, we are not in a position to share more information until we have, you know, tangible feedback from the agency. And I think the last thing that we want to do is in any way jeopardize the program by, you know, sharing something that's not correct or that could change in the future or new information is emerging or new data is being generated. And so until we have an agreed upon path forward with the FDA, you know, anything else at this point would really be speculation on our part. And so I think we're really trying to balance this idea of providing information and keeping you in the loop on what we're doing, but also making sure that we're not, you know, speculating basically until we have a definitive path forward.

And I absolutely get that’s frustrating. And frankly, you know, it's difficult for us as well, because we want to be able to give you the answers that you all, you know, really, really need. But this is really the pathway that is required. And the regulatory agencies, you know, have these different processes in place to ensure patient safety, and to make sure that we're really, you know, doing everything that's necessary to bring the product back in the most appropriate way possible. And so that's really the rationale for why it appears that we don't, you know, we're not sharing more information.

Dave Murdoch:

OK.

Bob Sanders:

Did it again. Did it again. OK, so should people continue to contact the FDA and their legislators? And is this helping with the recall?

Cheryl Schwartz:

Yes. I'll answer that one. I know the question has come up in the past as well. And, you know, in our side, you know, we can’t advise patients or, you know, any consumers about whether or not to reach out to the FDA or their legislators. You know, we, at Takeda, continue to work really closely with the FDA. We've been really appreciative of the agency's collaboration and the feedback and we really are working together to come up with the best path forward to resupply Natpara.

We'll continue to do that to work really diligently with the FDA on this issue. We'll continue to keep you informed, but we are not in a position to make any other recommendations to you all on an individual level.

Bob Sanders:

OK. I think that's about all we have. We had a couple other questions. I think Dave was going to go ahead and read out if that's – if you're ready to do that, Dave.

Dave Murdoch:

Thanks, Bob. I'm looking through – I think we've covered most of them. There is a question about – information about upcoming clinical trials, but I assume that's too early to talk about since we're still in discussions with the FDA. But I'll just throw that to Tom or Cheryl.

Danette Astolfi:

Sorry, this is Danette Astolfi. So I think one of the questions specifically was around how patients would be informed of a clinical trial, how they could receive any information if there was one that was planned. Would there be any sort of communication cycle either from the association or from Takeda to inform patients?

Tom Koutsavlis:

Danette, so – this is Tom, maybe I can take that. And then thank you for the question, excellent question. And as you know, with any study that we do, and it is across the industry, we will go directly into working with investigators, healthcare practitioners to understand who is able to conduct studies and what they can do. And then obviously, as they look for patients that can enter these studies, they very much reach out to the patient community. And at times, we will reach out even more publicly and mention that the studies are ongoing.

So when a study does kick off, it that does become a public document that we can share. And so patients would be aware– as will their health care practitioners. So rest assured, if there's that direction that we take that we're required to do some form of a study, we will absolutely get to you either through HypoPARA Association, which may be a way to be able to reach all of you as quickly as possible, through the healthcare community, endocrinology teams that will be running studies, all of that will come to you. So you'll be aware very, very quickly.

Again, not knowing now which direction the details may bring with the regulatory agency and waiting for their input as well and whether additional studies would be required. We wouldn’t know today to be able to share any of that, but be rest assured that if there is a study that's needed, you'll be the first to know. And hopefully, you'll be able to participate as well, of course. But I think if all goes well, I'm hoping that we don't have to go through any large clinical trial programs in order to be able to get Natpara back to the market, and that's the goal. But until we hear from the FDA, we won't have that information.

Dave Murdoch:

I had a question. Are there any plans for research that would better characterize the risks of discontinuation of parathyroid hormone?

Tom Koutsavlis:

So thank you, but I can take that as well. And I think it goes back to one of the earlier questions we had as we look at adverse events that come in. I think that's probably the best research we can conduct right away, where we actually have data that comes in to the FDA with adverse events that have occurred since discontinuation. And I think it reinforces what we've known very well and what you all know, as patients, what you struggled with before Natpara.

Beyond that, we'd have to conduct more formal studies. So to get a little bit more deeper into that information. But again, I don't think – I'm sharing anything with any of you that you don't already very much know yourself clinically as you individually go through this. So what we can amalgamate with the FDA adverse event work will be something FDA will do as well.

Dave Murdoch:

OK, great. That's all the questions that have come in. Maybe Tom and Cheryl and Bob, I just hand it back to you just for some closing comments.

Bob Sanders:

Yes, I'll start off. I just wanted to thank the community for being engaged and helping us come up with some of these questions and continuing your – the engagement. Also I wanted to thank Takeda and Tom and Cheryl and Dave and the entire team that you guys have in place to help put this together and helping keep the community informed. And Danette for helping me figure this thing out as we go.

No, but it's been a really good evening and I think we got a lot of good information and hopefully answered a lot of questions. And with that, again, I just want to say thank you to everybody for taking their time out tonight and joining us. And from there, I will turn it over to Cheryl and Tom.

Cheryl Schwartz:

Yes. Thank you so much, Bob. And huge thanks to Bob and Danette and the HypoPARA Association for, you know, continuing to work so closely with us through what has been a really challenging situation. And I know which has been, you know, further exacerbated by this very unusual world that we're living in right now in the midst of COVID-19. I know that adds a whole new level of stress and concerns for everyone involved with this process. So thank you, first and foremost, and to all of you for joining and for bearing with us through what's been a really challenging situation.

I do want to just kind of call it out, you know, from the Takeda side by reiterating, you know, that we are highly committed, this remains a top priority and we are in regular conversations with FDA on, you know, on a regular basis, as we've been in discussions with them. This week, we expect to hear from them again, in the coming days as well based on some of our communication.

So I just want to, you know, reassure people that even though there is so much else going on in the world right now, that this is a priority for us, and it is a priority for FDA. So it's not no way been falling through the cracks or been kind of replaced by other activities. And we're really very much committed to working on this and bringing the product back to all of you who need this medicine so much and as soon as possible.

So we'll continue to keep you informed as we have more information. And as I mentioned earlier, we do hope to reach out to you and just understand, you know, just how this particular situation is impacting you. And if there's anything else that we can do to support you during this time, so please do keep an eye out for additional communications from us in the coming weeks as well. And I'll turn it to Tom for any other comments on his side as well.

Tom Koutsavlis:

Well, thanks Cheryl. A very big thank you to all of you and yourselves, patients as well as your families and for all the support that you provided. Thank you for keeping close in touch with us as well and being able to come on our call and spending time with us in our conversation.

Just to reiterate Cheryl's point, I mean, certainly I know COVID has been challenging for all of us. Nothing has changed in terms of our commitment and the work that we're doing. The communications we have with the FDA are still moving along just as they were prior to coronavirus. We definitely are still 100 percent committed to moving through this and the same speed or faster if we can.

So it's been really a pleasure being able to connect with all of you and really being able to get some more information from you. And I encourage you to continue to reach out to us. And then of course, please again, I would like to reiterate that if there's any change in your condition, especially as we go through these challenging times in the world right now, please circle back to your endocrinologist so that they can also circle back to the Takeda SUP program.

And I think on that note, I just wish everyone a good evening and maybe pass it back to Bob.

Bob Sanders:

OK, yes. It doesn't look like we have any other comments or questions. So again, I want to thank everybody for calling in tonight. And continue to stay engaged and we'll look for look forward to doing something altogether for World HypoPARA Day coming up. Mark that on your calendar, so excited for that.

And again, good night, everybody and thank you.

Cheryl Schwartz:

Thank you.

Dave Murdoch:

Thank you.

Tom Koutsavlis:

Thank you.

Thank you for listening to the recording of this call.  Please stay on to hear Important Safety Information about NATPARA.

Important Safety Information

What is the most important information I should know about NATPARA?

Warning: Possible bone cancer (osteosarcoma).

  • During animal drug testing, NATPARA caused some rats to develop a bone cancer called osteosarcoma. It is not known if people who take NATPARA will have a higher chance of getting osteosarcoma. Tell your doctor right away if you have pain in any areas of your body that does not go away, or any new or unusual lumps or swelling under your skin that is tender to touch.
  • NATPARA is only available through the NATPARA Risk Evaluation and Mitigation Strategy (REMS) Program. The purpose of the NATPARA REMS program is to inform patients about the potential risk of osteosarcoma associated with the use of NATPARA. For more information about this REMS program, call 1-855-NATPARA (628-7272) or go to www.NATPARAREMS.com.

NATPARA may cause other serious side effects, including:

High blood calcium (hypercalcemia)

  • NATPARA can cause some people to have a higher blood calcium level than normal.
    • Your doctor should check your blood calcium before you start and during your treatment with NATPARA.
    • Tell your doctor if you have nausea, vomiting, constipation, low energy, or muscle weakness. These may be signs that you have too much calcium in your blood.

Low blood calcium (hypocalcemia)

  • People who stop using or miss a dose of NATPARA may have an increased risk of severe low blood calcium levels.
  • Tell your doctor if you have tingling of your lips, tongue, fingers and feet, twitching of face muscles, cramping of feet and hands, seizures, depression, or have problems thinking or remembering.

Tell your doctor right away if you have any of these signs and symptoms of high or low blood calcium levels.

Who should not use NATPARA?

Do not use NATPARA if you are allergic to parathyroid hormone or any of the ingredients in NATPARA.

What should I tell my healthcare provider before using NATPARA?

Before you start using NATPARA, tell your doctor about all of your medical conditions. Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of NATPARA?

NATPARA may cause serious side effects like allergic (hypersensitivity) reaction, including anaphylaxis. Tell your healthcare provider or get emergency medical help right away if you have any of the following symptoms of an allergic reaction:

  • swelling of your face, lips, mouth, or tongue
  • breathing problems
  • fainting, dizziness, feeling lightheaded (low blood pressure)
  • fast heartbeat
  • itching
  • rash
  • hives

The most common side effects of NATPARA include: tingling, tickling, or burning feeling of the skin, low or high blood calcium, headache, nausea, reduced sense of touch or sensation, diarrhea, vomiting, pain in joints, too much calcium in urine, and pain in limbs.

These are not all the possible side effects of NATPARA. For more information, talk with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see Prescribing Information, Medication Guide, and Instructions for Use available on NATPARA.COM.

Thank you!

Latest Updates

Sorted by Most Recent

Letter to Patients

September 15, 2021
FDA Regulatory Filing Update - Prior Approval Supplement
Download PDF version of Letter to Patients

Letter to Patients

March 31, 2021
Recall Update
Download PDF version of Letter to Patients

Letter to Patients

February 5, 2021
Special Use Program Supply Update 5 - Resolved
Download PDF version of Letter to Patients

Letter to Patients

December 22, 2020
Special Use Program Supply Update 4
Download PDF version of Letter to Patients

Letter to Patients

December 16, 2020
Special Use Program Supply Update 3
Download PDF version of Letter to Patients

Letter to Patients

November 16, 2020
Special Use Program Supply Update 2
Download PDF version of Letter to Patients

Letter to Patients

October 20, 2020
Special Use Program Supply Update
Download PDF version of Letter to Patients

Letter to Patients

January 21, 2020
Update on timeframe for NATPARA® (parathyroid hormone) availability
Download PDF version of Letter to Patients

Updated FDA Classification

October 9, 2019
FDA classifies NATPARA recall as a Class 1 recall requiring return of all unused product
Download PDF version of Updated FDA Classification

Letter to Patients

September 25th, 2019
Additional details about the NATPARA Special Use Program
Download PDF version of Letter to Patients

Special Use Program

September 24th, 2019
Takeda announces NATPARA Special Use Program in the US
Download PDF version of Special Use Program

Recall Press Release

September 13th, 2019
Message about the US NATPARA Recall
Download PDF version of Recall Press Release

Recall Information

September 5th, 2019
Takeda issues US recall of NATPARA due to the potential for rubber particulate
Download PDF version of Recall Information

NATPARA Special Use Program

If you are a patient previously prescribed NATPARA who is facing life-threatening complications as a result of discontinuation of NATPARA, despite efforts to actively manage your condition, your healthcare provider will be able to request NATPARA for you. The Medical Review committee will then evaluate eligibility on a case-by-case basis.

Get Details

OnePath® is Here to Help

Our experienced Patient Support Managers can work with you one-on-one to answer your questions at 1-866-888-0660, Monday through Friday 8:30 a.m. to 8:00 p.m. ET.

View Program
+
-

Warning: Possible bone cancer (osteosarcoma).

  • During animal drug testing, NATPARA caused some rats to develop a bone cancer called osteosarcoma.
+
-

Important Safety Information

What is the most important information I should know about NATPARA?

Warning: Possible bone cancer (osteosarcoma).

  • During animal drug testing, NATPARA caused some rats to develop a bone cancer called osteosarcoma. It is not known if people who take NATPARA will have a higher chance of getting osteosarcoma. Tell your doctor right away if you have pain in any areas of your body that does not go away, or any new or unusual lumps or swelling under your skin that is tender to touch.
  • NATPARA is only available through the NATPARA Risk Evaluation and Mitigation Strategy (REMS) Program. The purpose of the NATPARA REMS program is to inform patients about the potential risk of osteosarcoma associated with the use of NATPARA. For more information about this REMS program, call 1-855-NATPARA (628-7272) or go to www.NATPARAREMS.com.

NATPARA may cause other serious side effects, including:

High blood calcium (hypercalcemia)

  • NATPARA can cause some people to have a higher blood calcium level than normal.
    • Your doctor should check your blood calcium before you start and during your treatment with NATPARA.
    • Tell your doctor if you have nausea, vomiting, constipation, low energy, or muscle weakness. These may be signs that you have too much calcium in your blood.

Low blood calcium (hypocalcemia)

  • People who stop using or miss a dose of NATPARA may have an increased risk of severe low blood calcium levels.
  • Tell your doctor if you have tingling of your lips, tongue, fingers and feet, twitching of face muscles, cramping of feet and hands, seizures, depression, or have problems thinking or remembering.

Tell your doctor right away if you have any of these signs and symptoms of high or low blood calcium levels.

Who should not use NATPARA?

  • Do not use NATPARA if you are allergic to parathyroid hormone or any of the ingredients in NATPARA.

What should I tell my healthcare provider before using NATPARA?

  • Before you start using NATPARA, tell your doctor about all of your medical conditions. Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of NATPARA?

  • NATPARA may cause serious side effects like allergic (hypersensitivity) reaction, including anaphylaxis. Tell your healthcare provider or get emergency medical help right away if you have any of the following symptoms of an allergic reaction:
    • swelling of your face, lips, mouth, or tongue
    • breathing problems
    • fainting, dizziness, feeling lightheaded (low blood pressure)
    • fast heartbeat
    • itching
    • rash
    • hives
  • The most common side effects of NATPARA include: tingling, tickling, or burning feeling of the skin, low or high blood calcium, headache, nausea, reduced sense of touch or sensation, diarrhea, vomiting, pain in joints, too much calcium in urine, and pain in limbs.

These are not all the possible side effects of NATPARA. For more information, talk with your doctor.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see Prescribing Information, Medication Guide, and Instructions for Use.