Amicus Therapeutics, Inc. (FOLD) Earnings Call Transcript & Summary

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Conference Call and Webcast subject to FDA's approval of Pombiliti + Opfolda. [Operator Instructions] As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Vice President of Investor Relations. You may begin, sir.

Good morning. Thank you, operator, and thank you, everyone, for joining our conference call to discuss the U.S. FDA approval of Pombiliti and Opfolda. Today's call will be led by Bradley Campbell, President and Chief Executive Officer; and he'll be joined by Dr. Jeff Castelli, Chief Development Officer; and Mike Keavany, President of U.S. Commercial Business and Head of Global Marketing. Available for Q&A is Simon Harford, Chief Financial Officer; and Sébastien Martel, Chief Business Officer. As referenced on Slide 2, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements, which speak only to the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statements and risk factors section of our annual report on Form 10-K for the year ended December 31, 2022, as well as our quarterly report on Form 10-Q for the quarter ended June 30, 2023, filed with the Securities and Exchange Commission. At this time, it is my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Brad?

Great. Thank you, Andrew, and good morning, everyone. Thank you for joining us today. It is with great pleasure that I announce the FDA approval of Pombiliti and Opfolda. Today has been more than a decade in the making and marks a transformative moment, not just for Amicus, but more importantly for adults in the United States living with late onset Pompe disease who now have a treatment option that's new to them. This milestone highlights the persistence and dedication of the team of passionate entrepreneurs at Amicus, who have exemplified what it takes to execute on a vision and deliver innovative new medicines to people living with rare life-threatening conditions. And let me also just say how thankful all of us are at Amicus to the clinical and medical community around the world and especially to the many patients and families living with Pompe disease, who sacrificed so much to take part in these clinical studies. Without your participation and efforts throughout the years, we would not be where we are today announcing an approval of a new therapy for a disease where there is still so much unmet medical need. The entire Pompe community has served as a constant inspiration to us, and we thank you all for it. Before I turn the call over to Jeff and Mike, I'd like to provide a brief summary of today's announcement starting on Slide 3. As a reminder, late onset Pompe disease is a rare and fatal genetic disease and one of the most prevalent lysosomal storage disorders. It is still significantly under-diagnosed. Within late onset Pompe disease, individuals typically experience impaired motor function and respiratory difficulties as the disease progresses, which tend to become more serious and life-threatening over time. Based on the high unmet medical need in Pompe disease, our researchers set up to design a therapy to address some of the key challenges in the delivery of the therapeutic enzyme recombinant human acid alpha-glucosidase, or rhGAA. What resulted was the development of Pombiliti and Opfolda, the first and only 2-component therapy with a unique mechanism of action for the treatment of adults with Pompe disease who are not improving on their current therapy. Over the past decade, we have compiled a body of clinical evidence for Pombiliti and Opfolda that demonstrate improvement in ERT-experienced adults. We are confident that the consistency and durability of effect in patients across key manifestations of the disease illustrates the potential impact Pombiliti and Opfolda can have for many individuals. We're also very pleased with the outcome of the label. As we've discussed leading into this milestone, we believe the label has everything we need to support a successful launch. And Jeff will provide more detail later in the call. The current global Pompe market, as a reminder, is a little over $1.2 billion, expected to grow to $1.8 billion by 2027. And with multiple products now in the market, patient identification and diagnosis efforts underway, we expect continued robust growth in this market for many years to come. We believe Pombiliti and Opfolda is poised to capture a significant portion of this commercial opportunity as we help many, many patients around the world. We expect to achieve the peak global revenue potential for Pombiliti and Opfolda of around $1 billion by resetting treatment expectations for this devastating disease. Our clearly differentiated data, particularly within the ERT-experienced population, coupled with our highly experienced and comprehensively trained teams, sets us up for success in the launch of this new therapy. Indeed, in our first wave of international launched countries in Germany and the U.K., we are seeing excellent health care professional engagement and higher-than-expected levels of use and rapid conversion of our clinical trial and expanded access patients. And finally, we have a strong and broad patent portfolio with exclusivity out to the end of the 2030s, which provided us with plenty of time to continue delivering Pombiliti and Opfolda to many patients for a long period of time. I'd now like to hand the call over to Jeff to provide an overview of late onset Pompe disease, our unique mechanism of action and our FDA label. Jeff?

Thank you, Brad, and I'd like to reiterate our excitement on this long-awaited approval and our sincere thanks to everyone that has contributed along the journey. So starting on Slide 4, a quick overview of Pompe. Late onset Pompe is a rare, debilitating and life-threatening lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase, or GAA. And reduced levels of GAA lead to the accumulation of the substrate glycogen in muscles and other tissues and predominant manifestations we see are skeletal muscle weakness and progressive respiratory involvement often leading to decline in organ failure and premature death. Pompe can be diagnosed at different stages of life, from childhood to adulthood, and affects around 5,000 to 10,000 individuals globally. Although recent newborn and high-risk screening studies suggest there could be actually closer to 40,000 to 50,000 people living with Pompe globally. We see multiple publications in natural history studies showing the initial benefits of treatment in patients on alglucosidase alfa, which are often then followed by continued long-term decline on key measures of disease for many individuals. We continue to recognize that Pompe continues to pose a range of health challenges for people affected by the disease and having therapeutic choices is crucial. Moving to Slide 5. This provides an overview of Pombiliti and Opfolda, our innovative 2-component approach to treating late onset Pompe disease. This treatment is the first and only 2-component therapy, consisting of Pombiliti, a naturally expressed bis-M6P-enriched recombinant human GAA enzyme that facilitates high affinity uptake while retaining its capacity for processing into the most active form of the GAA enzyme in the lysosome. It's combined with an oral enzyme stabilizer Opfolda that is designed to reduce the loss of enzyme activity in the blood during the infusion. Used in combination, this unique 2-component therapy is designed to stabilize the enzyme in the blood, increase uptake in the muscle and maximize activity in the lysosome. Moving on to Slide 6. As a result of our dialogue with the FDA, we're very pleased with the U.S. label outcome as it reflects the value that Pombiliti and Opfolda provides to patients. For us, it was important that the label covered our differentiated data set in ERT-experienced patients and the improvement our therapy could potentially bring to those patients. Challenging therapeutic expectations for those patients that have been on any other ERT. We ultimately see this becoming the therapy of choice for adults who are not improving on their current therapy. Pombiliti and Opfolda is indicated for adults with late onset Pompe disease weighing more than 40 kilograms and who are not improving on their current enzyme replacement therapy. This indication is approved based on positive data from the Phase III pivotal PROPEL study, the only controlled trial in LOPD to ever study the high unmet need ERT-experienced population. In the ERT-experienced subgroup, the population covered by approved indication, study participants receiving Pombiliti and Opfolda demonstrated mean improvement in measures of 6-minute walk distance and stability in forced vital capacity versus the comparator. This data set is crucial to highlight as it reflects the reality of many people living with and being treated for LOPD. We look forward to resetting therapeutic expectations for both physicians and patients by showing them that with Pombiliti and Opfolda improvement in ERT-experienced patients is possible. On safety, Pombiliti and Opfolda has a similar AE profile to the other approved ERTs. The most commonly reported adverse events for Pombiliti and Opfolda of more than 5% of patients were headache, diarrhea, fatigue, nausea, abdominal pain and pyrexia. Overall, we are very pleased with the label as we're the only approved therapy that showed improvements in this ERT-experienced population that control clinical trial. Notably, this indication is agnostic to whichever enzyme replacement therapy patients are receiving. So all adult patients on ERT today in the U.S., whether receiving alglucosidase alfa or avalglucosidase alfa and who were not improving are eligible for Pombiliti and Opfolda. And with that, let me now hand the call over to Mike to discuss the Pombiliti and Opfolda launch in the U.S. Mike?

Thank you, Jeff, and good morning, everyone. I'd like to start by also thanking my Amicus colleagues, our external partners, our clinical trial patients and all members of the Pompe community. Each has been instrumental in the research and development of their therapy. This has been a long awaited day for adults living with late onset Pompe disease. And it is your passion and dedication that got us to where we are today with the approval of Pombiliti and Opfolda in the United States. We're on Slide 7, and I'd like to take a few moments to discuss the U.S. Pompe market in where we see the long-term market opportunity for Pombiliti and Opfolda. For more than 16 years, the market had only 1 enzyme replacement therapy approved. 2 years ago, a second ERT product from the same company came to the market. Within the U.S., there is a very sizable market opportunity of over $500 million, and global, it stands at $1.2 billion. As you can see on the map, with 44 states doing newborn screening and multiple products, we see continued growth in this market, tracking towards $0.75 billion in the U.S. and $1.8 billion globally by 2027. Clearly, there are a significant number of patients who could benefit from Pombiliti and Opfolda. And between our data and the label we've been given today, we see this becoming the therapy of choice for experienced patients not improving on other enzyme replacement therapies, and we are very confident that we can address the needs of the market with Pombiliti and Opfolda. If we move to Slide 8. Amicus is operating from a position of strength as we look to leverage our current U.S. infrastructure from the commercial launch of Galafold to drive another successful launch. We have a team of passionate individuals across all areas required to launch excellence, including regulatory, supply chain, medical, commercial, reimbursement and access. Our commercial team is fully hired, trained and in place across the U.S. to support this launch. Our team has established relationships with key physicians and treatment centers across the country, and they're excited to introduce our new 2 component therapy. Our launch will focus on 4 main areas. First, as I just highlighted, is our highly experienced team, and they will be critical to driving the launch success of Pombiliti and Opfolda. The second key component is education. We intend to educate health care professionals about this product in a variety of methods, including medical congresses, publications and promotional efforts. Data dissemination is one of the most important launch components and where we can really make an impact by leveraging the strength of our data. In the adult ERT-experienced population, that data demonstrated improvement in both 6-minute walk and forced vital capacity in patients who switched from their existing ERT therapy to Pombiliti and Opfolda. Our sales and marketing teams are fully equipped to start disseminating our promotional material, generating awareness with physicians and educating on the benefits of switching to Pombiliti and Opfolda when patients aren't improving. The third area of focus, as we've always had at Amicus, is ensuring broad patient access. We have a highly skilled market access team with extensive experience in reimbursement and access who have been actively engaging with payers to demonstrate the value of Pombiliti and Opfolda. We've also expanded Amicus assist, our robust patient support program in the U.S., and they are ready to aid patients on their journey with this product. Through Amicus Assist, we provide the key elements to fully support rare disease patients, including help with benefits investigations and insurance processes, co-pay assistance for eligible patients, and we have patient education liaison available to provide disease education and product support. We have approximately 40 eligible clinical trial patients who will be transitioning to commercial product. That transition will begin today, and we expect all of them to be commercial patients by the end of the year. Also beginning today, our patient hub is prepared to start receiving patient referral forms from any physician in the U.S., and our goal is to have products labeled and available for sale within 2 weeks. And the fourth and final critical component of any launch is always execution. We've clearly showed that the Galafold launch and subsequent success from the last 5 years that we can execute on our plans. With our second commercial launch, we've already laid the groundwork and identified key opinion leaders and treatment centers throughout the country. And in the coming days, they will begin to hear about an exciting new option, Pombiliti and Opfolda. We're excited to see the value of Pombiliti -- that the value of Pombiliti and Opfolda can bring to eligible patient population. And we certainly look forward to updating you on our launch success. With that, I'd like to thank you and hand the call back to Brad for some additional thoughts on the launch and closing remarks.

Great. Thanks, Mike. Turning to Slide 9. I'd just like to quickly discuss and remind everybody of our pricing and access strategy from Pombiliti and Opfolda. This is really rooted in our founding beliefs. We made a promise very early on that our medicines must be fairly priced and broadly accessible. At Amicus, we recognize that our efforts in advancing Pombiliti and Opfolda towards the market will only be successful if patients who may benefit from this 2-component therapy have access to it. Therefore, we very early on established a pricing strategy that supports a streamlined and efficient pathway for patient access. We used this strategy successfully in Galafold and now we'll apply it to Pombiliti and Opfolda as well. First, we believe that we should price our medicine at no additional cost to payers compared to similar competitor products. And second, and this is very important, we will never raise the annual price of our products more than CPI, the consumer price index. This is something we've carried with us through the Galafold launch and has proven to be a sound business strategy as well. Effectively keeping price increases at CPI encourages access to the drug, which we believe means more patients can get access to Pombiliti and Opfolda, they can move more quickly through the reimbursement process. And ultimately, this means that more patients can come on to the drug at a quicker rate. Third, we continue to honor our pledge to reinvest a portion of the revenue from any Amicus marketed drug back into research and development for that specific disease until there is a cure. And fourth, we will continue to provide world-class patient support services, including needs-based financial support globally to ensure all who need access to our therapies have them. With these principles in mind and taking to account the weight-based dosing, we've determined that the U.S. listed prices Pombiliti and Opfolda to be around $650,000 for a patient weighing approximately 70 kilograms, which is about the average adult weight. By pricing at a modest discount to currently available ERTs for adult patients, we believe we deliver the value of this 2-component therapy to patients and to payers in the health care community. And as just mentioned, we do so while also limiting the annual price increases of Pombiliti and Opfolda to consumer inflation. In closing, we are highly confident in our team responsible for launching Pombiliti and Opfolda, who have already exemplified what it takes to push important medicines through the clinical trial process over the regulatory finish line and into the hands of those living with rare, life-threatening conditions. Today's U.S. approval, coupled with the prior European Commission in U.K., MHRA approvals of Pombiliti and Opfolda, respectively, marks an important milestone for Amicus as this 2-component therapy is now fully approved for adults with LOPD in the 3 largest Pompe markets around the world. We are incredibly proud of our team's progress throughout these initial stages of launch. And we will look to these 3 key markets to support early launch momentum and set the stage for what we believe will be a very successful commercial product launch of our second approved therapy. We look forward to broadening patient access to Pombiliti and Opfolda into additional markets in the very near future as well. Today's approval is not just a milestone, it's the beginning of a new chapter, one way where we will continue to push the boundaries of science and innovation and work tirelessly to transform the landscape of rare diseases. With that, we can now turn the call over to Q&A.

[Operator Instructions] Our first question will be coming from Ritu Baral of TD Cowen.

My -- I have two questions, and they relate to the Pombiliti label specifically, including the -- I guess, the indication in the data charts. One, in your conversations with clinicians and payers, how do they define the word improving? Does that need to encompass both FVC and 6-minute walk? And is it improving versus expectations? Or is it squishy? And then as we look at the switch data in the label, I believe it's Figure 2 and Figure 3, there's a footnote about the alglucosidase alfa product not used in this clinical -- was not used in this clinical trial or U.S. approved one. Can you comment on where that comes from? What drives that note? And does it matter?

Yes. Thanks, Ritu. Why don't we take your first question first, and then we'll come back to the second question around the footnote, which is just a very technical, I would say, FDA commentary. So on the first question around improving, I'll let Jeff talk clinically in a minute, but I think the important thing there is that this is a language that we very carefully negotiated with the FDA. It's something we wanted to see in the label. And as Jeff highlighted on the call, it speaks to what we think is a very differentiated opportunity and a chance for us to really reset the standard for expectations for treatment. But Jeff, maybe talk a little bit about the -- what, from a clinical perspective, improving means and the flexibility really that provides for the physicians?

Thanks, brad, and thanks, Ritu, for the questions. So I think most importantly, as Brad said, we intentionally wanted to have this language around not improving because it really leaves the latitude up to the physician to make that call. It's not on any specific parameter. And as we know from all the publications, from all the discussions we've had, the vast majority of patients after the first year or 2 that are on ERT tend to either be stable or declining. So there's a real opportunity there to engage and ask if those patients are actually having improvement on different parameters. When you look today, the average patient in the U.S. has been on ERT more than 3 years. So certainly, we think the majority of those patients are in a point in their trajectory where they're stabilized or declining. And we feel really good about this label as an opportunity to go in and really have a potential treatment option for the vast majority of patients currently on an ERT. And importantly, as I mentioned during the call, it's whether they're on alglucosidase alfa or avalglucosidase that the label would apply to them.

Yes. Great. Thanks, Jeff. Hopefully, that helps answer your question, Ritu. Again, we think this really is exactly where we want to position the drug, and that means that even a patient who's stable will be eligible potentially to switch, which is great. On your second question, again, this is kind of a technical point, but all of the alglucosidase alfa, so the comparator product used in the clinical trial was sourced from a single global license holder, which, of course, is Sanofi. And all of that product was manufactured at a single site using identical materials and process. However, some of it was that was used in the clinical trial was sourced from batches that were technically released in Europe. And therefore, in the end, the FDA from a technical perspective, said we should refer to it as a non-U.S. approved product. But the reality is this is going to have no impact on how physicians view data, and it's really a reflection of the FDA's view of the technical release criteria. So we don't see this as anything that significant and physicians will certainly understand it for what it is. And so again, we're very pleased with everything in the label and don't see any of these as any kind of challenges.

[Operator Instructions] Our next question will be coming from Anupam Rama of JPM.

Congratulations on the approval. I know it's been a real long process to get here. So that's great. So congrats again. You talked about making this sort of the ERT experience switch agent of choice. So what's really driving that statement? Can you maybe talk about, is it the patient population enrolled in PROPEL relative to Nexviazyme's patient population enrolled? Are there aspects of the label that make you confident in this? Like maybe you could expand on that?

Yes. Thanks, Anupam, and I'll turn it over to Jeff for -- in a minute here. But I think from our perspective, it goes back to what we've talked about all along, which is, number one, we have a very differentiated mechanism. Number two, the trials were designed and enrolled a very different patient set. And then number three, that resulted in what we think is very differentiated data for Pombiliti and Opfolda. So maybe, Jeff, remind us a little bit about that differentiated data set and why we believe this is so important for driving the experienced patient population utilization. And again, just a reminder to everybody out there, the experienced patients represent the vast majority, over 90% of the opportunity for Pombiliti and Opfolda in the United States in the foreseeable future. There's very few new patients that come on therapy every year compared to the number of patients who are already on therapy. And so from our perspective, switching those patients is the most important and largest opportunity for the long time to come. And so this is exactly in line with what we expected and what we think we needed for a successful commercial launch. But Jeff, maybe remind us a little bit about why we see this as differentiated from the mechanism to the population to the data set.

Yes. Thanks, Brad, and thanks for the question, Anupam. So we view this as a very differentiated mechanism. It's a 2-component therapy. We have this small molecule stabilizer during the infusion. We have the better uptake by having the higher bis-M6P. And importantly, that bis-M6P was naturally added to the enzyme so that it has retained processing to the active form. So we think that's a real strong aspect of the product as we go out and launch about talking about the differentiated mechanism for other ERTs. And then for sure, we have a very differentiated data set clinically, especially in terms of the ERT-experienced population, we have the only controlled data set in those patients. 80% of the patients in PROPEL were ERT-experienced. And in those patients, we saw, as Mike mentioned, improvements in both 6-minute walk and FVC. And we've recently shown improvements on a host of other parameters as well and that were studied across things like patient-reported outcomes and muscle strength. So that data set we think is very differentiated. And also, our long-term data, we believe, is something that longer and longer will differentiate in terms of the durability of effect that we're seeing. We've now reported data out to 4 years from our Phase II data showing very durable improvements in the different measures. So we think the differentiated mechanism, differentiated data set will allow us to go out and really engage and appropriately talk about why we think that this is a great treatment option for ERT-experienced patients.

And our next question will be coming from Ellie Merle of UBS.

Congrats on the approval. Maybe just on reimbursement in Europe, I guess, just what's the latest in terms of your expectations in terms of the time frame to reimbursement in the U.K. and Germany. And then maybe just from a market share perspective versus Nexviazyme maybe what are some of the learnings around market share from some of the initial time on the market in regions such as the U.K. and Europe, where both Nexviazyme and ATG were available?

Thank you, Ellie, great to hear from you. And Sébastien, I'll ask you perhaps to comment on the market share questions that were raised. From an overall market access perspective, things are going incredibly well. So remember, in Germany, you have a sort of 6-month, what's globally are known as a free pricing period and then we'll follow that up with our pricing strategy as we've announced, which is parity or modest discount. That was something that I think proceeded incredibly well with Galafold and we expect it to go very well in Germany as well. So far, as we've indicated in previous conversations, the conversion process has been incredibly quick. We've converted all of our expanded access patients and have scheduled now all of our clinical trial patients that have already started to switch some new commercial patients as well. So launch is going great there. I think NICE and the reimbursement process there is probably the best indication of the way that payers are seeing the value of this medicine. So remember that NICE recommended reimbursement for Pombiliti and Opfolda prior to MHRA approval. I think this is one of the fastest ever products going through that process. We know that NICE is notoriously a challenging reimbursement authority from a global perspective and the fact that they recognize the value in their evaluation document and recommendations, I think, is a great example of the strategy we've used here, which again, is to maximize speed to the reimbursement process so that every patient can have access to Galafold who's appropriate as quickly as possible. So that's gone very well. And we now have reimbursement in the U.K. It takes about 30 days from NICE recommendation and approval to funding at the local level. We've had commercial orders placed and we're now starting to infuse patients in the U.K. as well and starting that conversion process. So Europe, I think, has gone incredibly well. Maybe Sébastien just talk quickly about kind of the state of affairs from a market share perspective in the U.S. and the EU and why we think that sets us up for success.

Yes. So the state of the market in Europe just prior to our launch, and I'm going to be referring here to the Q2 sales results that Sanofi released. So the split between Myozyme and Nexviazyme was 77% Myozyme sales and 23% Nexviazyme sales in Europe. In the U.S., this was more like 50-50. You were talking early about U.K. and Germany. So while here, I was expressing a breakdown of sales. I can also say that we've had patients on Pombiliti, Opfolda in those markets in -- even prior to launch through the [indiscernible] program and through clinical trials. So in the U.K. alone, we had somewhere between 40 and 50 patients, again, prior to launch on treatment that accounts for about somewhere between 20% and 25% of the whole U.K. market. And as you remember, we've committed to ensure that there will be conversion of those patients within 90 days post launch. So definitely, definitely before the end of the year. And then in Germany, we had close to 20 patients on drug on atga prior to launch, and those were either through the early access program cup or through a clinical trial. And again, we are making great progress in converting those patients. within a short amount of time.

And our next question will be coming from Joseph Schwartz of Leerink Partners.

Congrats on the approval. I was wondering to what extent is the Nexviazyme launch a good analog for how we should think about the ramp for Pombiliti and Opfolda? Do you have a sense the types of patients that Nexviazyme has been adopted to or by -- to a greater or lesser degree? And is that the same pattern that you expect to see? Any insight on any subsets within the LOPD market that we should be mindful of for your launch?

Yes. Thanks, Jeff, and thanks for the congrats. I think that we should be careful not to comment on Nexviazyme aside from the kind of factual stuff that Sébastien provided. I think what I would point us to as one of what we think could be a very helpful indicator of the traction with Pombiliti and Opfolda is, first of all, I think the early results that I've shared with you on Germany and the U.K. But second of all, I think if you look at the EAMS program, as an example, as a reminder, that's an early access medicine scheme program in the U.K. We've talked about this before. That is a program where prior to approval, the MHRA provides early access to a medicine if it has certain criteria. We're not allowed to promote that product, so it's entirely up to physicians as to whether or not they want to use it. And as Sébastien just highlighted within a year's period of time. And by the way, as a reminder, Nexviazyme was available at the same time through a very similar program. And of course, Lumizyme was on the market already. Within about a year with no promotion whatsoever, we already were up to 15% to 20% market share of treated patients. So if you think about them being able to promote the product, being able to talk to the data that we've already talked about, we think there's a ton of opportunity here to have a fantastic launch. So Hopefully, that gives you some sense of what that thought experiment might look like. In terms of patients that we'll target, I think the answer is we'll target all eligible patients according to the label in each region. And as I mentioned before, we're already seeing uptake in commercial patients in Europe in addition to the conversion, we have that large pool of conversion patients that Mike talked about. And again, on top of that, be looking to bring as many new commercial patients on who are eligible over the course of the year.

And our next question will be coming from Dae Gon Ha of Stifel.

This is Benazir on for Dae Gon. Congratulations on the approval. We just had one question. Can you elaborate on why you've implemented the 40-kilogram weight cutoff? And how you see this impacting the potentially eligible patient population?

Yes. I'll let Jeff talk to that. Go ahead, Jeff.

Yes. No, thanks for the question. So it's really just based on the population studied in the label population of adult LOPD. So we did not see any patients that were adult LOPD that were below 40 kilograms in any of our trials or access. So it's really just based on theoretically, if there happened to be an adult that weighed that amount or below that amount, there might need to be some adjustment to dose to sort of match PK. So we are in our pediatric study in LOPD and IOPD, we are looking at, obviously, kids that weigh lower than 40 kgs, and we're looking at alternative dosing to sort of have a PK matching for the miglustat and for the Pombiliti. But we don't really view that from an adult LOPD population that this is at all going to impact any significant number of patients. We haven't seen any yet of the 200.

And our next question will be coming from Kristen Kluska of Cantor.

Congratulations on the approval. I wanted to ask you what your latest thoughts were on the market and the percent split in terms of those patients that are naive versus those that are ERT-experienced? And wondering if this has also changed since the next Biozyme approval?

Yes. Thanks, Kristen. Sorry, we couldn't get to this news a day earlier when we were at your conference, but glad we were on track for the coming days guidance we gave yesterday. So Sébastien, maybe just talk a little bit about the overall patient population in terms of number of patients treated, what a large opportunity that is. And then in the U.S., where this is for experienced patients what we estimate is the number of new patients that come on therapy a year with a small segment of the population that is?

Absolutely. Thanks, Brad. So Kristen, on a global basis, we estimate that the total number of treated patients for Pompe disease is north of 3,500, probably close to 4,000 as we speak. If you -- so that's the pool of potential switch patients, if you want. If you think of newly diagnosed naive patients, depending on the year, you're somewhere between 100 to 200 on a global basis again. So I think you can definitely understand that the commercial opportunity really lies in the switch population. And that's why we're very pleased with the label in the U.S.

Our next question will be coming from Jeff Hung of Morgan Stanley.

Congratulations on the approval. You previously indicated that initial supply for the U.S. launch is in a free trade zone. So how far does that get you into the launch? And how are you thinking about U.S. inventory and distribution?

Yes. Maybe, Mike, I'll let you talk about kind of the supply chain in the U.S. and how we think about that. I would just say on a global basis, we have about 2 years of go-forward inventory. And as you said, we -- in anticipation of launch to make sure that we could go as quickly as possible that 2-week period that Mike talked about, we already brought some supply over to the U.S. in the free trade zone that we can now quickly label and get into the supply chain. But Mike, maybe give a little bit more detail there and share some of how we think about that within the United States.

Yes. Thanks, Brad. We -- as Brad said, we have product in the free trade zone in the U.S. right now that because we have to put the label on it will be about 2 weeks and that product will be salable. We're following that up with an additional supply that we'll be getting in the next, say, 4 weeks post that. Between those 2 supplies, it's well over a year's worth of inventory, and then we'll be supplementing that as we go on. So we don't believe -- we stocked up on inventory early. You never know what happens in a launch, and you always want to be prepared. So we're prepared for over a year of supply before we hit the first 6 weeks of launch. So I think we're in really good shape with supply. We've got a distribution network set up. And we've got the -- all of the points of contact covered hospitals, infusion suites and specialty pharmacies are all set up to take product as soon as we have patients moving forward.

Our next question will be coming from Salveen Richter of Goldman Sachs.

This is Elizabeth on the line for Salveen. On the global peak revenue potential of approximately $1 billion, help us understand kind of what's baked into that assumption in terms of the market share and penetration? And then our second question is we noticed that the OLE data isn't included on the label? And are you considering taking action to include that? And kind of what would the time line be there to add it to the label?

Yes. Great questions. Thank you. So on the first one, in terms of our sort of overall expectations, of course, this is our projection. But Today, the market is a little over $1.2 billion by the end of the decade. We think -- and I think most people expect it will pass well over $2 billion. And as we think about the product profile and the differentiated therapy here, we really believe this can be greater than 50% market share, and therefore, you get to that over $1 billion opportunity. And then on the second part of your question, Jeff, maybe you could talk about the point around the long-term extension data and our plans there.

Yes, thanks. We had not submitted that full OLE data set yet as part of the submission. But we certainly will look as we go along here, I think several label updates likely coming next for us. One would be getting labeling in the 12- to 17-year-old LOPD group, which I think is data that we've compiled and will be discussing with regulators soon. And also, as you mentioned, things like the OLE data and other long-term data that we could submit that potentially could add long term to the indication. With our market research, we don't think that, that's a real driver here. The data is already available for physicians in multiple presentations and manuscripts. And that's our focus right now. But I think updating the label when appropriate is certainly something that we'll love to store.

And our follow-up question is coming from Ritu Baral of TD Cowen.

Just wanted to ask what your planned activities are for World Muscle coming up next week now that you are approved heading into that? And also will Amicus Assist be used sort of as a commercial strategy like a differentiating tool either to make physicians life easier with reimbursement or assist with patient services that maybe the Sanofi hub is not providing?

Yes. I'll let Mike talk to both of those activities, World Muscle as well as the World Amicus Assist. I will say as it relates to Amicus Assist, this was something that when we were building the strategy for Galafold. We debated a lot of companies will use an external hub when they think about offering these services in the United States. And we really felt like it was important to have an Amicus employee, who is on the line with physicians and patients, helping them through that reimbursement process in particular. And we think it has been a real advantage for us and a real important tool for us to support the Fabry community. And likewise, we expect the same for the Pompe community, but Mike, maybe provide a little bit of an overview of the World Muscle plans and then also the services that we provide through Amicus Assist that we think are so important for the community.

Well, for World Muscle, and I'll let Jeff chip in as well here. We were sort of going down parallel past depending on whether we got approval or not. And based on the approval today, we will have a full commercial presence at World Muscle Society and our symposia that was planned will be a promotional symposia as well. Jeff, I don't know if you want to make any additional comments there.

No, Mike, great point. I think we also have a number of abstracts, presentations, oral presentations on continued data from our various trials as well. But we were sort of waiting on this pivotal decision here to decide if those symposia were going to be commercially oriented or not, but we're real excited now to be in Charleston next week.

Yes. We clearly didn't want to miss this opportunity and now we get a chance to take advantage of it. In terms of Amicus Assist, I think a lot of rare disease companies offer very good services to patients like Amicus Assist. We -- as Brad said, we decided to build our own hub. Our original 3 case managers for Galafold are still with us. And we've added 2 more case managers for the Pombiliti, Opfolda launch. We've added 4 patient education liaisons who will help patients sort of with patient education and product support. And again, we have seen a tremendous success with Amicus Assist in the Fabry community. And I believe we'll see the same thing in the Pompe community. We have dedicated people that will be helping patients and helping give physician offices some information that helps them move forward for approval. All of that usually comes from the physician's office, but Amicus Assist helps them with getting the right information that they need. So it's clearly helped with Galafold in terms of getting patients on therapy quicker, and we believe the same thing will be happening in Pompe.

There are no more questions in the queue. I would like to turn it over to management for closing remarks.

Yes. Thanks very much for all the questions. Again, really excited today to be able to hit this next milestone. I think a really important event both for Amicus, but more importantly, for the Pompe community. I appreciate all your questions, and hope everybody has a great day.

This concludes today's conference call. Thank you all for joining. You may now disconnect, and everyone, enjoy the rest of your day.