Earnings Call Transcript
AMICUS THERAPEUTICS, INC. (FOLD)
Earnings Call Transcript - FOLD Q1 2024
Operator, Operator
Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics First Quarter 2024 Financial Results Conference Call and Webcast. As a reminder, the 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.
Andrew Faughnan, Vice President of Investor Relations
Thank you, everyone. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' First Quarter 2024 Financial Results and Corporate Highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer; Sebastien Martel, Chief Business Officer; Dr. Jeff Castelli, Chief Development Officer; and Simon Harford, Chief Financial Officer. Joining for Q&A is Dr. Mitchell Goldman, Chief Medical Officer; and Ellen Rosenberg, Chief Legal 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 representations 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, 2023, and the quarterly report on Form 10-Q for the quarter ended March 31, 2024, to be filed with the Securities and Exchange Commission today. At this time, it is my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley?
Bradley Campbell, CEO
Great. Thank you, Andrew, and welcome, everyone, to our first quarter 2024 conference call. I'm pleased to report a strong start to the year across our global business. We continue to build on the momentum of significant top-line revenue growth on our way to our first full year of non-GAAP profitability while advancing our mission of bringing hope to patients and families affected by rare diseases. As we did in this morning's press release, let me just highlight several key points before I turn it over to the team to go into more detail. First, we continued our excellent commercial execution and delivered strong total revenue of $110 million, representing 28% growth year-over-year. Galafold continues its strong performance, and we remain very pleased with the continued commercial uptake globally. For the first quarter, Galafold revenue was $99 million globally, representing 16% growth year-over-year on a constant currency basis, coming in at the high end of our initial guidance for the year. For Galafold, we continue to observe strong trends across our key performance indicators in all key geographies in the first quarter, including continued demand through new patient starts from both the switch and naïve populations in all of our leading markets and sustained patient compliance and adherence rates of over 90%. All of this is against the backdrop of significant growth in the diagnosed and treated patients that we've highlighted previously. We see Galafold serving as the foundation of our business for the next decade and more. Based on Galafold's strong performance in the first year, we are pleased to be raising our full-year 2024 revenue growth guidance from 11% to 16% to now 13% to 17%. Second, let me highlight the strong global commercial launch of Pombiliti and Opfolda, our novel two-component therapy for late onset Pompe disease. Now launched in Germany, Austria, the U.K., the U.S., and also now in Spain, as we like to call it, Pombiliti, is a huge driver of growth for us this year, and our progress against our key performance indicators continues to demonstrate the strength of this launch. Our #1 focus for this year is to maximize the number of patients on therapy by year-end. It's great to report that the rate of new commercial patients coming on to Pombiliti in 2024 is double that of last year. In fact, for the month of April, we saw the largest number of new commercial patients start on Pombiliti and Opfolda since our launch. As of the end of last month, we had 155 patients on treatment or scheduled for treatment. We're incredibly pleased with the patient and physician demand for this new therapy globally and continue to hear inspiring anecdotes from treaters around the world on how their patients are responding to Pombiliti and Opfolda, which will help build momentum throughout the year and beyond. Sebastien will provide more details in a moment, but the switch dynamics in the U.S., Europe, and U.K. also continue to look strong, and we're seeing great progress on the reimbursement front globally, including moving patients more quickly through the insurance process in the U.S. as we anticipated we would. We've also just launched in Spain and are off to a great start there. Throughout the remainder of 2024, we'll focus on increasing patient access as we gain reimbursement and then launch in additional countries throughout Europe. Before I turn it over to the team, let me also comment on the new guidance we're providing. As we set upon approval and coming into this year, we needed about 6 to 12 months of trends before we had enough information to provide revenue guidance. Now with more than 6 months since launch in the 3 largest Pompe markets and the great trends we're seeing, we feel we now have enough information to accurately forecast our performance for the year. You'll see in the press release and in the slides, for the first time, we've announced that we expect to deliver $62 million to $67 million in global sales, which will again be a significant contributor to our growth and set us on a great course to achieve our ambition for Pombiliti and Opfolda to become the new standard of care in treating this devastating disease. We are incredibly pleased to be providing a real choice for patients and physicians and challenging therapeutic expectations for people living with Pompe disease. Finally, as we continue our excellent commercial execution across both therapies and prudently manage our resources, we are confident we are well on track to achieve full-year non-GAAP profitability in 2024. Ahead on Slide 4, as we laid out at the beginning of the year, we continue to focus on delivering against our 4 key strategic priorities, including sustaining double-digit Galafold revenue growth. On the back of strong sales, we've raised our expected annual growth rate of Galafold to 13% to 17% at constant exchange rates. Successfully executing on multiple commercial launches of Pombiliti and Opfolda, advancing our ongoing studies to support our medical and scientific leadership in Fabry and Pompe disease, and maintaining our strong financial position as we carefully manage our expenses and investments to achieve non-GAAP profitability for the full year. With that overview, let me now hand the call over to Sebastien to further highlight our commercial performance. Sebastien?
Sebastien Martel, Chief Business Officer
Thank you, Bradley, and good morning to everyone on the call. I'll start by providing you with more details on our Galafold performance so far this year. As you can see on Slide 6, for the first quarter 2024, Galafold reported revenue reached $99.4 million, primarily driven by strong patient demand, particularly from our leading markets. We ended the first quarter with more than 60% of the global market share of treated amenable patients, but we know there's still many more potential patients for our therapy. As Bradley highlighted, given Galafold's strong performance in the first quarter, we're increasing our full-year 2024 revenue growth guidance from 11% to 16% to now 13% to 17% growth. Turning on to Slide 7. Our results in the first quarter highlight the strength of our global commercial efforts. The demand for Galafold globally continues to be strong with patients added in all major markets, delivering operational growth of 16% over the same period in 2023. We're very pleased to see that Galafold grew at the high end of our initial guidance range in Q1 2024. Galafold continues to be the fastest-growing treatment for Fabry disease globally. Our leading markets such as the U.S., the U.K., top EU countries, and Japan remain the biggest drivers of patient demand and give great confidence in the growth this product has over the long term. With the global mix being about 42% switch and 58% naïve now, we're seeing stronger uptake in the naïve population. We continue to achieve high market shares in countries where we've been approved the longest. There's still plenty of opportunity to continue to treat patients over to Galafold and to keep growing the market as we penetrate the diagnosed untreated and newly diagnosed segments. All of this is underpinned by sustained compliance and adherence rates that continue to exceed 90%, reiterating our belief that those who go on Galafold predominantly stay on Galafold. As mentioned on past calls, due to a variety of factors, including uneven ordering patterns and FX fluctuations, the rate of growth within the year is typically nonlinear. We expect that to continue in 2024. So within the table on the right-hand side of the slide, we've provided a 5-year historical snapshot of the percentage of Galafold sales that occur each quarter during a given year. The average of quarterly sales distribution over the last 5 years corresponds precisely to what we achieved for the full year 2023. We would expect a similar trend to occur this year and, as an example, we expect Q2 sales in the current year to be around 24% of full-year sales. On Slide 8, we know that there is significant patient demand for Galafold and that the segments of the global Fabry market made of patients with amenable mutations have the potential to reach up to $1 billion in annual revenue by the end of the decade. We anticipate sustained growth in 2024 and beyond to be driven by several key co-drivers. First, the Fabry market is growing robustly and with a significant portion of growth coming from finding new patients and reaching the diagnosed and treated population. As we've mentioned previously, there were more than 2,500 individuals on Galafold at the end of 2023 and about 1,400 of those individuals were naïve to any treatment before Galafold. Second, we have seen many new patients going through treatment through newborn and family screening, and we continue to increase patient identification capabilities through ongoing medical education and support of noble screening initiatives, including the use of artificial intelligence through our partnership with OM1. Fabry is one of the most underdiagnosed rare diseases, so the more patients that can be identified, the more patients can be eligible for Galafold. We're also seeing many diagnosed untreated patients transition to treatment as the need for earlier treatments, especially in females, becomes better appreciated. The other piece is continuing to drive Galafold's market share of treated amenable patients through continued commercial execution. As noted, Galafold currently has more than 60% of the global amenable market, but we're seeing in our most mature markets that we can reach up to about 85% to 90% of market share. We know that there's the potential to reach those levels in the global market share as well. Again, all of these efforts are supported by solid compliance and endurance rates through physician and patient education and support programs. Additionally, we'll continue to make progress on expanding Galafold into new markets and extending the labels. There are still some markets in LatAm, the Middle East, and the Asia-Pacific region where Galafold is either newly reimbursed or we expect reimbursement. It's also important to note that we have exclusivity in the U.S. and Europe, in addition to our now 58 Orange Book-listed patents, 42 of which provide protection into 2038 and beyond, including 12 Composition of Matter patents. This provides us the opportunity to provide access to Galafold globally for a long time to come. We intend to continue to protect and enforce our broad intellectual property rights. Looking ahead, we expect steady double-digit growth for Galafold throughout 2024, and we remain confident that with our strong IP protection, Galafold is on a long runway well into the next decade. Turning now to Pompe disease on Slide 10. We outlined our global launch progress with Pombiliti and Opfolda. For the first quarter 2024, Pombiliti and Opfolda reported revenue reached $11 million. This represents an increase of 30% compared to the fourth quarter of 2023 and provides a strong foundation for 2024. In the first quarter, we actually achieved the same level of sales as we did in the last 6 months of 2023. In the U.S., we continue to see a majority of patients switching from Nexviazyme about 73%, and the remaining 27% from Lumizyme. This means we're switching patients proportionately from both products. We're also seeing a broadening and deepening of prescriptions with more sites coming online and multiple new prescriptions. Outside of the U.S., we're seeing patients from all 3 segments, some switching from Myozyme and from Nexviazyme at a proportional rate to the respective market shares and some from the naïve population. That's exactly what we want to see at this stage. A few updates on the launch in Europe. We're happy to announce that Pombiliti and Opfolda are now reimbursed and launched in Spain, occurring ahead of our internal expectations. We're very pleased with the fast reimbursement process here and increased access to this therapy throughout the European Union. In Germany, we're delighted with ongoing success in one of our largest European markets. Recently, the Federal Joint Committee, the G-BA, designated Pombiliti and Opfolda with minor additional benefits versus alglucosidase alfa or Myozyme, based on an extremely stringent review of the data in the PROPEL trial. This is a great achievement as Pombiliti and Opfolda are the only Pompe treatment to receive this designation and reflects the value HTAs are seeing in this therapy. This also reinforces our belief that this therapy has the potential to reset treatment expectations and that the improvement is possible. Finally, we remain very pleased with the uptake of Pombiliti and Opfolda in the U.K., where we are heading towards capturing the majority of market share. Currently, we're close to passing 1/3 of market share within the U.K. with the benefit of Pombiliti and Opfolda, which has actually been available for now 2 years in the U.K., and we believe that the current market share is indicative of how this product can perform over a 3-year period and is a great example of Pombiliti and Opfolda's potential demand and uptake. Given increased visibility on patient trends and pricing and reimbursement, we're now able to provide full-year revenue guidance for Pombiliti and Opfolda. For the year 2024, we anticipate revenue of $62 million to $67 million for Pombiliti and Opfolda. To put this in perspective, this is roughly 75% more sales than the first full year of sales from Galafold, which was a great launch by all accounts. Moving on to Slide 11. As Bradley mentioned earlier, there are about 155 patients on treatment or scheduled for treatment as of late April. That's about 130 currently on therapy and about 20 who are scheduled. We continue to be very pleased with the ongoing demand for this therapy as the rate of new commercial patients coming on to Pombiliti and Opfolda continues to increase across all 3 markets. Of note, Q1 was the best quarter yet for new commercial patient starts, doubling the rate of new patient starts from 2023. In particular, April was the strongest month to date for new patient starts and new commercial patients. This is important as it further supports our thesis that launch momentum will continue to build throughout the year as we move more quickly through the prescription to treatment process in the U.S. and as we launch in new countries in Europe. Our launch has leveraged our highly experienced cross-functional team, and we've had great outreach with key opinion leaders. We're seeing an increase in the depth and breadth of prescribers across all 3 markets and in particular, a growing number of prescribers who are not part of our clinical studies or expanded access programs. All core treating centers have been engaged, and we've had very positive feedback from HCPs and other stakeholders regarding our business approach, our support, and patient focus. Finally, we find an important metric to track is our progress with access and reimbursement. We have a highly experienced team who are engaging in positive conversations with payers to demonstrate the value of Pombiliti and Opfolda. In the U.S., with the start of the new year, many large payers have already put Pombiliti and Opfolda on their respective formularies, and we've also seen strong acceptance by Medicare and Medicaid. The overall time core from prescription to infusion is now down to around 70 days. The last few commercial patients have actually come in even quicker. We believe that this will continue to improve. Today, we launched in Germany, Austria, the U.K., the U.S., and now Spain. But we remain in active pricing and reimbursement discussions with additional major markets as we focus on securing broad patient access throughout the European Union. More than 10 reimbursement dossiers have been submitted. Overall, we're starting off the year strong, and we're very pleased with the building momentum on patient demand. Throughout 2024, our focus will be on maximizing the number of patients on therapy by year-end.
Jeffrey Castelli, Chief Development Officer
Thank you, Sebastien and good morning, everyone. Moving on to Slide 12. We remind everyone that we continue to build the body of evidence for Pombiliti and Opfolda through our ongoing clinical studies and our registry, as we also continue to execute on expanding commercial access through regulatory submissions. As we enter the second phase of launch, in addition to the 10 different reimbursement dossiers that we're in the process of submitting or have submitted that Sebastien mentioned, we also have multiple ongoing or planned regulatory submissions for marketing approval in new geographies and throughout this year. As we speak, our regulatory dossiers are being reviewed in Switzerland, Australia, and Canada, and we're working towards a submission in Japan in the second half of this year. Importantly, for the younger Pompe community, we continue to enroll the ongoing open-label ZIP study for children living with late-onset Pompe disease and the open-label study for children living with infantile-onset Pompe disease. We see this as an important opportunity to support label expansions into these patient segments in the years ahead and to address the significant unmet needs in these patients. Through our ongoing studies in the Amicus Pompe registry, we continue to generate evidence on the differentiated mechanism of action and the long-term impact of Pombiliti and Opfolda across endpoints and different patient populations. As a reminder, in February, we once again had a significant presence and a very successful set of interactions at the 20th Annual World Symposium. We had 11 posters and an oral presentation highlighting our continued work across Fabry and Pompe disease. Our medical conference presence is an important part of our education efforts. Finally, as highlighted in the pipeline side in the appendix for our earlier-stage pipeline, we continue to focus on novel approaches for Fabry and Pompe, including the delivery of our engineered GLA and GAA transgenes as well as the next-generation Fabry Chaperone.
Simon Harford, CFO
Thank you, Jeff. Our financial overview begins on Slide 14 with our income statement for the first quarter ending March 31, 2024. For Q1, we achieved total revenue of $110 million which is a 28% increase over the same period in 2023. At constant exchange rates, revenue also grew 28%. The global geographic breakdown of total revenue during the quarter consisted of $73 million or 66% of revenue generated outside the United States, and the remaining $37 million or 34% coming from within the U.S. Cost of goods sold as a percentage of net sales was 12% in Q1 due to validation costs in the quarter as compared to 8% for the prior year period. Total GAAP operating expenses increased to $125 million for the first quarter of 2024 as compared to $117 million in the first quarter of 2023, an increase of 6% due primarily to a facilities lease restructuring charge. On a non-GAAP basis, total operating expenses increased to $86 million for the first quarter of 2024 as compared to $81 million in the first quarter of last year. We define non-GAAP operating expenses as research and development and SG&A expenses excluding stock-based compensation, loss on impairment of assets, changes in fair value of contingent consideration, restructuring charges, and depreciation. On a GAAP basis, net loss for the first quarter of 2024 reduced to $48 million or $0.16 per share compared to a net loss of $53 million or $0.18 per share for the first quarter of last year. In Q1 2024, there was a non-GAAP net loss of $4.6 million driven by the negative impact of foreign exchange. This was in line with our expectations of a small loss in Q1 as absolute dollar revenue is traditionally lower in the first quarter. Cash, cash equivalents, and marketable securities were $240 million as of March 31, 2024, compared to $286 million as of the end of December 2023.
Bradley Campbell, CEO
Thank you, Simon. We are guiding to full-year 2024 total revenue growth of 25% to 30%. This is driven by the increase of our full-year 2024 Galafold revenue growth guidance from 11% to 16% previously to 13% to 17% currently at constant exchange rates. It is also driven by guidance of $62 million to $67 million for Pombiliti and Opfolda sales for the full year. On full-year 2024 non-GAAP operating expense guidance remains at $345 million to $365 million. With our commitment to full-year non-GAAP profitability during the first full year of launch of Pombiliti and Opfolda, we are keeping operating expense growth in low single digits year-over-year at the midpoint of guidance. As a reminder, we continue to have R&D commitments, including registry studies in both Fabry and Pompe, the ongoing Pompe Phase III study in countries not yet reimbursed, as well as next-generation manufacturing for Pombiliti. With our total revenue guidance of 25% to 30%, we remain comfortably on track to achieve our first full year of non-GAAP profitability in 2024 as profitability grows quarter-by-quarter for the remainder of the year. And with that, let me turn the call back over to Bradley for our closing remarks. Bradley? Thank you, Simon, Jeff, and Sebastien for the insights. As you can see, we have been highly focused on execution this year across the business, and we've made a strong foundation for what will be another important year of commercial execution. We're confident in our leadership position in Fabry and Pompe disease and that Amicus can continue to drive sustainable long-term value and deliver life-changing therapies to people in need. With that, operator, we can now turn the call over to questions.
Operator, Operator
The first question comes from Anupam Rama with JPMorgan.
Anupam Rama, Analyst
As you guys were constructing sort of the guidance for Pombiliti and Opfolda and you started looking at Street models, where did you see the biggest disconnect between your guidance and Street models, given that almost $30 million divergence there?
Bradley Campbell, CEO
Thanks, Anupam. Appreciate the question. I think the most important thing is that when we came into the launch and then into this year as well, we were pretty clear that we needed 6 to 12 months of trends to be able to provide accurate guidance. I think you've seen historically that once we provide guidance, we do a great job of executing against that, as we saw again here at Galafold. The biggest drivers of what we really needed to understand coming into the year were the timing to get new patients through the insurance process in the U.S. And I think we have a clear view of that now. I think that's probably the biggest disconnect from where just the rate of revenue was coming into the year versus what perhaps the Street was initially modeling. Of course, we wanted to see the rate of new commercial starts, which as we've seen now, is building momentum, which we expected to see. The timing of launches in Europe is also something we needed to understand in terms of reimbursement process and positive outcomes. The last piece is just factoring in that time from prescription to infusion, which you also get a higher distribution of revenue coming from European patients, which, of course, is at a lower price. Those two things have compounding effects. But now, look, we have the information we need. The big picture here is that this is shaping up to be a very successful launch. As Sebastien said, it's almost double the first year that we did for the first full year of Galafold. The big picture for us is that this will be a significant driver of growth this year, and it sets us up for our ambition to become the standard of care in Pompe, which is a $1 billion product opportunity that we are aiming for. Now we can do what we do best, execute against our guidance and drive growth in both of these products and then deliver on our financial performance with that non-GAAP profitability.
Operator, Operator
The next question comes from Joseph Schwartz with Leerink.
Unknown Analyst, Analyst
This is Will, on for Joe. One for us, just on manufacturing. We've seen the BIOSECURE headline. Could you just remind us of how you feel about your ability to navigate the situation? Has the new manufacturing facility come online in Ireland yet? Can you just remind us of where you are in that process and your ability to supply and stockpile throughout the year?
Bradley Campbell, CEO
Yes. Great question. Thanks, Will. I'm glad you raised those headlines that came in right when we were doing the call here. I think Endpoints reported what we had anticipated coming out, which is the new markup from the Senate of the BIOSECURE bill that proposed a 2032 grandfathering phase. Effectively, contracts that are existing would be grandfathered in out to 2032. So that would give us plenty of time to find a second source of manufacturing if that bill ever comes to pass. I know that’s been a big focus for investors and analysts. It has been for us as well. But behind the scenes, we have grown increasingly confident that now Congress understands the importance of that transition period, and I think that 8-year proposition would be plenty of time for us to find a second source. In the meantime, we've made great progress against any supply challenges. As a reminder, our target is 18 to 24 months of supply throughout the supply chain, and we are currently trending at about 24 months at the high end of that range. We also began moving products out of China into a central depot in Europe, and we have started moving those products further into the market, sitting in the U.S., U.K., and Europe. More than 65% today of our global supply fits either in Europe, the U.K., or the U.S. We've done a great job mitigating our supply chain issues. In Ireland, we are making great progress. PPQ batches are underway, and we expect that product will come into the supply chain in the back half of next year or early 2026. We've made significant progress in all of these areas, and we have high confidence that whatever comes from the BIOSECURE Act, we have plenty of time to react.
Operator, Operator
Our next question comes from Eliana Merle with UBS.
Eliana Merle, Analyst
In terms of your guidance, can you help us understand the assumptions that go into it for Pombiliti, specifically the mix between U.S. and ex-U.S. revenues as patients start? What markets ex-U.S. does this include salesforce? Also, can you remind us what proportion of the EAP and clinical trial patients are now reimbursed versus the proportion that are not yet reimbursed?
Bradley Campbell, CEO
Yes. So on the first part regarding the rate of new patient capture, our current focus is on our launch countries: the U.K., the U.S., Germany, and now Spain and Austria. We expect several European countries to come on board throughout the second half of the year, including EU5 or EU4 countries, which will contribute to the patient capture rate, making it crucial to maximize the number of patients by year-end to improve our run rate for next year. In terms of revenue distribution between the U.S. and Europe, keep in mind we had more patients in clinical trial and EAP drug coming out of Germany and the U.K. than we did in the U.S. For the majority of this year, we expect it to be still weighted more heavily towards Europe, especially as we have more launch countries in the second half of the year. Over time, we would expect the revenue proportion to shift to something around 40%, coming from the U.S., and the balance coming from ex-U.S. markets. Regarding the clinical trial EAP patients in Europe, we currently have about 65 or 70 adults, with about 40 of them located in Europe, while the rest are in regions like Japan, Australia, Canada, etc. Among those in Europe, roughly 20 are in the countries where we anticipate reimbursement this year, which will be staggered toward the second half of the year.
Jeffrey Castelli, Chief Development Officer
Yes. Thanks, Brad. Regarding remaining patients in trials and expanded access, there are about 65 or 70 adult patients, with approximately 40 in Europe. The remainder is spread across regions like Japan, Australia, Canada, etc. Of the 40 in Europe, around 20 are in those 10 countries that we expect will secure reimbursement this year, although this will also be staggered towards the second half of the year. Furthermore, we have several pediatric patients and continue our enrollment effort that should help us build the patient base.
Operator, Operator
The next question comes from Ritu Baral with TD Cowen.
Chi Wen Chin, Analyst
This is Athena, on for Ritu. Another question on BIOSECURE. If the Ireland plant changes hands, what percentage of the raw material going to Ireland or from China stores? And what percentage are sole-sourced China supply?
Bradley Campbell, CEO
Yes. It's a fair question. We are also exploring ways to source materials from outside of China. That 8-year extension period would give us ample time to make those adjustments. Many of the raw materials have, in fact, come from European-based companies already. Congress has come to understand that while this initiative might be important, the number of patients who take a medicine that has components manufactured elsewhere necessitates a transition period. An 8-year timeframe would be sufficient for us to handle those changes.
Operator, Operator
The next question is from Jeffrey Hung with Morgan Stanley.
Unknown Analyst, Analyst
This is Catherine on for Jeff. We just had one on the Amicus Pompe registry. Can you talk more about what your goals are here? And any expectations that you have on how many patients you might be able to enroll on an annual basis, especially when it comes to your goal of maximizing Pombiliti and Opfolda patients on treatment by year-end? Any color here would be appreciated.
Bradley Campbell, CEO
Sure. Thanks for the question. The registry serves two primary purposes. First, it is a commitment for post-marketing requirements. We have an obligation to conduct it. However, the real value lies in the real-world evidence generated, which can further provide proof to physicians and patients regarding how they are responding to treatment. One unique opportunity we have is that we have a registry coming from the U.K.'s EAMS program, where patients have already been on drug for 1 to 2 years. This provides a real-world near-term opportunity to generate data for prescribers and patients. Jeff, could you provide additional details on both that registry and the broader enrollment goals?
Jeffrey Castelli, Chief Development Officer
Yes. Thanks, Brad, and thank you, Catherine, for the question. The vision for the Pompe registry is to cover reporting requirements for safety and continue to collect real-world data on these patients. Our plan is to have that registry available in most of the countries where we go commercial, targeting hundreds of patients. The exact aspirations remain in the hundreds, but importantly, it focuses on commercial patients who transition from access and trials into the registry to continue to collect long-term data in terms of both safety and quality of life. So, we have registries targeted in places like the U.S., U.K., Germany, Spain, where we are actively going commercial.
Operator, Operator
The next question comes from Kristen Kluska with Cantor Fitzgerald.
Kristen Kluska, Analyst
For Galafold, we haven't really seen much of a slowdown in terms of the ability to continue finding naïve patients. So can you comment on your expectations for the cadence of this moving forward? Or do you see any early signals of this slowing down?
Bradley Campbell, CEO
Thanks, Kristen. Yes, I think that's a really important point. We highlighted previously in our presentation the remarkable growth in this market. Just as a quick reminder, when we launched 7 or 8 years ago, we estimated about 10,000 diagnosed patients, about half of them being treated. Now we're treating 11,000 patients globally between Galafold and other therapies, but there are a further 6,000 diagnosed untreated patients. We think that this rate of diagnosis will continue to grow. It's due to a combination of low-cost, readily available genetic testing, much more intelligent screening, and of course, the work we are doing along with others in artificial intelligence. We think you're going to continue to treat those diagnosed untreated patients as they move through their disease progression, and we've seen that already with Galafold. If we look at the statistics, this is the most under-diagnosed genetic disease, and there could be double or even triple the number of patients living with Fabry who are not yet diagnosed. This is a critical aspect of Galafold's narrative, and we believe it would serve as a great option for newly diagnosed patients.
Operator, Operator
The next question comes from Tazeen Ahmad with BofA Securities.
Tazeen Ahmad, Analyst
Brad, I just wanted to ask you about Pompe. When you came up with your guidance for the year, what proportion of that guidance is an assumption that you're making about how long patients are staying on Nexviazyme products before switching? We have had conversations suggesting that patients could start switching as early as 2 years from their previous drug. How is that working out in the real-world setting so far? And what part of your guidance is that contributing to?
Bradley Campbell, CEO
Yes. Thanks, Tazeen, and I look forward to seeing you at the conference next week. It's a great question. What we had heard coming into the launch was that physicians estimated they needed about a year's treatment before they would consider switching patients. However, there are patients expressing a willingness to switch within 6 months if they aren’t doing well. If you look at the switch dynamics that we highlighted for the United States, we’re observing around 75% of our switch patients coming from Nexviazyme and 25% from Lumizyme. This appears to accurately reflect the situation since Nexviazyme launched a little over 2 years ago, leading to a bolus of patients transitioning past that kind of 1-year period. There are physicians who may be considering a 2-year period, and our expectation is that as we demonstrate favorable outcomes for switching patients, this could shape their thinking to be more proactive in that regard. This will be key to building momentum. Overall, the trends we're observing are in line with our expectations for that 1 to 2-year switch period. Internationally, remember that Nexviazyme has been in the market for a shorter duration, and we are launching head-to-head, seeing switches primarily from Lumizyme and also impressive uptake from naïve patients. As long as those trends continue, we think they will actually strengthen and thereby enhance our guidance with ample room for growth.
Operator, Operator
The next question comes from Dae Gon Ha with Stifel.
Benazir Ali, Analyst
This is Benazir, on for Dae Gon. Can you speak to your appetite for business development opportunities to bolster the pipeline? Have the volatile markets and depressed valuations drawn your attention, and if so, what therapeutic areas are you considering?
Bradley Campbell, CEO
Thank you for the question. Yes, Sebastien, can you provide an overview of how we're approaching business development opportunities?
Sebastien Martel, Chief Business Officer
As you know, we still have plenty of growth opportunities arising from Galafold, as we've discussed, and we are in the midst of launching Pombiliti and Opfolda. Thus, the very strong growth rate that we've provided today reflects this. Yet, we've also been conducting our homework on the business development and M&A side and have developed a framework around potential disease areas or specific indications that might be of interest. We have developed a solid infrastructure from a commercial and medical standpoint, as well as from a development and regulatory viewpoint, enabling us to leverage existing resources. We are considering what we term 'adjacency', looking at conditions like Fabry as a rare renal or rare cardiac disease or Pompe as a rare neuromuscular or broadly rare neuro disease. These therapy areas are of interest to us. When it comes to the stage of development, we are more focused on later-stage opportunities. This includes marketed assets, recently approved drugs, or products in the launch phase or currently undergoing Phase III trials. Our emphasis is on assets that can quickly provide a fair amount of accretion rather than those investing in early-stage tech platform opportunities.
Operator, Operator
The final question comes from Salveen Richter with Goldman Sachs.
Unknown Analyst, Analyst
Can you share what you're hearing from physicians in the EU regarding the real-world experience with Pombiliti and Nexviazyme? Are there any emerging details as real-world data becomes available? Additionally, in the U.S., could you discuss the measures being taken to reduce the timeline from prescription to infusion, which currently stands at 70 days?
Bradley Campbell, CEO
Yes. Thank you for the two questions. We continue to hear incredibly positive anecdotes from physicians and patients in the United States, especially regarding their experiences with Pombiliti and Opfolda. I won't speak on other products, but I believe these will significantly influence word of mouth and confidence in this product. We are observing truly inspiring outcomes, which will encourage others. Dr. Mark Roberts, who presented at the World Congress this year, shared case studies from the EAMS program, being one of the EAMS physicians in the U.K. That’s a valuable resource. Additionally, another EAMS site published a poster last year regarding several patients’ treatment responses. We are continuing to build that body of evidence through both the registry, as Jeff noted, and by focusing on physicians who have those experiences. This will be an essential momentum driver moving forward. In response to the second question regarding the insurance process, there are two main components. First, when launched, we are not included in formularies. Consequently, undergoing what's called a letter of authorization process takes longer—this is something we have learned from Galafold. Then, at the start of the new year, we begin to become part of formularies for hospitals and major insurers, which we've been successfully navigating, even seeing positive results with Medicare and Medicaid. The second aspect involves purchasing the drug, scheduling the infusion, and managing effective timelines. Our goal during steady state is to ideally bring this down to approximately 30 to 45 days, much like we see with Galafold. As a note, the last two recent commercial patients in the U.S. were in that timeframe. We are committed to making meaningful improvements in this area, and we've made good progress so far this year.
Operator, Operator
That was your last question. This concludes today's conference call. Have a great day.