Amicus Therapeutics Inc

NASDAQ:FOLD

Earnings Call Analysis

Amicus Therapeutics Shows Strong Growth and Raises Revenue Guidance

Amicus Therapeutics reported impressive financial results for the second quarter of 2024, with a 34% year-over-year increase in revenue, totaling $127 million. This was led by their flagship product, Galafold, which generated $111 million in revenue, representing a 19% growth. Strong patient demand and strategic market penetration raised the 2024 revenue growth guidance for Galafold to 14%-18%. Additionally, the company's new therapy, Pombiliti and Opfolda, contributed $16 million and is expected to reach $62-$67 million in sales for the full year. Amicus is on track to achieve its first full year of non-GAAP profitability in 2024.

Growth Trajectory of Galafold

In the second quarter of 2024, Amicus Therapeutics showcased a robust revenue performance, driven by their leading product, Galafold. The revenue from Galafold alone reached $111 million, translating to a notable 19% growth year-over-year when adjusted for constant currency. Given this solid performance, the company raised its guidance for full-year revenue growth from its earlier range of 11% to 16% to a new range of 14% to 18%. This adjustment reflects both the strong demand and the expanding patient base for Galafold across various markets, especially in the U.S., where new patient starts have been at the highest level seen in five years.

Revenue and Guidance Overview

Overall, Amicus reported total revenue of $127 million for Q2 2024, marking a significant 34% increase compared to the same quarter the previous year, or a 36% growth at constant currency. The company has also raised its full-year 2024 total revenue guidance to a range of 26% to 31%, an increase from 25% to 30%. This solid growth trajectory is bolstered by ongoing global demand for both Galafold and new therapies, Pombiliti and Opfolda, which have also begun generating revenue.

Emerging Potential of Pombiliti and Opfolda

For the newly launched Pombiliti and Opfolda, the revenue reported in Q2 was $16 million, reflecting a 44% increase compared to Q1 2024. This significant uptake positions these therapies as potential game-changers in the treatment of late-onset Pompe disease. Amicus is targeting $62 million to $67 million in combined sales from both therapies for the full year, emphasizing their robust market acceptance. With ongoing efforts to enhance patient access and the positive reception from healthcare providers, Amicus believes they are well on their way to achieving this goal.

Market Penetration and Patient Demand

The company concluded the second quarter with more than 60% of the global market share for treated Fabry patients with amenable mutations, with expectations that there are still many potential patients to be diagnosed and treated. The appeal of Galafold is underscored by its sustained compliance and adherence rates exceeding 90%. In addition, the company reported significant growth in newly diagnosed patients, particularly females, which offers further upside potential as awareness and treatment approaches continue to evolve.

Steady Pipeline and Expansion Strategy

Forward-looking efforts focus on sustaining double-digit revenue growth for Galafold while successfully launching Pombiliti and Opfolda across new markets. The company remains committed to enhancing its pipeline to include next-generation therapies targeting both Fabry and Pompe diseases, indicative of a long-term strategic vision. They also plan to expand into markets such as Japan and Australia, which could further increase their footprint.

Focus on Non-GAAP Profitability

The strong revenue performance is underpinned by effective cost management, which has seen total GAAP operating expenses decline to $100 million in Q2 from $104 million in the prior year. This careful management of expenses alongside growing revenues positions Amicus towards achieving non-GAAP profitability for the entire year, an important milestone as they scale their operations.

Conclusion and Strategic Outlook

In summary, Amicus Therapeutics is navigating a promising growth trajectory with Galafold exhibiting strong market adoption and expanding its leadership in the Fabry market while Pombiliti and Opfolda are quickly adding to the revenue stream. The upward revisions in revenue guidance reflect confidence in their operational execution and the broad acceptance of their therapies in the medical community. As Amicus continues its strategic focus on expanding market access and enhancing profitability, investors may find a compelling narrative in its commitment to delivering value for patients and shareholders alike.

Earnings Call Transcript 2024-Q2

[Operator]

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Second Quarter 2024 Financial Results Conference Call and Webcast. [Operator Instructions] As a reminder, this conference call 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, executive]

Thank you. Good morning, and thank you for joining our conference call to discuss Amicus Therapeutics' Second Quarter 2024 Financial Results and Corporate Highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer; Sébastien 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 this 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 representation by us that any of our plans will be achieved. Any or all 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 risk and uncertainties that may impact them, we refer you to the forward-looking statements and Risk Factors sections 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 June 30, 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, executive]

Great. Thank you, Andrew, and welcome, everyone, to our second quarter 2024 conference call. I'm pleased to highlight what has been a very successful first half of the year, across our global business and particularly an incredible quarter. In this time, we have continued to build on our top line revenue growth momentum to put us well on our way to our first full year of non-GAAP profitability, while also advancing our mission of bringing hope to individuals and families affected by rare diseases.

As we did in this morning's press release, let me highlight several key points. First, we continued our excellent commercial execution and delivered total revenue of $127 million in the quarter, representing 34% growth year-over-year or 36% on a constant currency basis. This strong performance has led us to increase our total revenue guidance for the full year to 26% to 31%, from 25% to 30% previously. At the product level, Galafold continues its strong performance with $111 million in global revenue in the quarter, which represents 19% growth from last year on a constant currency basis. In the first half of the year, operational growth was 17% year-over-year at constant exchange rates coming in at the high end of our guidance for the year.

We continue to observe strong trends across our key performance indicators in all key geographies in the second quarter including continued demand through new patient starts, both from switch and naive 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 diagnosed and treated patients that we've highlighted previously. Eight years now after our first commercial launch, it's remarkable to see what the impact this medicine is having on people living with Fabry disease around the world.

And we continue to see Galafold serving as the foundation of our business for the next decade and beyond. Based on the sustained performance, we are increasing our full year 2024 revenue growth guidance range for the second time. We started the year with a growth range of 11% to 16%. In May, we raised it to 13% to 17%, and now we're projecting 14% to 18%. Second, let me highlight the continued strong global commercial launch of Pombiliti and Opfolda, our novel therapy for late onset Pompe disease. Pombiliti and Opfolda has been and will continue to be a huge growth driver for us this year. We've already made great progress against our key performance indicators, which continue to demonstrate the strength of this launch.

First and foremost, our #1 focus for the 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 Pombiliti and Opfolda in 2024, continues to progress exceptionally well. In the second quarter, we saw the largest number of new commercial patients, meaning patients who were not in our clinical studies. And as of the end of July, we had 186 patients who had been treated or scheduled for treatment. We are incredibly pleased with the demand globally from patients and physicians for this new therapy and consistently hear inspiring anecdotes from health care professionals around the world on how their patients are responding to Pombiliti and Opfolda, which will continue to fuel the momentum throughout the year and beyond. Sebastian will provide more details in a moment, but the switch dynamics in the U.S., Europe and the U.K. continue to look strong, and we're seeing great uptake in naive patients and markets outside the U.S. as well. We're also making significant progress on reimbursement front globally.

And this includes moving patients more quickly through the insurance process in the U.S. as we've anticipated. Throughout the remainder of the year, we'll focus on increasing patient access as we gain reimbursement and launch in additional countries throughout Europe. On the regulatory front, we're pleased to announce that in July, the Swissmedic approved Pombiliti and Opfolda for adults living with late onset Pompe disease in Switzerland. For the full year 2024, we're well on track to deliver our guidance of $62 million to $67 million in global Pombiliti and Opfolda sales, which will be a significant contributor to our growth and set us on a great course to achieve our ambition for PomOp. To become the new standard of care treatment in this devastating disease.

We're incredibly pleased to be providing a real choice for patients and challenging therapeutic expectations for both physicians and people living with Pompe disease. And finally, as we continue our excellent commercial execution across both therapies and prudently manage our resources, we are excited to share that Amicus was profitable on a non-GAAP basis in the second quarter as well as the first half of the year. We are confident we'll see this number increase as we look to achieve our full year non-GAAP profitability in 2024. A head on Slide 4, as we laid out the beginning of the year, we're focused on achieving our 4 key strategic priorities for 2024, including: first, sustaining double-digit Galafold revenue growth. And again, on the back of strong sales growth so far, we've raised the expected annual growth rate of Galafold to 14% to 18%. Successfully executing on multiple commercial launches of Pombiliti and Opfolda, advancing our ongoing strategy to support our medical and scientific leadership in Fabry and Pompe disease and maintaining a strong financial position as we carefully manage our expense investments to achieve non-GAAP profitability for the full year.

With that highlight, let me now hand the call over to Sebastien, who will go through our commercial performance in more detail. Sebastian?

[Sebastien Martel, executive]

Thank you, Bradley. And good morning to everyone on the call. As always, I'll start by providing you with more details on our Galafold performance for the quarter. On Slide 6 for the second quarter 2024 Galafold reported revenue reached $111 million, driven by strong patient demand, particularly from our leading markets. We ended the second quarter with more than 60% of the global market share for treated Fabry patients with amenable mutations. And the good news is there are still many more potential patients eligible for our therapy.

As Bradley highlighted, given the continued strong performance of Galafold throughout the second quarter, we're raising again our full year 2024 revenue growth guidance range to now 14% to 18%. Turning to Slide 7. Our results in the second 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 19% over the same period in 2023 at constant exchange rates. Our leading markets such as the U.K., the U.S., [ top ] new countries and Japan, remain the biggest driver of patient demand and gives great confidence in the growth this product has over the long term. The U.S., in particular, saw impressive patients demand the highest number of net new patients seen in over 5 years, and we expect this trend to continue for the full year.

Within the global mix, which is about 42% switch and 58% naive, we're now seeing a stronger uptake in naive populations. We continue to achieve high market shares in countries where we've been approved the longest. There's plenty of opportunity to continue to switch patients over to Galafold and to keep growing the market as we penetrate the diagnosed untreated and newly diagnosed segments. All of that is underpinned by sustained compliance and adherence rates that continue to exceed 90%. And reiterating our belief that those patients who go on Galafold predominantly stay on [ Gala ]. We're very pleased to see that prescribers continue to position Galafold as the treatment of choice for amenable [ public ] patients. This leadership position is supported by our continuous investment in medical education and evidence generation.

I'm very pleased to recognize the first publication of the results on our own Fabry disease registry, the following registry in July. These results were published in the Journal of inherited metabolic disease and constitute a really important milestone for Amicus as we continue to generate positive data for Galafold. Data from the study aligned very well with previous observations from clinical trials and extends the available data supporting the real-world multisystem benefits of Galafold. This publication, along with our ongoing medical indication of congresses, the important part of the story of this oral precision medicine and help support the long-term growth potential we envision Galafold will have in this growing public market.

On Slide 8, we know that there is a significant patient demand for Galafold then that the segment of the global Fabry market made of patients with amenable mutation has 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 drivers. First, the Fabry market is growing robustly the significant portion of growth coming from finding new patients and reaching the diagnosed and treated population. As we mentioned previously, at the end of 2023, there were more than 2,400 indivitals on Galafold and about 1,400 of those were individuals were naive to any treatment before Galafold.

And those numbers obviously continue to grow this year, and we'll be pleased to report an update at our year-end results. We've seen many new patients going to treatment through newborn screening, family screening, and we continue to increase patient adventication capabilities. Through ongoing medical indication and support of noble screening initiatives. Fabry remains unfortunately one of the most underdiagnosed rare diseases for the more patients that can be identified the more patients may benefit from Galafold. We're also seeing many diagnosed untreated patients transition to treatment as the need for earlier treatment, especially in females, becomes better appreciated.

The other piece is continuing to drive Galafold market share of treated amenable patients through continued commercial execution. As noted, Galafold currently has more than 60% of the global emailable market, what we're seeing in our most mature markets, it can reach up to 85%, 90% of market share. So we know that there's the potential to reach those levels in the global market share as well. And again, all of these efforts are supported by solid compliance and adherence rates through physician and patient education and support program.

Additionally, we'll continue to make progress on expanding Galafold into new markets and extending the labels. The steel some markets in Latin America, the Middle East and Asia Pacific regions for Galafold this either newly reimbursed or we expect reimbursement. Also important to note here, we have often drug exclusivity in the U.S. and Europe. And in addition to our now 61 [ Orange ] listed patents, 45 of which provide protection into 2038 and beyond, including 14 composition of [indiscernible].

This provides us the opportunity to continue 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 a long runway well into the next decade.

Turning now to Pompe disease on Slide 10. We outline our global launch progress with Pombiliti and Opfolda. For the second quarter 2024, Pombiliti & Opfolda reported revenue reached $16 million. This represents an increase of 44% compared to the first quarter of 2024 and provides a strong foundation for the remainder of the year. In the U.S., Q2 benefited from the remaining clinical trial patients transitioning to commercial treatments in addition to patients switching from other therapies. We continue to see a majority of patients switching from Nexviazyme about 68% and the remaining from Lumizyme. This means we're switching patients proportionately from both products in the U.S. We're also seeing a broadening and deepening of our prescriptions with more sites coming online and multiple new prescriptions from physicians coming in at accelerated and increased rates of [ Q1 ]. Outside of the U.S., we're seeing patients from all 3 segments, some switching from Myozyme, some from Nexviazyme at a proportional rate to the respective market shares and some from the naive population. That's exactly what we want to be seeing at this stage in the launch.

A few updates on the launch in Europe. We're pleased to announce that Switzerland, Swiss Medica has a pro Pombiliti and Opfolda a long-term enzyme replacement therapy, and enzyme stabilizer for adults with LOPD. And now the key regulatory authority who recognizes the benefits of this therapy. In Germany, we have successfully finalized our negotiations with the National Association of Statutory Health Fund, and we're very pleased with the outcome as it continues to reflect the value of health technology assessments are seeing this therapy. The price acknowledges the added benefit of Pombiliti + Opfolda for OPD patients and will be closely aligned with the price we set at launch.

Additionally, we anticipate this to be an important reference point for future pricing and reimbursement discussions. In Spain, our newest launch country, we've added a significant number of patients and new prescribing accounts, all within the country's first quarter of launch. We remain very pleased with the take of Pombiliti and Opfolda in the U.K., where we've captured greater than 1/3 of market share within the region. With the benefit of beams, Pombiliti and Opfolda have actually been in the market for 2 years in the U.K., and we believe that the current market share is indicative of how this product can perform over a 2-year period which is a great example of this potential demand and uptake.

Even Pombiliti and Opfolda solid commercial execution thus far, the first half of 2024, we're well on our way to deliver our full year revenue guidance of $62 million to $67 million for Pombiliti and Opfolda.

Moving to Slide 11. We're very pleased with the launch momentum through the first half of the year. As Bradley mentioned earlier, there are about 196 patients that have been treated or are scheduled to be treated at the end of July. That's about 170 for on therapy, and the remaining 12 were scheduled. We remain 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. Q2 in particular, was a very strong quarter, having seen the largest number of new demand patients being prescribed Pombiliti and Opfolda. These are individual not from clinical trials, choosing to come onto commercial therapy. This is important that it further supports our business that launch momentum will continue to be 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 launches leverage our highly experienced cross-functional teams, and we've had great outreach with [ TLP ] leaders.

We're seeing an increase in debt and breadth of prescribers across all 3 markets. In Q2 alone, the global number of prescribing accounts increased by 50%. All core treating centers have been engaged, and they've provided very positive feedback feedback from both HCPs and other stakeholders as to our business approach, our support and our patient focus.

Finally, we find an important metric to track is our progress with access and reimbursement. We have a highly experienced team are engaging these positive conversations with payers to demonstrate the value of Pombiliti and Opfolda. In the U.S., the largest payers have already put Pombiliti and Opfolda on to their respective formularies. And we have also seen strong acceptance by Medicare Medicaid. The overall time from prescription to infusion is now down to around 65 days with payer approvals happening in around 30 days. And the last few commercial patients have actually come in significantly quicker. We believe this will continue to improve.

Today, we're launched in Germany, the U.K., U.S., Australia, Spain, but we remain in active pricing and ribosome discussions with additional major European markets as we focus on securing broad patient access throughout the European market. Overall, we've had a very strong first half, and we're very pleased with the building momentum on patient demand. Throughout the second half of 2024, our focus will be on maximizing the number of patients on therapy by year-end.

So in summary, we're very pleased with the launches of Pombiliti and Opfolda across the first wave of countries, the strength of our clinical data, the depth of our experience and talent we have at Amicus gives us great confidence in our ability to make a real difference for people living with Pompe disease. We believe Amicus is in a great position with our second commercial launch. And with that, I will hand the call over to Jeff Castelli, our Chief Development Officer, to discuss the ongoing clinical study and regulatory time lines. Jeff?

[Jeffrey Castelli, executive]

Thank you, Sebastian, and good morning, everyone. On Slide 12, we outline how we continue to build the body of evidence for Pombiliti and Opfolda through our ongoing clinical studies as well as through our Amicus Pompe registry. As we also continue to execute on expanding commercial access through reimbursement dossiers and regulatory submissions. As we enter this second phase of launch, in addition to the various reimbursement dossiers that we have or are in the process of submitting. We also have multiple ongoing or planned regulatory submissions for marketing approval in new geographies throughout the year. .

We've just mentioned the approval in Switzerland. Additionally, we have our regulatory [ doses ] being reviewed in Australia and Canada, and we're working towards the submission here in Japan before the end of the year. For the younger Pompe community, we continue to enroll the ongoing open-label ZIP study for children living late onset Pompe disease and the open-label Rozelle 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 very importantly, to address the significant unmet needs for these children.

Through ongoing clinical studies in the Amicus Pompe registry, we continue to generate evidence on the differentiated mechanism of action and on the long-term impact of Pombiliti and Opfolda across endpoints and patient populations. Our medical conference presence and publications continue to be an important part of our education efforts.

Finally, as highlighted in the pipeline slide in the appendix for our earlier stage pipeline, we continue to focus on novel approaches to next-generation therapies in Fabry and Pompe diseases.

With that, I would like to now turn the call over to Simon Harford, our Chief Financial Officer, to review our financial results, guidance and outlook. Simon?

[Anupam Rama, analyst]

Thank you, Jeff. Our financial overview begins on Slide 14 with our income statement for the second quarter ending June 30, 2024. For Q2, we achieved total revenue of $127 million, which is a 34% increase over the same period in 2023. At constant exchange rates, revenue also grew 36%. The global geographic breakdown of total revenue during the quarter consisted of $77 million or 60% of revenue generated outside the United States, and the remaining $50 million or 40% coming from within the U.S.. Cost of goods sold as a percentage of net sales was 9% and in Q2 2024 as compared to 10% for the prior year period staying relatively flat.

Total GAAP operating expenses decreased to $100 million for the second quarter 2024 as compared to $104 million in the same period last year, a decrease of 4%. On a non-GAAP basis, total operating expenses decreased to $82 million for the second quarter of this year as compared to $84 million in the same period last year, a decrease of 2%. We define non-GAAP operating expenses, research and development and SG&A expenses excluding stock-based compensation, loss on impairment of assets, changes in fair value of contingent content consideration, restructuring charges and depreciation and amortization. On a GAAP basis, net loss for the second quarter 2024 reduced to $16 million or $0.05 per share compared to a net loss of $43 million or $0.15 per share for the second quarter of 2023.

In Q2, non-GAAP net income was $18 million or $0.06 per share compared to a loss last year in the same period of $20 million or $0.07 per share. Cash, cash equivalents and marketable securities were $260 million at June 30, 2024 compared to $286 million at December 31 in 2023. Turning now to Slide 15. We are raising our full year 2024 total revenue guidance range to 26% to 31%, up from 25% to 30%. This is driven by the increase of our full year 2024 Galafold revenue growth guidance, which started the year at 11% to 16% and was most recently 13% to 17% and now is 14% to 18% at constant exchange rates.

In addition, we are reiterating the guidance of $62 million to $67 million, so Pombiliti and Opfolda sales for the full year 2024. Our full year 2024 non-GAAP operating expense guidance has been narrowed from $345 million to $365 million to $345 million to $360 million or a $0.05 reduction at the top end. With our commitment to full year non-GAAP profitability during the first full year of launch for 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, which aren't yet reimbursed as well as next-generation manufacturing for combility. In the second quarter of 2024, non-GAAP profitability was $18.5 million and for the first half of 2024, $13.9 million. With our total revenue guidance of 26% to 31% growth, we remain comfortably on track for 2024 to be our first full year of non-GAAP profitability as profitability continues to accelerate in the second half of the year.

And with that, let me turn the call back over to Bradley for our closing remarks. Bradley?

[Bradley Campbell, executive]

Thank you, Simon, Jeff and Sebastian. As you can all see, we've been highly focused this year on our commercial execution continuing to strengthen our financial profile and ultimately delivering value for our shareholders. Our first half performance has laid the groundwork for us to continue to achieve our goals and fulfill our mission for delivering life-changing therapies.

And with that, operator, we can now open the call to questions.

[Operator]

[Operator Instructions] First question comes from this question comes from the line of Tazeen Ahmad with BofA Securities.

[Tazeen Ahmad, analyst]

Brad, I just wanted to get a sense from you about how we should be thinking about the cadence of the Europe launch versus how the U.S. launch is going, the label languages are slightly different. And so as we gain traction in U.S., how should we be thinking about how that could or could not be mirroring what you're seeing in Europe?

[Bradley Campbell, executive]

Yes. Thanks, Tazeen. Thanks for the question. So as you mentioned, a couple of things. So first of all, I think as you saw from the numbers today, continuing to see great progress in both markets. I think you have 2 different dynamics going on. In the U.S., it's the largest single market. And so the rate of new patients will continue to be strong as we go throughout the year. However, in Europe, we're adding new countries as we go. We launched in Spain. We just got approval in Switzerland. We're on another reimbursement.

So I think over the course of the year, the balance of the new patients will probably start to favor in the United States, but that's just a matter of size. But overall, I think both markets are progressing extremely well. In terms of the label, as you said, and as I kind of provided on the call, we and Sebastian provided this detail as well. In Europe, we're seeing switching patients from both Myozyme and Nexviazyme as well as new patients, which are on the label there. in the U.S., we're seeing predominantly switches from Nexviazyme and some switches from Myozyme as that's a much smaller portion of the population. So the key performance indicators we're watching are going really well. and we're excited to continue to see the momentum.

[Operator]

Your next question comes from Eliana Merle with UBS.

[Eliana Merle, analyst]

In terms of some of the dynamics in the U.S., I guess, of the patients that are switching from Nexviazyme, I guess, what are you seeing just in terms of trends of the types of patients that are switching -- and in particular, I guess what are you seeing in terms of like the patient average length on next line prior to switching to PomOp?

[Bradley Campbell, executive]

Yes, thanks. I think essentially, we're seeing what we had expected to see, which is the majority of patients switching from Nexviazyme, have been on treatment in that kind of year to 2 years. And that's a dynamic that we've described before. The good news is, of course, there's a huge Bolus of patients that are kind of rolling through that year to 2 years on therapy over the course of this year and into next year. And so I think we've got a large Bolus of patients that we can target -- we do see some patients who have switched earlier in their journey.

And I would say that probably comes more from word of mouth or from knowing other patients in the community who had a positive experience with Pombiliti and Opfolda. And I think that's another dynamic too, that we'll want to continue to see, which is as we establish this as an important new therapy as more physicians and more patients get experience and hopefully those experiences will be positive, I think that creates some demand to switch earlier when they realize what Pombiliti and Opfolda can do for them.

[Operator]

Your next question comes from the line of Anupam Rama with JPM.

[Anupam Rama, analyst]

And congrats on all the progress here. Just on the Galafold guidance increases increase, should we be thinking about any type of regional variation of where growth is coming from or region or regions that are leading to this guidance increase?

[Bradley Campbell, executive]

Maybe I'll start at a high level and then ask Sebastian to add a little color there. So Anupam honestly, we're seeing great growth in all of our key markets, which is what you want to be seeing -- but Sebastian, maybe provide a little bit of color on some of the unique elements of the U.S. and anything else that you think is relevant there.

[Sebastien Martel, executive]

Yes. So thanks, Brad. So Anupam, we're seeing very strong demand for naive patients, newly diagnoesd patients in the U.S. and have seen that for a number of quarters now, that's actually why we've now raised guidance twice this year on Galafold a growing proportion of newly diagnose patients really are put on Galafold as the very first line of treatment if you have amenable mutations. The number of PRS we've recorded in the first half of the year, as I said earlier, is close to the highest we've seen in the last 5 years. So very strong performance in the U.S. We continue to see the shift that we've seen -- for ever since launch, actually, that a growing proportion of female with Fabry disease are also being treated and the tendency of patients who have been diagnosed, moving to treatment. Earlier than they may have in the past. In Europe, we're very pleased to see a bit of a similar pattern with the majority of the growth coming from naive patients.

And we're seeing that in our largest markets in the U.K. We're seeing great performance in Japan as well. We actually think that we have more growth upside in Japan simply because the Japanese business to us is our fourth largest. If you look at the whole family market, Japan is actually the second largest market. So there's no reason why Japan couldn't at some point in time, become our second largest market as well. So these are some of the key highlights on regional performance.

[Operator]

Your next question comes from Joseph Schwartz with Leerink.

[Unknown Analyst]

This is Will on for Joe. Congrats on the progress this quarter. A question on PomOp. Based on the metrics for patients treated or scheduled to be treated, it seems like you've added roughly the same number of patients in the second quarter as compared to the first quarter. Is this within expectations? And how should we think about the inflection point in the back half of the year as other geographies come online in the EU?

[Bradley Campbell, executive]

Yes. Thanks for the question. I think one of the things we talked about, Sébastien highlighted, is the rate of new patients this year continues to be much higher than it was last year, and that's exactly what we expected. In terms of new patient adds, I think we'll continue to build momentum as we go towards the back half of the year, especially as we open up new countries. That being said, from a revenue perspective, especially because a lot of those reimbursement and launches will be in the fourth quarter. the rate of new revenue won't change very much. But obviously, next year, it will be really important. So that's why we focus on sort of maximizing the number of patients on therapy by the end of the year because that maximizes the run rate going into next year.

[Operator]

Your next question comes from the line of Ritu Baral with Cowen.

[Ritu Baral, analyst]

Congratulations on the metrics in this quarter. From our tracking survey, our promo tracking survey that we ran recently we got some input and some good flavor on the competitive dynamics, which for Genzyme, which seem to be quite considerable -- and we're hearing that at least the Nexviazyme team over there have some very interesting messaging about Nexviazyme superiority over Myozyme, Lumizyme that seem to be resonating with some of the doctors that we've surveyed, can you talk to -- I mean if that's the PomOp counter detail, can you talk to your counter detail on what's going on messaging wise, on what defines a decliner -- and how the 2 drugs sort of stack up against each other in the field? And then I've got a follow-up on insurance.

[Bradley Campbell, executive]

Maybe I'll start, and I'll have Jeff add some color. I would just -- I won't speak to how they're detailing. But what I would say is that -- we're the only product that has shown in a controlled phase of the Phase III and improvements in both 6-minute walk and forced capacity versus an active comparator. And for me, the most important thing here is the efficacy of the product. And I think that's a very important part of our label. And in fact, you may remember our promotional materials, which is pretty incredible. We can effectively say improvement is possible with this therapy. And I think that's a resonating message that we're very confident in -- and I think that describes why we've had such a great launch so far.

But Jeff, maybe talk a little bit more about the kinds of things that positions are focusing on and maybe the importance of some of the other data points that they're following.

[Jeffrey Castelli, executive]

Yes. Thanks, Brian. Thanks Ritu for the question. Brett, I think you hit on the main point, which is from an Amicus perspective, we obviously are still educating people on the data from our Phase III, which was largely in ERT-experienced patients. randomized blinded where we showed in that large group of switch patients, improvements on [indiscernible] and the key end points. And as we presented across all the endpoints in that trial, we see patients when switching have improvements across numerous aspects of muscle strength, quality of life, breathing, et cetera. So we're still really educating everyone about that data.

Obviously, that data is one of the highest levels of evidence that you can have. And then there's still continuing education around mechanism of action and unique differences in what we think we've built here with Pombiliti and Opfolda. Moving forward, as I talked about briefly in the transcript, we are really, it's invested in real-world evidence and the registry, looking to see how people are doing when they're on PomOp across all sorts of parameters, whether that's naive patients outside the U.S., people switching from Nexviazyme, people switching from Lumizyme. And we'll continue to report out that data importantly from our registry and other areas. And we're really excited from everything we're hearing anecdotally about the patient physician response to Pombiliti nand Opfolda. But yes, certainly, it's -- there's different messages out there, but we're really pleased with where we are today and where we're going in the future.

[Ritu Baral, analyst]

Great. And some of these are very focused on the reimbursement landscape and there seems to be sort of continuing griping from some of the respondents about insurance coverage. Not that it's terrible, but we've heard anecdotes of not getting the Opfolda component covered, can you speak to how that has been going now that you got a full quarter worth of your new [ J code ]. Has this all mostly been resolved?

[Bradley Campbell, executive]

Yes. Thanks, Ritu. It's funny. I -- one thing we would remind everybody, I think when the first prescription happens and all of these therapies, frankly, the physician or their staff has to go through the preapproval process, and there are 2 products here. But I would say that we haven't seen any patients be denied coverage for either the ERT or the small molecule and we shared the trends. We're seeing things proceed exactly as we'd hoped, which is shortening the time from prescription to infusion. We're now down to 65 days. And then shortening the insurance time, too, which is now down to 30 days.

So I'm sure that the very first time the staff goes through any new therapy, they have to get used to the paperwork, et cetera. And I'm sure that's what you're seeing in some of your surveys. But everything we're seeing and the facts are that we're getting patients through the system, and we're getting them through much more quickly than we did at the start of the process, which is what we want to see. And yes, I'm sure the coding piece helps to some extent there as well.

[Operator]

Your next question comes from the line of Jeffrey Hung with Morgan Stanley.

[Michael Ulz, analyst]

This is Michael Ulz. on for Jeff. Congrats on the progress on all fronts. For Galafold, given the growth you had referenced and diagnosed and treated patients with Fabry, how much more room do you think there is left for new patients add? Just like curious as to why you saw a stronger uptake in naive patients versus switch in 2Q. Thank you.

[Bradley Campbell, executive]

Yes. I'll let Jeff maybe talk a little bit more about that. But the reality is the overall market just continues to grow, and that's fueled by, I think, improving diagnosis through the kinds of things that Sebastian talked about. But Jeff, maybe remind us the sort of change in market size from where we started the launch to today and then kind of what has changed in the diagnostic landscape that we think is fueling that.

[Jeffrey Castelli, executive]

Thanks for the question. As Brad said, it's about 10,000 patients when we launched Galafold 7 or 8 years ago, it's now over 18,000 patients diagnosed and when you look at all the numbers out there from various screening programs, whether it's newborn screening across different countries or at-risk screening of patients that likely have Fabry, it's clear that there are many, many more people and families living with Fabry that are undiagnosed to date than the 18,000 that are diagnosed. So the good news is there's a lot of tailwinds on diagnosis Medical education continues improves, so physicians know when to suspect Fabry. There's a lot more access to screening paradigms, low-cost genetic testing.

A lot more education about when you diagnose the Fabry patient, it's a late-onset male or female, about the burden of treatment and the need to treat earlier. And those are all things at Amicus along with the other companies in the space and physicians have really been doing from a medical education perspective, lots of exciting developments on the kind of medical record use of AI potential to screen for people that might be suffering with [ Fibria ] knowingly. So as we look moving forward, we think there's going to be lots and lots of people being diagnosed. Many of them will have amenable mutations. That's something we've learned here, too, that we seem to see more higher percentage of amenable patients in the new people being diagnosed than historically, largely because late onset patients are harder to diagnose late onset patients tend to have a medical mutations.

And more and more Galafold as a standard of care for people with this mutation. So lots of positive forces all coming together, I think, that are driving what the scene. And particularly in the U.S., the last thing I'll add is there is a newborn screening going on in 5 or 6 states. And when they find a newborn often, that is the index patient in a family and then there's ability to screen the families for the other 3 to 5 members that also have Fabry that are adults and likely suffering. So lots of great tailwinds for the community in terms of finding new patients.

[Sebastien Martel, executive]

And I would add that everything that Jeff said is actually highlighted in the growth rate that we see for the quarter, not just for our sales. So we were again the fastest-growing brand for Fabry with 19% growth when we look at the performance of Fabrazyme and [indiscernible] so the 2 ERTs we estimate that the overall Fabry market growth rate from a dollar standpoint was north of 12% in the second quarter. everything that, again, Jeff mentioned contributes to that overall healthy Fabry market growth.

[Operator]

Your next question comes from the line of Dae nGon Ho with Stifel.

[Dae Gon Ha, analyst]

Maybe going back to, I guess, similar to Ritu's question, our channel checks also indicate that there is a lot of enthusiasm to switch over to PomOp. But I guess what's interesting is A lot of these physicians say sort of the switching impetus is dependent on progression of disease or Nexviazyme not showing any meaningful benefit. Yet those same physicians also talk about how the muscle assessment or other functional assessment frequency hasn't really changed. -- since Nexviazyme launch or PomOp launch. So kind of curious, is that part of your marketing/commercialization strategy to maybe induce more of these assessments so that any kind of slowing of efficacy or progression of disease can be picked up quicker and therefore drive more switch?

[Bradley Campbell, executive]

Thanks, Dae Gon. Yes, it's -- you're hitting a very important issue, which is this is really the first time physicians have had a real choice between products. And frankly, now they have 2 products that they're looking at. And so it is an element of changing expectations for therapy, we focus on, but actually improvement is possible maybe for the first time. So yes, it's very much an educational effort, and it's very much -- again, as patients and physicians get experience with the product, I think it will encourage them to look more closely at not just the primary measure, 6-minute walk for vital capacity, but the other measures that may be changing suddenly over time. And if we can change the expectation of those physicians and patients of what they're getting from their therapy, I think that very much favors Pombiliti and Opfolda.

[Jeffrey Castelli, executive]

Brad, one other thing I'd add real quickly there. it's definitely something we're hearing from all the physicians about now kind of the need for more standardization about how often they're monitoring their patients, what parameters they're able to measure in a given clinical visit. So that's something that Amicus, I think the community is all now having to work together on as we have multiple treatments available and patients really need to be armed better with all the information about making informed treatment decisions. The other side of it from an Amicus perspective is -- we're out there reminding people about the data that we saw in people that switched from ERT to PomOp that those patients, many of them saw improvements across different parameters.

So in some way, stable is not necessarily the best outcome that there is potentially an opportunity to regain some function. So that's something that we're reminding people from the data we see.

[Operator]

Your next question comes from the line of Salveen Richter with Goldman Sachs.

[Unknown Analyst]

This is Sharon for Salveen. So do you see any trends that suggest that physicians are maybe preferring to switch patients from Myozyme to Nexviazyme before and evaluating them on that before going to mobility in both ERT-experienced patients in the U.S. as well as naive patients in Europe. And just a quick second part. You mentioned that you're now down to a 65-day lag between prescriptions to infusions. What is your best estimate around when you get to that 30 to 45 day target that you had mentioned early on?

[Bradley Campbell, executive]

Yes. Thanks for the questions. I think for the first one, no, I think what the dynamic is really that in the U.S., as an example, Nexviazyme was approved far earlier than us. So the majority of patients were already switched over. Although there are some remaining patients from Myozyme. And then outside the U.S., it's much more of a mixed circumstance. So what we've seen is that we're taking switch patients from kind of each segment equally. And I think that reflects that we're making very good headway with with the launch and that the physicians aren't preferencing switching to Nexviazyme first. I think it's just a reflection that we weren't available before.

And then in the second case, as it relates to the time to infusion, from prescription. Our goal is really by the end of this year. We should be down to that 30 to 45 days. That's kind of the journey we are on with Galafold by the end of the first full year of launch, we were down to sort of 30-plus days. The one difference here, the reason why we sort of say 30 to 45 days as these patients come in every other week for an infusion. So you probably have a 2-week kind of infusion window as well.

But yes, we're looking to do that by the end of this year.

[Operator]

Your next question comes from the line of Kristen Kluska with Cantor Fitzgerald.

[Rick Miller, analyst]

This is Rick Miller on for Kristen. Just one from us. For Pombiliti + Opfolda, can you comment on the prescriber mix you're seeing? Does this break down across larger academic centers versus community settings? And are there any other interesting trends that you're seeing on that front?

[Bradley Campbell, executive]

Yes. Great question. I think Sebastian highlighted one of the most important metrics, which is the number of prescribers globally has increased by 50%, and that's really important. We want to see continued breadth, i.e., new prescribers. We also want to see continued depth, which we're seeing as well, which is prescribers adding their second or third or fourth prescriptions. So both of those dynamics have been really strong. To your question in terms of where are they coming from Again, I think it depends on the market. So a market like the U.K., which is very concentrated, all of the key centers are prescribing in markets like Germany and the U.S., you do have a top prescriber base, and we're seeing great penetration into those.

But you also have sort of just because of the size of the geography and the more distribution of physicians. We do -- now we're starting to see more and more, I would say, distal physicians that are prescribing as well. And so those are dynamics we'll continue to speak to. And so far, we're seeing great uptake in both of those segments.

[Operator]

Thank you. That was your last question. This concludes today's conference. Have a great day.