Amicus Therapeutics - Q2 2023
August 8, 2023
Transcript
Operator (participant)
Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics' second quarter 2023 financial results conference call and webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Follin, Vice President of Investor Relations. You may begin.
Andrew Follin (VP of Investor Relations)
Thank you, Tana. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' second quarter 2023 financial results and corporate highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer; Daphne Quimi, Chief Financial Officer; Sébastien Martel, Chief Business Officer; and Dr. Jeff Castelli, Chief Development 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 representation by us that any of our plans will be achieved.
Any or all the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements, which speak only to the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof.
For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the Forward-Looking Statements and Risk Factors section of our annual report on Form 10-K for the year ended 21 December 2022, and the quarterly report on Form 10-Q for the quarter ended 30 June 2023, to be filed later today with the Securities and Exchange Commission. At this time, it's my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley?
Bradley Campbell (President and CEO)
Great. Thank you, Andrew, and welcome everybody to our second quarter 2023 conference call. I am very pleased to highlight what has been an incredibly successful first half of the year across our global business. As we did in this morning's press release, let me highlight several key points. First, Galafold continues its strong performance and remains the cornerstone of our success. We remain very pleased with the commercial uptake of Galafold globally, with $180 million in revenue for the first half of 2023. This represents 16% growth from last year on a constant currency basis. In the second quarter, operational growth was 17% year-over-year at constant exchange rates. We continue to observe strong trends across our key performance indicators in all key geographies in the second quarter.
This includes increasing demand through new patient starts from both the switch and naive populations, steady growth of in-person visits between our field team and Fabry treaters around the world, and sustained patient compliance and adherence rates of over 90%. Growth in the second quarter was driven primarily by patient demand, as Sébastien will highlight in a moment, we saw new patients starting on Galafold globally throughout the second quarter at rates we haven't seen since the first few years of launch. Based on Galafold's strong first half performance, we're pleased to be raising our full year 2023 revenue growth guidance range to 14%-18% at constant exchange rates. Second, we continue to make great progress on our global regulatory filings and have now commenced the commercial launch for Pombiliti and Opfolda, our novel two-component therapy for Pompe disease.
Following the EC approval of Opfolda in June, the commercial launch is well underway in Germany. Our team is in the process of switching over individuals from clinical studies and expanded access programs to commercial supply, and we're pleased to share that the first patients have now been dosed with commercial product, with multiple additional patients having been scheduled to start their infusions in the coming days and weeks. Importantly, we're well on track to transition the 20 clinical trial and expanded access patients within our 90 day target. Outside of Germany, we're focused on broader patient access across Europe as we navigate the country-by-country pricing and reimbursement process. In the U.S., we've been pleased to see an increased level of engagement from the agency in recent weeks and are confident now we're on track for an FDA approval in Q3.
Finally, the UK regulatory process for AT-GAA was initiated in December of last year, and we've just received word this morning that the MHRA has approved Opfolda . As a reminder, MHRA previously approved Pombiliti, and both have received orphan designation. We now expect the NICE final assessment document to come in the next few days, at which point it will take about 30 days for the NHS to provide funding to local centers to begin commercial infusions. With our international launch commenced and poised for regulatory approval in the U.S. and additional key markets on the horizon, we look forward to providing a real choice and challenging therapeutic expectations for both physicians and the people living with Pompe disease around the world.
Amicus has maintained a strong financial position as we continue to execute on the global expansion of Galafold and begin the global launch of Pombiliti and Opfolda . Importantly, based on the latest assumptions on approvals and launch of Pombiliti and Opfolda , combined with the strong growth we've seen in Galafold in the first half of the year, we are on track to achieve our target of non-GAAP profitability in the second half this year.
Ahead on Slide 4, we're making great progress across our key strategic priorities for 2023, including sustaining double-digit Galafold growth, now at an anticipated growth rate of 14%-18% at constant exchange rates; securing regulatory approvals of AT-GAA by the FDA, EMA, and MHRA and executing successful launches in those key markets; continuing to judiciously invest in the advancement of our best-in-class, next-generation Fabry and Pompe genetic medicines and capabilities, as well as our next-generation chaperone for Fabry disease; as always, maintaining a strong financial position as we carefully manage our expenses and investments on our path to non-GAAP profitability. With that overview, let me now hand the call over to Sébastien Martel, our Chief Business Officer, to further highlight our commercial performance. Sébastien?
Sébastien Martel (Chief Business Officer)
Thank you, Bradley, and good morning to everyone on the call. I'll start by providing you with more detail on our Galafold performance for the quarter. On Slide 6, for the second quarter 2023, Galafold reported revenue reached $94.3 million, driven by strong patient accruals, particularly in the U.S., but also in key European markets and Japan. Geographic breakdown of revenue during the quarter consisted of $57 million, or 61% of revenue, generated outside of the U.S., and the remaining $37 million, or 39%, coming from within the U.S., We're very pleased to see continued patient growth in countries across our leading markets. Turning on to Slide 7, our results in the first half of the year highlighted the strengths of our global commercial efforts.
The demand for Galafold globally continues to be strong, with patients added in all major markets, delivering a operational growth rate of 16% over the same period in 2022 at constant exchange rates. From a year-over-year perspective, the negative impact from foreign currencies was 3% in the period. As a result, Galafold reported revenue growth was 13%. Galafold continues to be the fastest-growing treatment for Fabry disease globally. I'm pleased to report that our monthly net new patient trends continued to increase in Q2. If you look at the growth in net patients on Galafold globally, we've seen significant net patient gains on Galafold at the end of the second quarter.
Throughout the first half of 2023, the number of patients coming onto Galafold has actually exceeded internal expectations. We're therefore raising our full year 2023 revenue growth guidance to 14%-18% growth at constant exchange rates. We ended the second quarter with about 60% of the global market share of treated amenable patients. Within the global mix, we're seeing stronger uptake in naive populations. While we're achieving high market shares in countries where we've been approved the longest, there's also plenty of opportunity to continue to switch patients over to Galafold and continue to grow the market as we penetrate into the diagnosed and treated and newly diagnosed segments. All of that is underpinned by impressive compliance and adherence rates that continue to exceed 90%, reiterating our belief that those patients who go on Galafold predominantly stay on Galafold.
As mentioned on past calls, due to a variety of factors, including uneven ordering patterns and effects fluctuations, the rate of growth within the year is typically nonlinear. We expect that to continue throughout 2023. To support quarterly forecasting, we provide a table showing distribution of Galafold revenue by quarter in the past five years in the appendix section of our IR presentation. On Slide 8, we know that there's significant patient demand for Galafold and that the segment of the global Fabry market made of those amenable mutations, has the potential to surpass $1 billion in annual revenue in around 5 years. We anticipate sustained growth throughout 2023 to be driven by several key growth drivers. First, continuing to penetrate into the existing markets, further uptake into the diagnosed and treated population, and expanding into new geographies and label extensions.
All of these efforts are supported by positive reimbursement and access mechanisms around the world. I'm pleased to share that we continue to make progress on expanding Galafold into new markets and extending the labels. Just to name a few examples, we recently received reimbursement in Taiwan for individuals over the age of 16 year old. The Fabry market within Taiwan is quite sizable, with over 350 individuals diagnosed with Fabry disease. Of note, Galafold was designated the first inline therapy for all amenable patients living with Fabry disease in Taiwan. Additionally, we've submitted a marketing authorization application in New Zealand, and we've entered into pricing and reimbursement negotiations for Galafold in Turkey.
In the longer term, we continue to see significant growth in the Fabry market globally, driven by diagnosing patients through a variety of measures, including high-risk screening, newborn screening, and other diagnostics initiatives, which we continue to support and invest in as well. With that, let me now hand the call over to Dr. Jeff Castelli, our Chief Development Officer, to highlight the regulatory updates on our AT-GAA program. Jeff?
Jeff Castelli (Chief Development Officer)
Thank you, Sébastien. Good morning, everyone. Starting on Slide 10, we remind everyone that late-onset Pompe is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders. Within LOPD, individuals may experience impaired motor function and respiratory difficulties as the disease progresses, with these potentially debilitating symptoms tending to become more serious and problematic over time, and often respiratory failure being a major cause of mortality in LOPD. Multiple publications and natural history studies continue to show that the initial benefits of treatment are often followed by continued long-term decline for many individuals. We recognize that Pompe disease continues to pose a range of health challenges for people affected by the disease, and having therapeutic options is crucial. Moving on to Slide 11, we briefly outline the current regulatory status of AT-GAA by key markets.
First, as noted, in June, Pombiliti and Opfolda , both were approved in the EU, and the commercial launch is underway, as Sébastien will detail in more in the next section. In the U.S., as Bradley noted, we're pleased to see the increased level of engagement from the agency in recent weeks and remain confident we are on track for approval in the third quarter for both components of AT-GAA. Finally, just this morning, we were very excited to receive the final full approval and orphan designations for both Pombiliti and Opfolda by MHRA, and now the team can begin launching this important new therapy for people living in the UK with Pompe disease, leveraging that positive reimbursement appraisal from NICE.
Moving on to Slide 12, we remind everyone of our ongoing clinical studies and multiple mechanisms of expanded access that support much of the early demand for AT-GAA. For the younger Pompe community, we continue to enroll the ongoing open label ZIP study in children up to 18 years of age, living with LOPD, and have begun enrolling the open label ROSELLA study for children living with infantile-onset Pompe disease. We have multiple expanded access programs in place in countries where we are not yet approved, and interest and momentum for AT-GAA continues to grow, and we're pleased to be able to provide access to those who are eligible through those programs.
As a reminder, at the time of our first approvals, which was, you know, first in the EU during that last week of June, there were approximately 200 patients worldwide being treated with AT-GAA across the clinical extension studies and expanded access programs. Importantly, experience with AT-GAA is quite broad, with approximately 75 centers participating in trials and various access programs around the world. Finally, as highlighted in the pipeline slide in the appendix, for our earlier stage pipeline, we continue to focus on novel approaches for Fabry and Pompe, including gene therapies to deliver our engineered GLA and GAA transgenes and the next generation Fabry chaperone program. With that, I'll turn the call back over to Sébastien to discuss the early commercial launch progress of Pombiliti and Opfolda . Sébastien?
Sébastien Martel (Chief Business Officer)
Thank you, Jeff. On Slide 13, we outline the initial launch progress and market opportunity for Pombiliti Opfolda in the EU. Pombiliti plus Opfolda is approved as the first and only two-component therapy available in the EU for the treatment of adults living with late-onset Pompe disease. We're very pleased with the strong indication for Pombiliti plus Opfolda , which addresses all adults living with LOPD. The EU market represents a sizable opportunity of $450 million plus, and is a key market to support these early stages of global launch. Within Europe, there are currently around 1,300 patients estimated to be on treatment, and of that 1,300, currently 60 patients are being treated with Pombiliti plus Opfolda , and around 20 of those in Germany and Austria.
As of today, we are launched in Germany, with progress being made on all fronts, and we're seeing the start of a very strong uptake. Importantly, in Germany, the Federal Joint Committee, or GBA, has classified Pombiliti as a new active substance. As a result, Pombiliti will undergo a benefit assessment under the AMNOG process with a free pricing period, after which there'll be negotiations on the final reimbursed price. Based on feedback from key treaters through our clinical development, access and medical affairs, we're very pleased with the progress made towards transitioning patients over to commercial supply. We've been successful in engaging with the top prescribers in Germany within the first 30 days. We've also been very pleased with the positive feedback from the expanded access program, CUP, from both a physician and patient perspective.
As Bradley mentioned, the first patients have been recently moved to commercial supply, and more are scheduled in the coming weeks. We're well on the way to achieving our goal of converting all of the 20 or so expanded access patients and clinical trial extension patients within the first 90 days of launch. On Slide 14, as you know, we've been preparing for the commercial launch of Pombiliti plus Opfolda , and are both excited and confident that through our world-class commercial and medical organizations, we're in a very strong position for a second successful launch at Amicus. In these early days, we're leveraging this experienced team to transition the first group of clinical trial and expanded access program patients in the EU onto commercial supply.
Given the significant overlap of treatment centers, hospitals, and physicians between Fabry and Pompe disease, we have the existing relationships with KOLs and the commercial infrastructure necessary to support a seamless transition. As of today, we've had the first patients infused, and multiple additional patients are scheduled to start their infusions. Within these early days, 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 interactions with payers to demonstrate the value of Pombiliti plus Opfolda . Today, we're launched in Germany. We're also in active pricing and reimbursement discussions with additional European countries as we focus on securing broad patient access to the EU.
We're working in partnership with physicians to ensure they have all the information they need on Pombiliti plus Opfolda for their patients and believe this two-component therapy is poised to have a significant commercial opportunity in the EU and additional markets as they come online. As Jeff indicated, from the late-breaking news, we're thrilled with today's approval of MHRA approval for Opfolda . We also were granted orphan drug designation for Opfolda , like we did with Pombiliti in the UK. The market opportunity within the UK includes more than 200 people with Pompe disease estimated to be on treatment. 45 of these 200 individuals are already on Pombiliti plus Opfolda , and that's a remarkable number, either through a clinical trial or through the Early Access to Medicines Scheme. As we've mentioned previously, all leading centers across the UK have requested access to Pombiliti plus Opfolda through EAMS.
Now that approval has been granted, our team anticipates a transition of all patients in the UK onto commercial product within 90 days. Importantly, that transition will be supported by the very positive NICE recommendation for reimbursement of Pombiliti plus Opfolda within the NHS, which is an achievement we are incredibly proud of. As stated in the guidance, NICE concluded that the cost-effectiveness estimates for Pombiliti plus Opfolda showed a positive net health benefit, and therefore, has recommended Pombiliti plus Opfolda for adults with LOPD as first line and later lines of therapy. With that, I'd like to now turn the call over to Daphne Quimi, our Chief Financial Officer, to review our financial results, guidance, and outlook. Daphne?
Daphne Quimi (CFO)
Thank you, Sebastien, good morning or afternoon, everyone. Our financial overview begins on Slide 16 with our income statement for the second quarter ending 30 June 2023. For the second quarter, we achieved total revenue of $94.5 million, which is a 17% increase over the same prior year period in 2022. This includes year-over-year operational revenue growth measured at constant currency exchange rates of 17% and a negligible currency impact at 0%. Cost of goods sold as a percentage of net sales was 9.6%, as compared to 10.2% for the prior year period. Total GAAP operating expenses decreased to $104.2 million for the second quarter of 2023, as compared to $133.1 million in the second quarter of 2022.
On a non-GAAP basis, total operating expenses decreased to $84 million for the second quarter of 2023, as compared to $119.2 million in the second quarter of 2022, primarily reflecting decreased program spend. We define non-GAAP operating expenses as research and development and SG&A expenses, excluding share-based compensation expense, loss on impairment of assets, changes in fair value of contingent consideration, and depreciation. Within other expenses in the income statement in the second quarter, we recorded approximately $11 million in unrealized foreign exchange losses. This compares to a $7 million gain in the second quarter of 2022. We are currently taking steps to mitigate our future exposure.
Net loss for the second quarter of 2023 was $43.2 million, or $0.15 per share, as compared to a net loss of $62.2 million, or $0.21 per share for the prior year period. Driven by the revenue growth of Galafold and careful expense management, we continue to make progress towards our path to non-GAAP profitability in the second half of this year. As of 30 June 2023, we had approximately 287 million shares outstanding. Turning now to Slide 17. With a focus on achieving non-GAAP profitability, I am pleased to share that we have revised both our revenue and operating expense guidance for the year. We are raising full-year Galafold revenue growth guidance to 14%-18% at constant exchange rates, driven by patient demand.
We are also reducing our full year 2023 non-GAAP operating expense guidance to $330 million-$350 million. The decrease in operating expense for 2023, as compared to 2022, will be achieved by continuing to drive efficiencies and prudent expense management, offset by continued investment in Galafold, AT-GAA clinical studies, non-recurring costs for manufacturing, as well as global launch activities. We anticipate operating expenses to be nonlinear this year due to these pre-launch and launch expenses. We also expect to see a larger portion of our operating expenses allocated to G&A this year, as we align our resources to support the launch of AT-GAA and the continued growth of Galafold.
Cash, cash equivalents, and marketable securities were $265.6 million as of 30 June 2023, compared to $293.6 million as of 31 December 2022. With that, let me turn the call back over to Bradley for our closing remarks.
Bradley Campbell (President and CEO)
Great. Thanks, Daphne, Jeff, Sébastien. As you can see, we've been relentlessly focused on execution across our global business, and we look forward to embarking on this next phase of Amicus as a company with two commercial therapies. Thank you to all of our employees who have enabled us to achieve these recent corporate milestones and who are always committed to our patient-focused mission of delivering life-changing therapies to people in need. Before I hand the call over to Q&A, as you may have also seen in this morning's filing, after 15 incredible years at Amicus, Daphne, our longtime chief financial officer, leader, and friend, has decided to retire. She'll remain in her current role as CFO until we appoint her successor, at which point she'll remain on with Amicus through the end of the year in order to support a smooth transition.
A search is underway. We've already identified a number of highly qualified candidates. I also just want to add that I'm personally grateful to you, Daphne, for your years of exemplary service here at Amicus, and in particular, for helping lead us on our course to achieve non-GAAP profitability later this year. On behalf of the Amicus Board of Directors and the entire executive team, I'd like to take this opportunity to formally thank you. You've been a significant part of the growth at Amicus and instrumental in guiding us to the sound financial position we're in today. With that, operator, we can now open up the call to questions.
Operator (participant)
Certainly. Ladies and gentlemen, if you do have a question, please press star one one on your touch-tone telephone.At this time, we request that you only ask one question. If you have any additional questions, please enter back into the queue. Thank you. One moment for our first question. Our first question comes from Tazeen Ahmad of Bank of America. Your line is open.
Tazeen Ahmad (Managing Director, Equity Research Analyst)
Hi, guys. Good morning, and thanks for taking my question. Brad, just wanted to clarify on the timeline that you've stated forward for GAA is still expecting approval in 3Q. Do you get a sense that the responses that you gave FDA post the Wuxi inspection facility have been sufficient for the agency? If not, what do you think are the remaining gating factors regarding questions around the facility in order to get approval? Thanks.
Bradley Campbell (President and CEO)
Thanks, Tazeen. Yeah, great question. Just as a reminder, we were very pleased with the outcome of the pre-approval inspection at Wuxi. We do feel like our responses were all adequate, and the color we provided on the call here today, I think is important, which is in the last few weeks, we've seen an increased level of engagement from the agency. So that gives us great confidence that we're on track for a Q3 approval.
Operator (participant)
One moment for our next question. Our next question will come from Anupam Rama of JPMorgan. Your line is open.
Anupam Rama (Analyst)
Hey, guys. Thanks so much for taking the question. Congrats on the progress, and Daphne, I'll miss you at the conference this coming year. On the Galafold guidance increase, maybe from a regional perspective, where is that coming from? Is it coming from deeper penetration in core countries or progress in some of the new emerging countries?
Bradley Campbell (President and CEO)
Yeah, I'm happy to have Sébastien add a little bit of color, but honestly, Anupam, it's coming from all of those things. Most importantly, we're seeing is across all of our key geographies, as we mentioned on the call, rates of net new patient starts at levels that we haven't seen since the first couple of years of launch, and so clearly demand for this product is growing. Sébastien, maybe touch on a few of the different growth drivers that Anupam highlighted to give a little bit more color there.
Sébastien Martel (Chief Business Officer)
Yeah, I'd just add, you know, for example, in the U.S. alone, remember we launched in 2018, the current rate of net new patients that we've seen in the second quarter is as strong as what we saw in 2019. We're really in a, you know, post-COVID world, as we speak, and we're seeing, you know, strong, strong patient accruals. This is also, you know, the case in Europe. Remember, in Europe, we were launched two years earlier, so our penetration rate within the amenable population is, you know, slightly greater, and yet we're seeing very strong demand. A lot of naive patients actually go on Galafold when they're diagnosed.
We've essentially established Galafold as the, you know, new first-in-line treatment for Fabry patients with amenable mutations in those markets.
Bradley Campbell (President and CEO)
Yeah, maybe Anupam, the other things I'd highlight. Sorry, I didn't mean to cut you off.
I was just gonna say, the other thing I'd highlight is, Sébastien mentioned it on the call, you know, not only are we seeing great penetration rates, we're opening up new geographies, we have some important countries coming on board this year. It's also in the backdrop of a very healthy and growing Fabry market, diagnosis continues to be strong. As we find those patients, as a reminder, typically you find one undiagnosed Fabry patient or newly diagnosed Fabry patient, and because it's an X-linked disease, that typically leads to four to five additional family members who are diagnosed. In addition to, I think, executing incredibly well, which we've continued to do, we're also seeing just a healthy growth of market. As Sébastien highlighted, we're actually the fastest growing of the Fabry treatments for patients with amenable mutations.
Anupam Rama (Analyst)
Thanks for taking our questions.
Operator (participant)
One moment for our next question. Our next question will come from Ritu Baral of TD Cowen. Your line's open.
Ritu Baral (Managing Director and Senior Biotechnology Analyst)
Good morning, guys. Thanks for taking the question. I want to go back to the Wuxi inspection and follow up on Tazeen, Tazeen's question. Brad, can you give any additional detail on the points of the increased level of engagement that the agency has had? Just wrapping all of this up, do you think that there's any possibility of requiring a re-inspection, or do you feel that the outstanding items are addressable over, you know, written, written or verbal interactions? Any additional detail would be helpful.
Bradley Campbell (President and CEO)
Yeah. Thanks, Tazeen. No, we do not believe that there'll be any additional inspections needed at Wuxi. We are very confident, and we're very pleased with the outcome of the inspection. Again, just based on the kind of level of re-engagement we've seen, increased level of engagement we've seen in the last couple weeks, it feels to us, as you said, that they're, you know, wrapping things up and we feel like we're getting close to an approval here, as we suggested, you know, on track for Q3. We're really excited. The team is ready. We're really pleased to see the UK news this morning, so I think the momentum is building, and really eager to see the remaining months of the year play out.
Ritu Baral (Managing Director and Senior Biotechnology Analyst)
Got it. If I can squeeze one more in.
Bradley Campbell (President and CEO)
Sure.
Ritu Baral (Managing Director and Senior Biotechnology Analyst)
About how much supply if, how much supply from the open label are German and UK patients sort of sitting with before they start on the path to commercial conversion?
Bradley Campbell (President and CEO)
Yeah, we have, our target is about two years of go-forward supply. Remember, we've made quite a bit of-
Ritu Baral (Managing Director and Senior Biotechnology Analyst)
Oh, I'm sorry. I meant, I meant like, how much supply clinic, how much drug supply do they have sitting at home from the open label?
Bradley Campbell (President and CEO)
Oh, that they're working through, right.
Ritu Baral (Managing Director and Senior Biotechnology Analyst)
Yep.
Bradley Campbell (President and CEO)
Right. Got it.
Ritu Baral (Managing Director and Senior Biotechnology Analyst)
Yes.
Bradley Campbell (President and CEO)
Sorry. Thanks for the clarity. Yeah, the good news is, unlike Galafold, where, you know, you would typically, because it, it was, you know, long shelf life, et cetera, you know, you could, you could be sitting on kind of three months of supply of Galafold as we were winding down the clinical studies and transitioning over to, to commercial. In this case, it's a much tighter supply situation. The clinical operations team at Amicus, who, of course, has already done this once before with Galafold, works very closely with the medical affairs team, with the sites themselves, and so it's a really fine and precise level of supply. That's what allows us to very carefully manage and have that goal of 90-day transition.
In Germany, so far, as I've said, you know, even in the first, you know, kind of 30 days of launch now, we are making those transitions, and we're confident we'll be able to execute that within the first 90 days, and I think likewise in the UK and in the U.S. when we see the approval.
Ritu Baral (Managing Director and Senior Biotechnology Analyst)
Great. Thanks.
Bradley Campbell (President and CEO)
Yep.
Operator (participant)
One moment for our next question. Our next question will be coming from Eliana Merle of UBS. Your line is open.
Eliana Merle (Executive Director and Biotech Equity Research)
Hey, guys. Thanks so much for taking the questions. Just if you could provide us a little bit more color on how you're thinking about new patient starts in Germany and the UK beyond the patients that are on expanded access. Maybe just of the 200, I think you said Pompe patients total in the UK, and I think you said 45 were already on AT-GAA. How should we think about the other 150 patients? Maybe what proportion might be on Nexviazyme already, and just the timelines for when you expect the NICE reimbursement to kick in from a revenue perspective?
Then in Germany, I know you mentioned, you have 20 patients already, on AT-GAA, but just, if you could give us a sense of maybe how many Pompe patients there are in Germany, and how you think about the landscape there, relative to next year's time. Thanks.
Bradley Campbell (President and CEO)
Sure. I'll take some of those, and then I'll ask Sébastien Martel to add some color as well. That was a cleverly worded one question, multi-question prompts, happy to address all of those. First of all, yeah, the first priority, as we've said in each of those markets, because we have such a large bolus of patients, is to convert the, the expanded access in clinical trial patients. Of course, in parallel, we're also working to start to begin bringing on new patients as well. We should be able to provide more color on that in the November call, again, first priority, low-hanging fruit, patients on AT-GAA already, and then in parallel, start to bring on new patient starts as well.
Maybe, Sébastien, just give the color that we know based on, you know, publicly available information, what progress has been with Nexviazyme in those two markets, and a sense for how large the German market is outside the 20 patients already on AT-GAA?
Sébastien Martel (Chief Business Officer)
Yes. Thanks, Bradley. The, the, you know, I can share with you the, the breakdown between Nexviazyme and Myozyme, across Europe. It's, you know, virtually 80%, 20% or, or 77%, 23%, to be precise. The vast majority of, patients are still on Myozyme, again, 77% across Europe and, 23% for Nexviazyme. If you look at the, the UK numbers, you know, you have them, you, you just, quoted them, Ellie. 200 patients total, 45, which is probably the largest, number of patients we have in, you know, in a country on, on clinical trial, plus, early access.
As Brad mentioned, you know, we think that given the fact that the 6 key centers have all had experience with Pombiliti and Opfolda , they will have patients transition, you know, from either clinical study or OLEs. They have experience. They've used the product before. They have, obviously, the, the, the feedback also from patients who have been on it. We think that, you know, the uptake and our ability to convert commercial patients from Myozyme or Nexviazyme is high in this market. In Germany, the market is a lot more fragmented than the UK. The number of patients is also higher, somewhere between 300-350 total patients.
We did mention here that we've got, around 20, in Germany and Austria, so those, you know, will be, you know, the first patients onto commercial product. As I mentioned, our team, you know, has been, visiting top, prescribers in Germany, for a number of weeks now and, have established, you know, those, those contacts and will be working with those, physicians to convert and switch patients from their existing treatment onto Pombiliti plus Opfolda over the next weeks and months.
Bradley Campbell (President and CEO)
I think you had maybe one more question, Ellie, on the, on the, the reimbursement process in the UK. As I mentioned in the call, once you have MHRA approval, what's remarkable here is we've already had the initial recommendation for reimbursement, by NICE, which we think is one of the fastest ever, to get to that answer prior to MHRA approval. The process from here should take a few days, to a week to get to the final, appraisal document by, by NICE. The draft is already posted, so that should come here relatively shortly. Then it takes around 30 days for NHS to provide funding to the local centers, and that's when we'll start, actually converting those, expanded access and clinical trial patients.
In the meantime, with the approval, our sales team can go out into the field, visit the physicians, and again, along with the medical affairs and clinical operations team, can begin scheduling those infusions ahead of time. Effectively, launch is underway, and we're waiting about 30 days for that reimbursement process to kick in at the local centers.
Eliana Merle (Executive Director and Biotech Equity Research)
Great. Thanks, really helpful.
Bradley Campbell (President and CEO)
Thanks, Ellie.
Operator (participant)
One moment for our next question. Our next question will be coming from Joseph Schwartz of Leerink Partners. Your line is open.
Joseph Schwartz (Managing Director and Senior Biotechnology Analyst)
Great, thanks very much for the update. I was wondering, beyond the EAP conversion to, of, of those patients to commercial status, how should we be thinking about the cadence of uptake for AT-GAA? What metrics will you be providing us in order to gauge your progress with that? Are there any analogs that you can think of that might be helpful for us when envisioning the launch curve? Can you remind us of your most recent thoughts around pricing and how this relates to the current price of Sanofi's products? Thank you.
Bradley Campbell (President and CEO)
Yeah, thanks, Joe. All good questions. I'll start with the last one first. You know, as a reminder, our pricing philosophy is parity or modest discount to standard of care, and we have shown with Galafold that that is an incredibly successful strategy. We went through the pricing and reimbursement process with Galafold much faster than industry average, and I think it's because, you know, the healthcare systems are recognizing that we're bringing significant value, but we're pricing at roughly parity, which, you know, takes the pricing decision off the table and focuses on the value of the product. What we saw is that way you can maximize access to therapy and maximize the number of patients on therapy as quickly as possible, versus, you know, taking time to negotiate for some extra dollars on the price.
I think the NICE appraisal that, that we saw here, which, again, I think is one of the fastest ever in our industry, is a great reflection of that value that the payers are seeing. We're confident that strategy will be very successful here, and we'll roll that out as we go through the process in Europe and the U.S. and, and other markets. On the first one, in terms of, in terms of key performance indicators that were shared, I think what you heard on the call today is, is some of the qualitative things we'll talk about in terms of getting out to see the key centers, in terms of having patients start on infusions and convert from clinical trial to, to commercial product.
We haven't given any forward-looking guidance for this year just because it's such a, you know, stub year. But I think you'll hear on the November call that we'll provide an update on the number of commercial patients on drugs, so that'll be a NICE milestone to wait for. In terms of kind of, you know, analogs, it's so unusual to have a second-generation product launched into the rare disease space. I don't know if there are great analogs. The one thing I would point to, though, is kind of a, you know, a thought experiment perhaps, is remember, we qualified for that EAMS program in the UK about a year ago. And within the first year, remember that you can't promote on that product, it's simply offering it, and it's purely demand-driven.
We were able to get to a 15%-20% market share through that program alone, again, with no promotion. For me, that gives you a great perspective on, on, you know, imagine what we can do once we're out there promoting the product. We'll do our best to give good, good color on how the launch is going, and, and hopefully, that gives you a flavor of how we can talk to that over the coming, you know, days and weeks. Again, November, I think, will be a really important update there.
Joseph Schwartz (Managing Director and Senior Biotechnology Analyst)
Thank you.
Operator (participant)
One moment for our next question. Our next question will be coming from Daegan Ha of Stifel. Your line is open.
Dae Gon Ha (Director and Biotechnology Equity Research)
Hey, good morning, guys. Thanks for taking the question. One clarification: the slide says first patients, as in plural, has been infused with LOPD, I guess, AT-GAA product. Just wanted to clarify that it was more than one.
Bradley Campbell (President and CEO)
Right.
Dae Gon Ha (Director and Biotechnology Equity Research)
Then, my actual question was, the physician feedback and engagement, what are you guys hearing in terms of the lineup that physicians will take once Nexviazyme, I guess, AT-GAA becomes available as a third line? I mean, does it get kind of placed as a third line, or do you see it being used sort of in the in-between Myozyme, Nexviazyme, or Nexviazyme onto AT-GAA? Any color on that would be helpful. Thanks.
Bradley Campbell (President and CEO)
Yeah, thanks. First and foremost, the, the feedback we've received has been incredibly positive from, from physicians, and we're hearing through them that the, that the experience from patients has also been very positive, so we feel like everything is going really well. Yes, to clarify that your first question, it is multiple patients have been converted, which is great, and multiple patients have been scheduled for their infusions, and we're working to, to schedule the remaining conversions within that 90-day target. Really, really good progress as we've gotten started here. Then, sorry, remind me the second part of your question. Oh, in terms of positioning. Yeah, so reminder, in Europe, the indication statement is for all patients, late-onset Pompe disease patients.
We'll position this, and we think this will be a great treatment option for all of those patients. Remember, though, that the, the vast, vast majority of the market opportunity here is patients currently on Myozyme. You know, you should see, just like we saw with Galafold, you should see the vast majority of growth in all of our markets coming from, you know, patients who we switch from Myozyme or Nexviazyme, frankly, to AT-GAA. I think that's an important point, right? Which is the indication is for all patients with LOPD in Europe who are on label, and that means that we can target any patient on therapy or frankly, any, any new patients that, that's diagnosed or has already been diagnosed.
Sébastien Martel (Chief Business Officer)
Just to add to what you were saying, Brad, I think to give a little bit of color on the global opportunity from a patient standpoint, there are 3,500 to 4,000 patients being treated worldwide for Pompe disease today. We estimate that depending on the year, you've got somewhere between 100 to 200 naive patients put on treatment every year. The commercial opportunity as of this point, is really looking to switch patients, whether they are on Myozyme or whether they are on Nexviazyme. This is the, you know, the main patient group that we're gonna go after.
Bradley Campbell (President and CEO)
The last thing I'll say there is, remember, you know, the real promise of this medicine is that we're the only product in a well-controlled clinical study that shows that patients who are on an existing ERT can switch to AT-GAA and see improvement in 6-minute walk and forced vital capacity. We think that's a truly differentiated component of this therapy, and we're just so pleased and eager to get this out to as many patients as possible, as quickly as possible.
Dae Gon Ha (Director and Biotechnology Equity Research)
Great. Thanks for the color, and Daphne, thanks so much for your service.
Operator (participant)
One moment for our next.
Sébastien Martel (Chief Business Officer)
Thanks, bye guys.
Operator (participant)
Our next question will be coming from Jeff Hung of Morgan Stanley. Jeff, your line is open.
Jeff Hung (Equity Research Analyst of Biotechnology)
Congratulations on the MHRA approval of Opfolda , and congrats to Daphne on her upcoming retirement. You've reduced expenses to support your path to non-GAAP profitability. Can you just talk about the importance of that relative to further bolstering your pipeline and advancing your next gen pipeline programs? Thanks.
Bradley Campbell (President and CEO)
Yeah, you know, the vast majority of our, our expenses go towards both the ongoing clinical studies to support the, you know, post-marketing commitments and, and remaining clinical studies extensions in Pompe disease, also manufacturing until recently, when now we have a commercial product, that ends up showing up in cost of goods. That's the vast majority, and then, of course, the G&A that goes along with that. We do have some modest spend towards the pipeline, and we are making, I think, good progress there. I think you should expect to hear us talk more about the pipeline as we come into next year. You should know that this laser focus on non-GAAP profitability does include a modest amount of investment in the pipeline.
That's an important part of the future of Amicus, again, we'll talk more about that, you know, probably coming into next year.
Jeff Hung (Equity Research Analyst of Biotechnology)
Thank you.
Operator (participant)
One moment for our next question. Our next question will be coming from Salveen Richter of Goldman Sachs. Your line is open.
Speaker 16
Hi, this is Srinathra on for Salveen. Thank you for taking our question. First on Galafold, in your view, how much of the growth that you need to get to your prior guidance of $1 billion peak sales for Galafold will be driven by new patient diagnoses, like genetic screen, like genetic screening, et cetera, versus the naive patients who are already diagnosed and switched patients? On AT-GAA, if you could provide some more color on the reimbursement process in Germany. Thank you.
Bradley Campbell (President and CEO)
Yeah. A couple of points there. If you think about just the market today for Fabry, the global market, you've got a little over $2 billion in global sales. You know, here we are sitting at, you know, with guidance of kind of $375 million-$388 million. If you assume a third to half of the patients on drug today have a Mendelian mutation, you know, you've got another maybe doubling of the current revenue base just by existing diagnosed and treated patients today. Lots of opportunity to continue to switch patients, which is great. Some of that comes through market expansion, and some of that comes through execution in our existing markets.
We also know if you look kind of globally, the global, that, that sort of over $2 billion in global sales represents about 11,000 treated patients, and we believe there's roughly another 5,000 or 6,000 diagnosed, untreated patients. So again, if you take that kind of third to a half number, that's, you know, an additional, call it, 3,000 or so, diagnosed, untreated patients who have a Mendelian mutation. Another significant chunk of existing patients today. I think that gets you sort of well towards that, that, you know, peak market opportunity that we've talked about. Then, as Sébastien and Jeff have highlighted in previous calls, we're seeing, you know, significant growth in the underlying diagnosis, maybe 10% or so a year.
You put all those together, and I think it's easy to see a way towards, you know, a billion-dollar product opportunity here. Then you had asked a little bit about Germany. Maybe, Sébastien, do you want to talk to the, the dynamics in, in Germany again?
Sébastien Martel (Chief Business Officer)
Thanks, Bradley. You know, as I mentioned, in Germany, the GBA has classified Pombiliti as a new active substance. We're very pleased with that. And as a result of that designation, Pombiliti will undergo benefit assessment under the AMNOG process. We have, you know, a six month free pricing period, and after that, there'll be negotiations on a final reimbursed price. You should expect to see an, an agreed-upon published price, sometimes around Q3 of 2024. We, we think we've set, you know, a competitive price for the combination of Pombiliti plus Opfolda versus other Pompe therapies available in the German market. And the price is, again, very much in line with the pricing policy that Bradley mentioned early on.
Operator (participant)
One moment for our next question. Our next question will be coming from Kristen Kluska of Cantor Fitzgerald. Your line is open.
Kristen Kluska (Equity Research Analyst)
Hi, good morning, and congrats on the additional approval this morning, and also sending my best to Daphne. I know it's early days in Europe, can you talk about what have been the biggest selling points in terms of generating interest over other therapies now that you have an approval in place beyond the prep work? For example, is it the mechanism, the full data set across the different patient populations, open label, a certain endpoint that they're focusing on, or is it really just a combination of this? Thank you.
Bradley Campbell (President and CEO)
I'll hit the top. Sorry about that. I'll hit the top line, then maybe Jeff talk about the, the broader data that we've showed in, in previous calls. You know, from a label perspective, clearly the most important two data points are the six minute walk test and the forced vital capacity, which, as I mentioned before, we've shown significant differentiation. We're the only therapy that's shown improvement in those measures in patients who've switched from existing ERT to AT-GAA. I think that's a really powerful story and, and one that physicians will clearly focus on. I do think another important part of this is the really broad data set that we've published now in various places.
It won't necessarily show up in the label, but as we continue our medical education strategy, we'll be able to share those. Maybe, Jeff Castelli, talk about what we've already published across those other endpoints and any other color you might want to provide.
Jeff Castelli (Chief Development Officer)
Yeah. Thanks, Brad, and thanks, Kristen, for the question. You know, as Brad noted, you know, the key there is, is showing the improvement on 6-minute walk and FVC in the switch patients in a controlled trial. You know, it's really that consistency across endpoints. You know, we've shown some of that previously at a high level. We're gonna continue to really focus on that here, in upcoming conferences, just to show, you know, the, the range of improvements that we're seeing across endpoints, for these patients. And I'd say the other thing is, is the emerging durability data as well. You know, we've now shown out to 4 years from our phase I/II, that the initial improvements observed across naive patients, switch patients in those trials have been maintained, long term.
We think that that's gonna be an, a continued focus of physicians and, and a strength so far of what we've seen with, with AT-GAA. You know, what's really strong core of data, you know, I think the MOA is also something that, you know, gives people reason to believe in some of those clinical observations, that there is a very differentiated MOA, and that's also something that I think we're gonna continue to, you know, provide publications and more information there as we continue to learn about the, the MOA.
Operator (participant)
One moment for our next question. Our next question will be coming from Ethan Markowski of Needham & Company. Your line is open.
Ethan Markowski (Equity Research Associate)
Good morning, and thanks for taking our question. Just a quick one on the current SG&A spend. You're currently relying somewhat on, on the existing sales force for, for Galafold, but we've definitely seen an uptick in SG&A. Just to give us an idea, now that you're rolling out, Pombiliti and looking at a U.S. launch, how, how much more of the SG&A spend increase should we expect in the near term? Thank you.
Bradley Campbell (President and CEO)
Daphne, I'll let you talk to the accounting treatment that, that I think is describing a lot of that change. From a infrastructure perspective, what we've said before is we've literally have hired less than a dozen employees globally to support the launch of AT-GAA. That includes some Amicus Assist, which is our, our case management hub in the United States, some patient education liaisons, some direct marketing and medical affairs people, but, but hugely leveragable existing commercial infrastructure. Maybe, Daphne, talk to the, the trends that the question was speaking to.
Daphne Quimi (CFO)
Yeah, sure. I would remind everyone, in prior years, we had a portion of the support for both Galafold and now AT-GAA in the research and development line. Now, as we are aligning resources to support two commercial products, some of that spend is now shifting from the R&D line to the SG&A line. It is a bit of a classification issue when you look at current year versus prior year.
Bradley Campbell (President and CEO)
... Yeah, it's not actually-
Daphne Quimi (CFO)
Versus a true increase, let's put it that way.
Bradley Campbell (President and CEO)
Exactly.
Daphne Quimi (CFO)
Mm-hmm.
Bradley Campbell (President and CEO)
Yeah. Thanks, Daphne. You should not expect to see significant additional increases in G&A. It was really more of a change in the classification for some of those employees who support the global business.
Ethan Markowski (Equity Research Associate)
Thanks, that, that was very helpful.
Operator (participant)
Thank you. One moment for our next question. Our next question will be coming from, Tazeen Ahmad of Bank of America. Your line is open.
Tazeen Ahmad (Managing Director, Equity Research Analyst)
Hi, guys. Thanks for taking my follow-up. Brad, just to maybe put people's minds at ease, in terms of going back to the GAA question about your timeline for 3Q, just because the PDUFA has officially passed, how are you getting your confidence that they will, the FDA will provide an answer this quarter?
Bradley Campbell (President and CEO)
Yeah.
Tazeen Ahmad (Managing Director, Equity Research Analyst)
Also, as we head into the later part of August, I think the assumption is that the folks at FDA kind of shut down for holiday, and so it slips into September. Is there any- anything to read into that? Thanks.
Bradley Campbell (President and CEO)
Yeah, good, good questions. Thanks, Tazeen. We'll provide as much color as we can here. The confidence really comes from two places. The first is, if you remember back when we announced the inspection, we estimated that, you know, if you look on average, you know, even though we don't have a specific PDUFA date, on average, it takes about 30 days from the inspection to finish the inspection report, then it's about 60-90 days for the agency to complete their review, we're kind of right in that zone. We feel very comfortable that it's kind of in the timeline that we've seen when you, when you look on average.
Then the other piece that gives us good confidence, to your point about, you know, about whether the FDA is around or not, is we have seen in the last couple of weeks an increase in the engagement by the review division, and that gives us good comfort that they're, you know, wrapping up their work. Again, we expect a decision here in Q3, and we're very confident in that.
Tazeen Ahmad (Managing Director, Equity Research Analyst)
Okay, thanks, Brad.
Bradley Campbell (President and CEO)
Thanks, Tazeen.
Operator (participant)
That was our last question. I'd like to turn the call back to Bradley Campbell for closing remarks.
Bradley Campbell (President and CEO)
Great. Thank you, everybody, for tuning in today and hope everybody has a great day. Take care.
Operator (participant)
Ladies and gentlemen, this concludes today's conference. Thank you, and have a great day.