PHILADELPHIA, PA — Amicus Therapeutics (Nasdaq: FOLD) this week provided its preliminary and unaudited 2021 revenue, corporate updates, and full-year 2022 outlook and revenue guidance.
- Global revenue for Galafold® (migalastat) in 2021 reached $306 million driven by strong new patient accruals and sustained patient adherence, representing a year-over-year increase of 17%.
- AT-GAA regulatory reviews are underway: In the U.S., the Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat, the two components of AT-GAA. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA. In the EU, the Marketing Authorization Applications (MAA) were submitted and validated in the fourth quarter by the European Medicines Agency (EMA).
- AT-GAA launch preparations are accelerating: Development of global launch plans, targeted investments in additional personnel, and launch inventory are fully underway as company believes AT-GAA can rapidly become the new standard of care treatment regimen for people living with Pompe disease.
- Pipeline of next generation genetic medicines to advance through both internal efforts and creation of R&D focused new company, Caritas Therapeutics.
- Cash Flow and Balance Sheet sufficient to achieve self-sustainability and profitability in 2023. Through careful management of expenses, the Company is on the path to achieve self-sustainability and profitability in 2023 as it executes on the global Galafold expansion and prepares for AT-GAA global launch.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “In 2021, Amicus made great strides for people worldwide living with rare diseases through the broad execution of our annual strategic priorities. Despite the resurgence of COVID with Delta and Omicron variants, the Galafold business remains very strong, and we delivered on our full year revenue guidance and expect robust growth this year driven by strong adoption across the globe for our Fabry disease precision medicine. We are underway with the global regulatory reviews and launch preparations for AT-GAA in Pompe disease with high expectations that this novel medicine has the potential to become the new standard of care in Pompe disease treatment and the potential to address unmet needs for thousands of Pompe patients in the years ahead. We see further opportunity ahead to impact the lives of those living with rare disease through our genetic medicine business and capabilities. Together, Amicus is in a stronger position than ever and we remain steadfast on our mission of transforming the lives of people living with rare, life-threatening conditions and creating significant value for our shareholders.”
Bradley Campbell, President and Chief Operating Officer of Amicus Therapeutics, Inc., stated, “We are looking ahead to transforming Amicus into a leading global rare disease biotechnology company led by two innovative therapies that we believe meaningfully impact the lives of people living with Fabry and Pompe disease. This year we will be focused on continuing to bring Galafold to patients around the world and delivering on the anticipated approval and launch of AT-GAA.”
Amicus is focused on the following five key strategic priorities in 2022:
- Continued double-digit Galafold growth (15-20%) with revenue of $350M to $365M
- Secure FDA approval and positive CHMP opinion for AT-GAA
- Initiate successful, rapid launch in the U.S. for AT-GAA
- Advance best-in-class next generation genetic medicines and capabilities
- Maintain strong financial position on path to profitability
Mr. Crowley and Mr. Campbell will discuss the Amicus corporate objectives and key milestones in a presentation at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022, at 3:45 p.m. ET. A live webcast of the presentation can be accessed through the Investors section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/events.cfm, and will be archived for 90 days.
Full-Year 2021 Revenue Summary and 2022 Revenue Guidance
Global revenue for Galafold in full-year 2021 was approximately $306 million, preliminary and unaudited, representing a year-over-year increase of 17% from total revenue of $260.9 million in 2020. Full-year revenue benefited from a positive currency impact of approximately $7 million. Fourth quarter Galafold revenue was approximately $84 million, preliminary and unaudited.
For the full-year 2022, the Company anticipates total Galafold revenue of $350 million to $365 million. Double-digit revenue growth (15-20%) in 2022 is expected to be driven by continued underlying demand from both switch and naïve patients, geographic expansion, the continued diagnosis of new Fabry patients and commercial execution across all major markets, including the U.S., EU, U.K., and Japan.
The current cash position is sufficient to achieve self-sustainability and profitability in 2023.
Updates and Anticipated Milestones by Program
Galafold (migalastat) Oral Precision Medicine for Fabry Disease
- Sustain double-digit revenue growth in 2022 of $350 million to $365 million
- Continue geographic expansion
- Registry and other Phase 4 studies ongoing
AT-GAA for Pompe Disease
- U.S. Prescription Drug User Fee Act (PDUFA) action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA
- EU Committee for Medicinal Products for Human Use (CHMP) opinion expected in late 2022
- Continue to broaden access through early access plans in the U.K., Germany, Japan, and other countries
- Ongoing supportive studies, including pediatric and extension studies
Gene Therapy Pipeline
- Advance IND-enabling studies, manufacturing activities, and regulatory activities for the Fabry disease gene therapy program towards an anticipated IND in 2023
- Progress preclinical studies, manufacturing activities, and regulatory activities for the Pompe disease gene therapy program
- Discontinue CLN6 Batten disease gene therapy program following review of long-term extension study data. It was recently determined that any initial stabilization of disease progression at the two-year time point was not maintained through the long-term extension study. Amicus plans to further analyze and share the Phase 1/2 data with key stakeholders in the CLN6 Batten disease community and work with the community to support continued research efforts to find better treatments and cures which are so desperately and urgently needed
- Advance CLN3 Batten disease program with the higher dose, different promoter, and intra-cisterna magna (ICM) route of delivery pending further Phase 1/2 clinical data and pre-clinical data expected in 2022. These data will inform timeline for commencement of any pivotal clinical study
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