Amicus Therapeutics, a patient-dedicated global biotechnology company focused on rare diseases, has announced its financial results for the first quarter of 2025, showing continued revenue growth and an important expansion of its product portfolio through a strategic licensing agreement.
Q1 2025 Financial Performance
Amicus reported total revenues of $125.2 million for the first quarter of 2025, representing a 15% year-over-year increase at constant exchange rates (CER). This growth was driven by the company's two commercial products:
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Galafold (migalastat): Generated $104.2 million in net product sales, a 6% increase at CER compared to Q1 2024. While reported growth was 5%, the company noted strong underlying patient demand of 14%, partially offset by order timing and higher than anticipated rebates in the UK.
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Pombiliti + Opfolda: Contributed $21.0 million in net product sales, representing a substantial 92% year-over-year increase at CER. This growth reflects the ongoing global launch of this treatment for late-onset Pompe disease (LOPD).
The company reported a GAAP net loss of $21.7 million, or $0.07 per share, for Q1 2025, compared to a net loss of $48.4 million, or $0.16 per share, for the same period in 2024. On a non-GAAP basis, Amicus achieved net income of $9.0 million, or $0.03 per share, compared to a non-GAAP net loss of $4.6 million in Q1 2024.
Cash, cash equivalents, and marketable securities totaled $250.6 million as of March 31, 2025, a slight increase from $249.9 million at the end of 2024.
Strategic Expansion: DMX-200 Licensing Agreement
In a significant portfolio expansion, Amicus has entered into an exclusive U.S. licensing agreement with Dimerix for DMX-200, a first-in-class treatment in Phase 3 development for focal segmental glomerulosclerosis (FSGS), a rare and potentially fatal kidney disease with no currently approved therapies.
"This aligns perfectly with our strategy to leverage our rare disease commercial infrastructure and brings a third program with blockbuster market potential to our portfolio," said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics. "Amicus is well positioned to create substantial value for shareholders and to deliver on our mission for patients, and we very much look forward to working with Dimerix to bring this much needed therapy to people living with FSGS in the United States."
The licensing deal includes an upfront payment of $30 million and has been incorporated into the company's updated financial guidance for 2025.
Commercial and Regulatory Progress
Amicus reported several important commercial and regulatory achievements during the quarter:
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Pombiliti + Opfolda was selected as the preferred treatment for adults living with late-onset Pompe disease in the Netherlands under a five-year agreement. The Netherlands has the highest prevalence of Pompe disease in Europe, with over 150 individuals living with the condition.
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Regulatory approvals for Pombiliti + Opfolda were granted in Canada and Australia for adult LOPD patients. The company anticipates a regulatory decision in Japan later this year and remains on track for up to 10 new launch countries in 2025, which include more than 650 individuals living with LOPD.
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A commercial manufacturing and supply services agreement was reached with Sharp Sterile to manufacture Pombiliti drug product in the U.S., further diversifying the supply chain.
Updated 2025 Financial Guidance
In light of Q1 results and the DMX-200 licensing agreement, Amicus has updated its financial guidance for 2025:
- Total revenue growth is now expected to be 15-22% at CER, adjusted from the previous 17-24% range.
- Galafold revenue growth guidance remains unchanged at 10-15% at CER.
- Pombiliti + Opfolda growth is now projected at 50-65% at CER, down from the previous 65-85% range.
- Non-GAAP operating expenses are expected to be $380-400 million, including the $30 million upfront license payment for DMX-200.
Despite these adjustments, Amicus reaffirmed its expectation to achieve GAAP profitability during the second half of 2025. The company also maintains its long-term growth outlook, anticipating surpassing $1 billion in total sales by 2028.
Management Perspective
"Amicus delivered another consecutive quarter of significant double-digit revenue growth," said Bradley Campbell. "Looking forward, the underlying patient demand we observed in Q1 will drive robust growth for Galafold, and we continue to expect accelerating Pombiliti + Opfolda sales as the year progresses, driven by patient starts from new launch markets as well as anticipated acceleration in U.S. switches."
Campbell added, "While some unexpected factors impacted revenue in the quarter, the key performance indicators for both products are very strong and we remain on-track to achieve GAAP profitability during the second half of 2025 and to deliver double-digit revenue growth this year and beyond."
About Amicus Therapeutics' Products
Galafold (migalastat) is an oral pharmacological chaperone for the treatment of Fabry disease in adults with amenable galactosidase alpha gene (GLA) variants. The treatment works by stabilizing the body's own dysfunctional enzyme to clear accumulated disease substrate. Galafold is approved in more than 40 countries worldwide.
Pombiliti + Opfolda is a two-component therapy for late-onset Pompe disease. It consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor, and miglustat, an oral enzyme stabilizer designed to reduce loss of enzyme activity in the blood.
DMX-200, the newly licensed product, is a first-in-class treatment in Phase 3 development for FSGS, a rare kidney disease with significant unmet medical need.
As Amicus continues to expand its rare disease portfolio and global commercial presence, the company remains focused on its mission of developing and delivering novel high-quality medicines for people living with rare diseases while creating value for shareholders.
