Amicus Therapeutics (FOLD 0.46%), a specialist in rare disease medicines, delivered quarterly results on July 31, 2025 that notably surpassed expectations on both GAAP revenue and non-GAAP earnings. The company reported GAAP revenue of $154.7 million, up from $126.7 million (GAAP) a year earlier and $8.0 million ahead of the $146.7 million analyst target. Non-GAAP earnings per share came in at $0.01, far better than the non-GAAP EPS estimate of –$0.12. The quarter showed solid commercial momentum for the company’s lead therapies, with ongoing expansion of its pipeline and continued global launches. Despite higher operating expenses and a narrowed growth outlook for a key product line, overall performance marked another solid step in Amicus’s growth plan.
| Metric | Q2 2025 | Q2 2025 Estimate | Q2 2024 | Y/Y Change |
|---|---|---|---|---|
| EPS (Non-GAAP) | $0.01 | $(0.12) | $0.06 | (-83.3 %) |
| Revenue | $154.7 million | $146.7 million | $126.7 million | 22.1 % |
| Galafold Revenue | $128.9 million | $110.8 million | 16.3 % | |
| Pombiliti + Opfolda Revenue | $25.8 million | $15.9 million | 62.8 % | |
| Non-GAAP Operating Expenses | $127.8 million | $82.1 million | 55.7 % |
Source: Analyst estimates provided by FactSet. Management expectations based on management's guidance, as provided in Q1 2025 earnings report.
About Amicus Therapeutics and Its Focus
Amicus Therapeutics develops and markets treatments for people with rare diseases, focusing on genetic disorders. Its business centers on two key therapies: Galafold, a daily oral pill for Fabry disease, and Pombiliti + Opfolda, an enzyme replacement system for Pompe disease. Both products target lifelong, serious conditions for which there are few treatment options.
Amicus’s recent business drive has focused on broadening patient access, securing regulatory approvals in new markets, and expanding into related rare disease therapies. Success factors include continued revenue growth from Galafold and Pombiliti + Opfolda, innovation in the development pipeline, and careful management of intellectual property to protect its commercial edge.
Quarterly Highlights and Key Developments
Amicus delivered its seventeenth consecutive period of double-digit revenue expansion on a constant currency basis in Q2 2025, reinforcing its rare disease portfolio strength. The majority of company sales come from Galafold, an oral therapy for Fabry disease. Galafold contributed $128.9 million in revenue, up 16% year-over-year. Growth was fueled by new patient starts, continued high uptake in established markets, and strong product compliance. Galafold is now approved in more than 40 countries, and management reports global patient demand remains high.
Pombiliti + Opfolda, an enzyme system for Pompe disease, saw sales jump 63% to $25.8 million. This growth resulted from launches in five new countries and new patient initiations. Regulatory wins were key—Japan granted approval for adult late-onset Pompe disease in June 2025, and broader access deals were secured in various markets. While the segment’s sales growth was strong, management lowered full-year expectations last quarter to 50–65% for 2025 due to delayed patient starts and an unexpected increase in UK rebate rates—factors that affected both the pace and profitability of new launches. For context, the UK implemented a rebate increase from 15% to 22% in Q1 2025, impacting the revenue trajectory for the remainder of the year.
Beyond commercial products, the company made a notable advance in its research pipeline by licensing U.S. rights for DMX-200, a late-stage experimental drug for focal segmental glomerulosclerosis (FSGS). This rare kidney disorder has no approved therapies in the United States. Amicus paid a $30 million upfront fee as part of the licensing agreement, joining a pivotal phase 3 trial that is set to complete enrollment by year end. This move aims to diversify the company’s rare disease portfolio and capitalize on its marketing and regulatory strengths.
Operating expenses were higher, reflecting both the DMX-200 licensing payment and ongoing investments in research, development, and new market launches. Non-GAAP operating expenses rose to $127.8 million, up 56% from the prior year. Despite these costs, cash, cash equivalents, and marketable securities stood at $231.0 million. The balance sheet remains sound, with management noting that the upfront license drove the main change in cash levels from December.
Financial Outlook and What to Watch
Amicus reaffirmed its 2025 outlook, maintaining its expectation for total revenue to rise 15–22% at constant exchange rates, with Galafold revenue is projected to grow 10–15% at constant exchange rates (CER) in 2025 and Pombiliti + Opfolda revenue is expected to increase 50–65% at constant exchange rates (CER) in 2025. The company expects gross margin to normalize in the mid‑80% range for 2025. It reiterated its target for positive GAAP net income in the second half of 2025, a key milestone as it moves toward sustainable profitability. No changes were made to this earnings guidance since last quarter.
Looking ahead, investors should monitor further progress on new country launches, especially for Pombiliti + Opfolda, where successful patient onboarding and resolution of regional issues could boost growth. Key watch points also include trends in operating expenses given ongoing pipeline investments, and any shifts in payer or regulatory landscapes that affect product uptake or net pricing. Execution on these fronts will be vital for Amicus’s long-term growth trajectory.
Revenue and net income presented using U.S. generally accepted accounting principles (GAAP) unless otherwise noted.
