Amicus Therapeutics, Inc. is a U.S.-based biotechnology company focused on medicines for rare diseases, with commercial franchises in Fabry disease and late-onset Pompe disease. Its marketed products include Galafold® (migalastat), an oral therapy for Fabry patients with amenable genetic variants, and the two-component Pompe regimen Pombiliti® + Opfolda® (cipaglucosidase alfa-atga/miglustat). The company sells globally through specialty distributors and pharmacies, with reimbursement and government payors playing a central role in access and pricing. Amicus also expands its pipeline via partnering and licensing, including an exclusive U.S. commercialization license for Dimerix’s Phase 3 candidate DMX-200 for FSGS and other indications.
6,3 %
88,5 %
−4,3 %
+20,0 %
2.84
1.88
| % | |
|---|---|
| Galafold (Fabry disease) | 80% Oral precision therapy for Fabry patients with amenable genetic variants, sold commercially across multiple countries. |
| Pombiliti + Opfolda (Pompe disease) | 17% Two-component therapy for adults with late-onset Pompe disease, with approvals and reimbursement expansion underway globally. |
| Pipeline and partnered programs (e.g., DMX-200) | 3% Clinical-stage and in-licensed assets where Amicus funds development/regulatory work and prepares for commercialization in targeted markets. |
Amicus’s direct customers are typically specialty distributors and pharmacies that purchase Galafold® and Pombiliti® +...
Buy Galafold and Pombiliti + Opfolda for downstream dispensing; central to order flow and channel inventory dynamics.
Drive initiation and switching decisions based on efficacy, safety, convenience, and clinical guidelines for Fabry/Pompe.
Approve coverage, set reimbursement terms, and generate rebates/discounts that materially affect net product sales.
Influence diagnosis journeys, therapy selection, and affordability support; important for rare-disease adoption and persistence.
Amicus commercializes Galafold® in over 40 countries, including the United States, European Union, United Kingdom, and...
Amicus is prioritizing growth of its two commercial franchises by expanding geographic reach, securing reimbursement,...
Rare-disease revenue growth depends on approvals, reimbursement, and patient identification across countries.
In-licensed or acquired assets can diversify beyond two products and extend growth runway in rare/orphan diseases.
Outsourced manufacturing and regulatory approvals can constrain supply and limit commercial execution if not de-risked.
Amicus’s revenue base is concentrated in two rare-disease products, so competitive entries, label changes, or...
Although EMA approved, the facility may not be approved by FDA/other regulators, potentially delaying supply resilience and requiring additional capital.
Distributors may underinvest, face financial/compliance issues, or terminate/expire arrangements, interrupting sales and reducing revenue.
Attacks could lead to data loss, ransomware costs, operational disruption, and reputational harm; supply-chain incidents can have similar effects.
Non-completion within expected timeframe or at all could adversely affect business, financial results, and operations.
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: 11/08/2026