Amicus Therapeutics, Inc.

Company and mission

Amicus Therapeutics is a global biotechnology company focused on developing and delivering medicines for people living with rare diseases. Its mission is to redefine expectations in rare disease by advancing internally developed, jointly developed, acquired, or in-licensed medicines and platforms.

Marketed products

• Galafold (migalastat HCl)

  • Oral pharmacological chaperone for Fabry disease; approved as monotherapy for adults with amenable GLA variants.
  • Approved in the U.S., EU, U.K., Japan and more than 40 countries in total.
  • Holds orphan designation in the U.S., EU, U.K., Japan and other countries.

• Pombiliti + Opfolda (cipaglucosidase alfa-atga + miglustat)

  • Two-component treatment for adults with late-onset Pompe disease (LOPD).
  • Approved in the U.S., EU, U.K., Canada, Switzerland, Australia and Japan; holds orphan designation/status in several regions and EU data exclusivity.

• DMX-200 (CCR2 inhibitor) for focal segmental glomerulosclerosis (FSGS)

  • Small molecule in a pivotal Phase 3 program (ACTION3), licensed from Dimerix Bioscience for U.S. rights.
  • Dimerix funds ACTION3; Amicus is responsible for the U.S. regulatory dossier submission and U.S. commercialization activities.

Pipeline and partnerships

• DMX-200

  • Amicus holds exclusive U.S. rights licensed from Dimerix as of April 30, 2025.
  • The licensing arrangement includes upfront and potential milestone payments plus royalties; Dimerix continues to fund ACTION3.

• Key collaborations

  • GlaxoSmithKline (GSK): collaboration to develop and commercialize Galafold globally as monotherapy and in combination; includes post-approval milestones up to $40 million and tiered royalties in the mid-teens in eight major markets outside the U.S.
  • Dimerix: exclusive U.S. commercialization rights for DMX-200 with a $30 million upfront payment and potential milestone and royalty payments across indications; the agreement establishes a joint steering committee for U.S. development and commercialization.

Manufacturing and distribution

Amicus relies on contract manufacturers, distributors and contract research organizations to supply Galafold, Pombiliti + Opfolda and pipeline products.

Financial position (selected 2025 figures)

• Revenue by product (2025):
  • Galafold: $521.7 million consolidated revenue (increase of $63.6 million versus prior year).
  • Pombiliti + Opfolda: $112.5 million consolidated revenue (increase of $42.3 million versus prior year).
• Cash and equivalents: $293.5 million in total cash, cash equivalents and marketable securities as of December 31, 2025.
• Profitability: The company has incurred losses historically and expects to continue incurring losses.
• Employees: 511 full-time employees as of December 31, 2025. Workforce composition: 58% women globally; 27% of executive management; 33% of the Board of Directors are women.

Geography and regulatory status

Galafold and Pombiliti + Opfolda are approved across multiple major markets (U.S., EU, U.K., Japan, Canada, Australia, etc.). The products benefit from orphan drug designations and other regulatory exclusivities in several regions, including EU data exclusivity. Both products are subject to ongoing post-approval commitments and potential labeling, safety and reimbursement considerations.

Corporate and strategic developments

• Merger agreement: On December 19, 2025, Amicus entered a definitive merger agreement with BioMarin Pharmaceutical Inc. Under the agreement, BioMarin would acquire Amicus through a merger, with each outstanding share (excluding excluded or dissenting shares) canceled for $14.50 per share in cash. The transaction is expected to close in the second quarter of 2026, subject to regulatory clearances, stockholder approval and other customary conditions.
• Implications and terms: If completed, Amicus common stock would no longer be publicly listed. The agreement includes customary closing conditions and termination rights, including a possible $175 million termination fee in specified circumstances. While the merger is pending, Amicus is subject to certain restrictions on its activities and must manage transaction and integration considerations.

Additional notes

Amicus concentrates on Fabry and Pompe disease while expanding its franchise through in-licensing, collaborations and internal development. The company’s use of third-party manufacturing, distribution and research partners introduces supply and development risk factors typical for the industry.

Summary

Amicus is a rare-disease focused biotech with two commercial therapies—Galafold for Fabry and Pombiliti + Opfolda for Pompe—and a DMX-200 program for FSGS licensed from Dimerix. In 2025, Galafold and Pombiliti + Opfolda generated $521.7 million and $112.5 million in revenue, respectively, and the company held $293.5 million in cash and equivalents. Amicus employed 511 people as of year-end 2025 and is pursuing a strategic merger with BioMarin that would change its corporate structure and public market status upon closing.