Business Description

Overview We are a leading, global biotechnology company with a clear and compelling mission to develop and deliver transformative medicines for people living with rare diseases. With extraordinary patient focus, we strive to redefine expectations in rare disease. Our two marketed therapies are Galafold ® , the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti ® + Opfolda ® , a novel two-component treatment for adults living with late-onset Pompe disease. Galafold ® (also referred to as "migalastat") is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold ® has been granted orphan drug designation in the U.S., E.U., U.K., Japan, and several other countries. Pombiliti ® + Opfolda ® (also referred to as "cipaglucosidase alfa-atga/miglustat") is approved in the U.S., the E.U., the U.K., Canada, Australia, Switzerland, and Japan. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti ® + Opfolda ® has been granted orphan drug designation or status in the U.S., U.K., Switzerland and Japan and data exclusivity in the E.U. On April 30, 2025, we entered into an exclusive license agreement with Dimerix Bioscience Pty Limited ("Dimerix") for the U.S. commercialization rights of Dimerix' Phase 3 drug candidate, DMX-200 for treatment of Focal Segmental Glomerulosclerosis ("FSGS") and other indications. On December 19, 2025, we entered into a definitive merger agreement (the "Merger Agreement") with BioMarin Pharmaceutical Inc. ("BioMarin") and its wholly owned acquisition subsidiary. Under the terms of the Merger Agreement, and in accordance with the General Corporation Law of the State of Delaware (the "DGCL"), if the transaction is completed, BioMarin's wholly owned acquisition subsidiary will merge with and into Amicus (the "Merger"), and Amicus will continue as the surviving corporation as a wholly owned subsidiary of BioMarin. In addition, all shares of our common stock outstanding, immediately prior to closing (other than Excluded Shares or Dissenting Shares (as defined in the Merger Agreement)) will be cancelled and converted into the right to receive $14.50 per share in cash, without interest and subject to any applicable withholding of taxes (the "Merger Consideration"). Following completion of the Merger, shares of our common stock will no longer be publicly listed. The transaction is expected to close in the second quarter of 2026, subject to regulatory clearances, approval by our stockholders and other customary closing conditions. Refer to "— Note 15. Merger Agreement," in our Notes to Consolidated Financial Statements for additional information. Our Strategy Our strategy is to create, manufacture, test, and deliver the highest quality medicines for people living with rare diseases through internally developed, jointly developed, acquired, or in-licensed products and product candidates. We are leveraging our global capabilities to develop and broaden our franchises in Fabry and Pompe disease, with focused discovery work on next-generation therapies and novel technologies. Highlights of our progress include: • Commercial success in Fabry disease . For the year ended December 31, 2025, Galafold ® revenue was $521.7 million of consolidated revenue, which represented an increase of $63.6 million compared to the prior year. We continue to see strong commercial momentum and expansion into additional geographies. • Commercial and regulatory success in Pompe disease . For the year ended December 31, 2025, Pombiliti ® + Opfolda ® revenue was $112.5 million of consolidated revenue, which represented an increase of $42.3 million compared to the prior year. Pombiliti ® + Opfolda ® has been approved by the respective regulatory authorities in the E.U., U.S., U.K., Canada, Switzerland, Australia, and Japan. Additionally, throughout 2024 and 2025, we established reimbursement agreements in multiple countries. -5- Table of Contents • Pipeline advancement and growth. On April 30, 2025, Amicus licensed exclusive rights to commercialize DMX-200, a small molecule currently in a pivotal Phase 3 study ("ACTION3"), in the U.S. for treatment of FSGS and other indications. • Financial strength.…