BioMarin Pharmaceutical Inc. completes $4.8B acquisition of Amicus Therapeutics, adding Fabry and Pompe disease therapies and expanding its rare disease portfolio.
Written By: Samiksha Jadhav, BPharm
Reviewed By: Pharmacally Editorial Team
BioMarin Pharmaceutical Inc. has completed its previously announced acquisition of Amicus Therapeutics in an all-cash transaction valued at approximately $4.8 billion, with Amicus shareholders receiving $14.50 per share. The deal expands BioMarin’s presence in rare genetic diseases, including lysosomal storage disorders such as Fabry and Pompe diseases.
The acquisition adds two approved therapies to BioMarin’s commercial portfolio: Galafold® (migalastat) for Fabry disease and Pombiliti® (cipaglucosidase alfa-atga) in combination with Opfolda® (miglustat) for Pompe disease.
BioMarin also gains U.S. rights to DMX-200, a Phase 3 investigational therapy for focal segmental glomerulosclerosis (FSGS), a rare and progressive kidney disorder.
BioMarin Chief Executive Officer Alexander Hardy said the acquisition aligns with the company’s strategy to diversify its growth profile while advancing treatments for rare diseases, adding that its global infrastructure and manufacturing capabilities are expected to support broader patient access.
Galafold is an oral pharmacological chaperone that stabilizes the alpha-galactosidase A enzyme in adults with Fabry disease who have amenable GLA gene variants, helping reduce the accumulation of disease-related substrate. It is approved in the United States, European Union, and more than 40 countries, based largely on biomarker reduction. Common adverse events include headache, nasopharyngitis, urinary tract infection, nausea, and fever.
Pombiliti and Opfolda combine an enzyme replacement therapy with an oral stabilizer designed to maintain enzyme activity in circulation. The regimen is indicated in the United States for adults with late-onset Pompe disease who are not adequately responding to existing enzyme replacement therapy. Safety considerations include hypersensitivity reactions, infusion-associated events, and a contraindication in pregnancy due to potential embryo-fetal toxicity.
Reference
BioMarin Pharmaceutical Inc. – BioMarin Completes Acquisition of Amicus Therapeutics
About the Writer
Samiksha Vikram Jadhav is a B. Pharm graduate with a strong academic foundation in pharmaceutical sciences, pharmacology, and drug development. She specializes in pharma market research, with a focused interest in mergers and acquisitions, strategic partnerships, and global pharma and biotech deals. Her work centers on analyzing industry transactions, market positioning, and business strategies, translating complex developments into clear, accurate, and insightful scientific and commercial reporting.