BioMarin completes US$4.8B acquisition of Amicus Therapeutics to expand rare disease portfolio

USA—BioMarin Pharmaceutical has completed its acquisition of Amicus Therapeutics in an all-cash transaction valued at approximately USD$4.8 billion.

Under the agreement, BioMarin acquired Amicus at US$14.50 per share, marking the culmination of a deal that was originally announced in December 2025.

The transaction strengthens BioMarin’s position in the rare disease therapeutics space and expands its commercial and clinical portfolio across multiple indications.

The acquisition brings Amicus Therapeutics fully under BioMarin’s global operations, integrating its late-stage and commercial-stage rare disease assets into BioMarin’s established infrastructure.

The company stated that it will leverage its global manufacturing capabilities and distribution networks to accelerate access to therapies across key markets.

Expansion of rare disease treatment portfolio

Through the acquisition, BioMarin adds two important therapies targeting lysosomal storage disorders, a group of rare inherited conditions characterized by enzyme deficiencies.

These include Galafold (migalastat), a treatment for Fabry disease, and the combination therapy Pombiliti (cipaglucosidase alfa-atga) with Opfolda (miglustat) for Pompe disease.

In addition, BioMarin secures US rights to DMX-200, an investigational oral small-molecule therapy currently in Phase III clinical development for focal segmental glomerulosclerosis (FSGS), a rare kidney disorder with limited treatment options.

Leadership perspective on the acquisition

BioMarin President and Chief Executive Officer Alexander Hardy described the transaction as a strategic milestone that supports the company’s long-term growth objectives in rare disease innovation.

He noted that the integration of Amicus Therapeutics enhances BioMarin’s ability to diversify its pipeline and strengthen its commercial presence.

He further emphasized that BioMarin’s global reach, established commercial infrastructure, and in-house manufacturing expertise will help expand patient access to both Galafold and Pombiliti + Opfolda across international markets.

Oral therapy for Fabry disease

Galafold is supplied as a 123 mg oral capsule and functions as a pharmacological chaperone that stabilizes alpha-galactosidase A (alpha-Gal A) enzyme activity.

It is designed for adults with Fabry disease who carry amenable GLA gene variants.

Clinical and epidemiological estimates from Amicus Therapeutics indicate that approximately 35% to 50% of individuals living with Fabry disease globally may carry amenable GLA variants, although prevalence varies by region.

The therapy has received regulatory approvals in more than 40 countries, including the United States, Japan, the United Kingdom, and across the European Union.

Pombiliti and Opfolda combination therapy for Pompe disease

The Pombiliti + Opfolda regimen combines cipaglucosidase alfa-atga, an enzyme replacement therapy enriched with bis-mannose-6-phosphate to improve cellular uptake, with miglustat, which helps stabilize enzyme activity in circulation.

In the United States, the combination is indicated for adult patients with late-onset Pompe disease who weigh at least 40 kilograms and who have not shown adequate clinical improvement on existing enzyme replacement therapies.

The dual-mechanism approach is designed to improve therapeutic efficacy in patients with limited response to standard treatment options.

Pipeline and legal advisory role

Beyond approved therapies, the acquisition also strengthens BioMarin’s late-stage pipeline through DMX-200, an investigational oral therapy being evaluated in Phase III studies for FSGS, a progressive kidney disease with high unmet medical need.

Cooley LLP served as legal adviser to BioMarin Pharmaceutical during the transaction, supporting the company through the acquisition process and regulatory closing requirements.