BioMarin acquires Amicus Therapeutics for $4.8bn

The deal was originally announced in December 2025.

With the acquisition, BioMarin adds two new treatments for lysosomal storage diseases, Galafold (migalastat) for Fabry disease, and Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) for Pompe disease.

The company also secures US rights to DMX-200, an oral investigational small molecule for focal segmental glomerulosclerosis (FSGS), currently in Phase III trials.

BioMarin president and CEO Alexander Hardy said: “The completion of the Amicus acquisition advances BioMarin’s strategy to strengthen and diversify our growth profile while furthering our mission to deliver medicines for people living with rare diseases.

“BioMarin’s global scale, established commercial infrastructure, and advanced in-house manufacturing capabilities build on Amicus’ legacy and position us to bring Galafold and Pombiliti+Opfolda to more patients around the world.”

Supplied as a 123mg capsule, Galafold is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) intended for the treatment of Fabry disease in adults with amenable galactosidase alpha gene (GLA) variants.

According to Amicus Therapeutics, 35%-50% of people with Fabry disease globally may have amenable GLA variants, though rates vary geographically.

Galafold holds approvals in more than 40 countries, including Japan, the UK, the US, and the European Union.

Pombiliti plus Opfolda comprises cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA for enhanced enzyme uptake, and miglustat, which reduces enzyme activity loss in blood.

In the US, this combination is indicated for adult patients with late-onset Pompe disease weighing at least 40kg and not improving on their current enzyme replacement therapy.

Cooley advised BioMarin Pharmaceutical for this transaction.

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