BioMarin ends year with $4.8bn play for Amicus
BioMarin's chief business officer James Sabry
2025 has been a reset year for BioMarin Pharmaceutical, and as 2026 approaches, it has reached a $4.8 billion deal to buy Amicus Therapeutics, further extending a renewed push into rare diseases.
The $14.50 per share agreement, if completed, will add two fast-growing treatments for two lysosomal storage disorders, Fabry and Pompe disease, plus a third drug in phase 3 testing for the rare kidney disease focal segmental glomerulosclerosis (FSGS).
It's the second acquisition made by BioMarin since the company enticed former Roche dealmaker James Sabry to join the company, coming after it bought Inozyme for $270 million earlier this year, and the largest in its history.
By adding two products that are already on the market – oral Fabry disease drug Galafold (migalastat) and Pompe disease combination treatment Pombiliti (cipaglucosidase alfa) plus Opfolda (miglustat) – adding Amicus would also help BioMarin leap towards its target of growing annual sales to $4 billion in 2027.
First launched in 2018 as an oral alternative to injectable Fabry therapies, Galafold continues to show healthy growth, with sales rising 10% to $371 million in the first nine months of the year. BioMarin's play comes straight after patent litigation with Aurobindo Pharma and Lupin – who were seeking to bring generics of the drug to the US market – was resolved with an agreement that will delay entry until 2037.
Meanwhile, Pombiliti + Opfolda – launched in 2023 and still rolling out onto international markets – climbed nearly 60% to $77 million in the same period.
Combined, the two drugs made revenues of nearly $600 million in the last four quarters, while BioMarin has forecast that its current product portfolio will reach up to $3.2 billion this year. In a statement, Amicus and BioMarin said the merger would accelerate the launch of Galafold and Pombiliti + Opfolda into new markets.
BioMarin slashed staff in 2024 as part of a restructuring drive that also included scaling down operations supporting haemophilia A gene therapy Roctavian (valoctocogene roxaparvovec) to three key markets, following a disappointing rollout, and narrowing its R&D programmes.
The company's biggest product is achondroplasia therapy Voxzogo (vosoritide), which made $654 million in the first nine months of the year, but could soon face competition from Ascendis Pharma's TransCon CNP (navepegritide), currently under review by the FDA with a decision due at the end of February, as well as BridgeBio's infigratinib and Ribomic's umedaptanib pegol (RBM-007), which are in phase 3 and phase 2 clinical testing, respectively.
