BioMarin Pharmaceutical has agreed to buy rare disease biotech Amicus Therapeutics, adding three approved or late-stage lysosomal storage disorder therapies to its portfolio.
Under the terms of the agreement, BioMarin will pay cash for all of Amicus’ shares for a total consideration of $4.8bn. This deal will set BioMarin back $14.50 per share, constituting a 33% premium on Amicus’ $10.89 18 December stock value at market close.
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BioMarin’s stock has increased 17.71% on the acquisition, from an 18 December close of $51.95 to a 19 December close of $61.15.
This multi-billion-dollar deal, which is the biggest in BioMarin’s history, will give the pharma rights to Amicus’ Fabry disease therapy, Galafold (migalastat), which has been on the US market since 2018. In 2024, the drug brought in $458.1m for Amicus, an 18% increase on 2023.
Galafold is currently the only US Food and Drug Administration (FDA) approved chaperone therapy for Fabry disease – a rare genetic disorder characterised by the buildup of fatty substrates in the cells of an affected patient.
Following a US patent litigation with generics giant Teva Pharmaceuticals, Galafold now holds market exclusivity until January 30, 2027.
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By GlobalDataBioMarin will also acquire the rights to Amicus’ FDA-approved Pompe disease combination, Pombiliti (cipaglucosidase alfa-atga) plus Opfolda (miglustat), which got the FDA go-ahead for use in patients who are not responding to standard enzyme replacement therapy back in 2023. The drug generated $22m in 2024.
Like Fabre disease, Pompe disease is a lysosomal storage disorder that prevents the metabolism of glycogen, leading to its harmful buildup within the cells. This can lead to progressive muscular weakness and heart problems.
Alongside these approved therapies, BioMarin will take ownership of the US rights to the late-stage, investigational oral small molecule, DMX-200, which is currently in Phase III trials for rare kidney disease, focal segmental glomerulosclerosis (FSCS). Amicus originally purchased the US rights to DMX-200 from the drug’s creator, Dimerix, for up to $105m in May 2025.
BioMarin eyes future growth
Over the last couple of years, BioMarin has been looking to further its growth in the pharmaceutical sector.
In a bid to achieve this, the company underwent a significant restructuring in 2024, which saw it let go of 225 employees.
Moving into 2025, BioMarin has employed a strong dealmaking strategy, which saw the company sign 10 deals in 2025. According to GlobalData’s Pharmaceutical Intelligence Center, this is the highest number of deals to be signed in one year since 2010.
One of the more prominent deals was BioMarin’s acquisition of Inozyme, which set it back $270m, while adding Inozyme’s late-stage enzyme replacement therapy, INZ-701, to its pipeline.
Following its merger with Amicus, BioMarin noted that both Galafold and Pombiliti-Opfolda have “high growth potential,” meaning the acquisition could further accelerate the company’s revenue growth moving forward.
This could be further supported by the expansion of Galafold and Pombiliit-Opfolda’s global footprint, which BioMarin mentioned as a possibility in a 19 December statement.
Analysts at GlobalData, parent company of Pharmaceutical Technology, currently forecast that Galafold will generate sales of $840m in 2031, while Pombiliti-Opfolda is expected to bring in $691m during the same year.
