BioMarin’s presence in rare enzyme disorders grows with $4.8 billion acquisition of Amicus Therapeutics

Rare disease drug developer BioMarin Pharmaceutical is looking to boost revenue with a $4.8 billion deal for Amicus Therapeutics, a company whose two marketed products each offer blockbuster potential and also complement their acquirer’s long history in therapies for rare enzyme deficiencies.

The deal announced Friday is BioMarin’s second acquisition under CEO Alexander Hardy, the Genentech veteran who joined the company two years ago. At that time, BioMarin envisioned its growth would rely on the hemophilia A gene therapy, Roctavian. But lackluster sales of the product led the company to pivot, turning to mergers and acquisitions to grow its pipeline and portfolio.

BioMarin’s first marketed product was an enzyme replacement therapy, and the San Rafael, Calif.-based biotechnology company currently markets five such products. The portfolio is led by a newer product, Voxzogo, a treatment for achondroplasia, the most common form of dwarfism. Approved in 2021, this peptide drug has become BioMarin’s best-seller. But looming competition in this indication from Ascendis Pharma and BridgeBio Pharma is putting pressure on BioMarin to find other ways to increase revenue.

“We do this [acquisition]as we said, about [business development]”, Hardy said in a conference call Friday morning. “At the same time, we are very confident in our ability to compete with Voxzogo in the achondroplasia space. But we can do a lot of things at the same time and we can create extraordinary value, we believe, for all of our stakeholders by doing deals like this.

For the nine months ended September 30, BioMarin reported revenue of $2.3 billion. Roctavian accounted for only $23 million of that total. In October, BioMarin announced it would pursue options to divest the gene therapy. The acquisition of Amicus continues BioMarin’s strategy of leveraging its strengths in the area of ​​rare enzyme deficiencies. In May, it agreed to pay $270 million to acquire Inozyme Pharma, whose lead program is in late-stage development for enzyme deficiency and for which there are currently no FDA-approved treatments. When this deal was announced, Hardy said the Inozyme therapy would benefit from BioMarin’s experience in identifying eligible patients to increase sales of its enzyme deficiency therapies. On Friday, he said the same thing about Amicus products.

Amicus was founded in 2005 by John Crowley, an entrepreneur who was researching new treatments for two of his children born with Pompe disease, a rare enzyme deficiency. The biotech’s first commercialized product was Galafold, a drug approved in 2018 for another enzyme deficiency called Fabry disease. In 2023, Amicus won FDA approval for Pombiliti and Opfolda, a two-drug regimen for Pompe. Together, these Amicus products accounted for $448.9 million in revenue during the first nine months of 2025, an 18.5% increase over the same period in 2024.

Brian Mueller, BioMarin’s chief financial officer, said that despite the size of the acquisition, the deal carries no clinical trial risks and that the two commercialized Amicus products generate a revenue base that is growing at a faster rate than BioMarin’s portfolio. The company anticipates that each Amicus product will become a successful seller. Alexander added that cash flow from the combined operations will allow BioMarin to build its financial reserves over the next 12 to 24 months to deploy towards more transactions. In the meantime, he said BioMarin would explore smaller deals to grow its R&D pipeline.

The Amicus acquisition should immediately accelerate BioMarin’s revenue growth, Leerink Partners analyst Joseph Schwartz said in a note sent to investors. The company estimates that the two commercialized Amicus products will represent $630 million in revenue in 2025, growing to more than $1 billion by 2027 and nearly $2 billion by 2035. Schwartz added that while there are few details on synergies and cost savings, in general the business combination “seems to make a lot of sense based on the strategic and financial overlap.”

“We have long heard the company’s views regarding its sustainable and growing enzyme therapy business, so the acquisition of Amicus makes perfect sense for us,” he said.

The acquisition terms announced Friday call for BioMarin to pay $14.50 in cash for each Amicus share, representing a premium of 33% over Thursday’s closing price and 58% over the average stock price over the past 60 days. BioMarin said it plans to finance the transaction with its cash on hand and approximately $3.7 billion in debt financing. The boards of both companies approved the deal, which still needs approval from Amicus shareholders and regulators. The companies expect to complete the transaction in the second quarter of 2026.

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