4 Trillion Won M&A Expands Rare Disease Treatment Portfolio, Raises Revenue Guidance

BioMarin Pharmaceutical Inc. (BMRN) reported first-quarter revenues for the period ended March 31 of approximately $766 million, a 3% increase year-over-year. Growth in enzyme replacement therapies and VOXZOGO sales drove the results, partly offset by a decline in ROCTAVIAN revenues following its voluntary sales suspension. Reflecting the Amicus Therapeutics acquisition, the company raised its full-year 2026 revenue guidance by $500 million to $3.8–3.9 billion, while slightly lowering its non-GAAP diluted EPS guidance due to acquisition-related dilution and higher operating expenses.

To finance the acquisition and secure working capital, BioMarin arranged approximately $3.7 billion of non-convertible debt and, on April 27, entered into a new syndicated loan agreement led by Citibank. During the quarter, the company also provided updates on the clinical progress of multiple pipeline programs—including FDA approval of PALYNZIQ for adolescents—and outlined key Phase III readouts and regulatory milestones scheduled for 2026–2027.

On April 27, BioMarin completed its acquisition of Amicus Therapeutics in an approximately $4.8 billion all-cash transaction, adding the Fabry disease therapy Galafold, the Pompe disease treatment Pomfility Plus Oppolda, and the late-stage clinical candidate DMX-200 to its portfolio. The acquisition was funded through new secured borrowings, the issuance of notes maturing in 2034, and existing cash, positioning the company to accelerate mid-term growth in the rare disease sector.

Headquartered in San Rafael, California, BioMarin is a global biotech specializing in rare and genetic diseases, with core strengths in enzyme replacement and gene therapies targeting lysosomal storage disorders. The rare disease therapeutics market—while serving small patient populations—features high pricing and significant barriers to entry, driving ongoing competition among major pharmaceutical companies and specialized biotechs for pipeline assets and M&A.