BioMarin Expands Rare Disease Portfolio with $270M Inozyme Acquisition

On May 16, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced a $270 million acquisition of Inozyme Pharma, aiming to expand its rare disease research. The all-cash deal, backed by both companies’ boards, is expected to close between July and September, giving BioMarin control of INZ-701, an enzyme replacement therapy targeting ENPP1 and ABCC6 deficiencies, life-threatening disorders caused by mineral buildup in soft tissues.

A close-up of a hand holding a vial of biopharmaceutical drugs ready to be administered.

With no FDA-approved treatments, INZ-701 aims to restore inorganic pyrophosphate levels, preventing tissue calcification. Late-stage trial results for ENPP1 deficiency are expected next year, with potential FDA approval by 2027. Analysts view the deal as a strategic expansion, complementing BioMarin Pharmaceutical Inc.’s (NASDAQ:BMRN) existing enzyme replacement therapies, including Vimizim, Naglazyme, and Palynziq.

Despite a rare patient pool of around 10,000 globally, experts see commercial potential, expecting the acquisition to strengthen BioMarin’s pipeline development. While some analysts praise its strong strategic fit, others believe it won’t significantly impact BioMarin’s overall market position.

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Disclosure: None.