RARE Daily

BioMarin Acquires Inozyme to Strengthen Enzyme Therapy Business

May 19, 2025

Rare Daily Staff

BioMarin Pharmaceutical agreed to acquire Inozyme Pharma for $4 per share in an all-cash transaction worth approximately $270 million, a deal that will strengthen the company’s enzyme therapy business.

The acquisition will add a late-stage enzyme replacement therapy, INZ-701, which is currently being evaluated for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency, a rare and progressive genetic condition that affects blood vessels, soft tissues, and bones. The condition is associated with increased cardiovascular mortality risk across all age groups, especially in infants. It is also associated with severe rickets and a softening of the bones in children and adults.

Data from the first phase 3 pivotal study of INZ-701 in children is expected in early 2026, with potential regulatory approval in 2027.

“BioMarin has been deeply committed to advancing enzyme therapies for children and adults living with serious genetic conditions for more than 25 years, and today’s agreement builds on our legacy,” said Alexander Hardy, president and CEO of BioMarin. “This acquisition brings to BioMarin an important medicine that has the potential to be the first treatment for children and adults with ENPP1 Deficiency, improving care for people living with this serious condition.”

The transaction, which has been unanimously approved by the boards of directors of both companies, is expected to close in the third quarter of 2025, subject to regulatory approval, successful completion of a tender offer and other customary closing conditions.

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