BioMarin Acquires Inozyme Pharma for $270M, Gaining Phase 3 Rare Disease Therapy

BioMarin Pharmaceutical has acquired Inozyme Pharma for $270 million in an all-cash deal, announced on May 16, 20251234.

The acquisition brings INZ-701, Inozyme's lead candidate, into BioMarin’s pipeline. INZ-701 is a first-in-class enzyme replacement therapy in Phase 3 development targeting ENPP1 Deficiency, a devastating rare genetic disorder affecting blood vessels, bones, and soft tissues, with no approved treatments34.

Phase 1/2 studies of INZ-701 have shown encouraging safety and efficacy signals, including no serious treatment-related adverse events and improvements in biomarkers and quality of life4.

BioMarin aims to strengthen its position in rare disease therapies and expand its enzyme-based drug portfolio, which already includes eight approved treatments for rare genetic disorders14.

The acquisition marks BioMarin’s first major M&A move under CEO Alexander Hardy, positioning the company to address a high-unmet-need, high-value market and potentially dominate the ENPP1 Deficiency therapeutic landscape34.

Sources:

1. https://firstwordpharma.com/story/5962344

2. https://www.biopharmadive.com/news/biomarin-inozyme-acquisition-deal-enzyme-rare-disease/748371/

3. https://www.fiercebiotech.com/biotech/biomarin-inks-270m-inozyme-buyout-bagging-phase-3-prospect-first-ma-move-sabry-era

4. https://www.ainvest.com/news/biomarin-bold-move-strategic-play-dominate-rare-disease-therapies-inozyme-breakthrough-inz-701-2505/