BioMarin Expands Rare Disease Portfolio with $270 Million Acquisition of Inozyme Pharma

In a significant move to expand its rare disease portfolio,
California-based BioMarin Pharmaceutical announced Friday its plans to acquire Boston’s Inozyme Pharma in an all-cash transaction valued at $270 million. The deal, which has received unanimous approval from both companies’ boards of directors, is expected to be finalized between July and September 2025.

The acquisition will provide BioMarin access to Inozyme’s leading experimental therapy, INZ-701, which targets rare genetic conditions ENPP1 deficiency and ABCC6 deficiency. These life-threatening disorders occur when the body fails to produce sufficient amounts of an enzyme crucial for creating inorganic pyrophosphate, a molecule that prevents mineral deposits in soft tissues.

Without proper levels of this molecule, patients can experience severe complications including bone weakness, pain, and dangerous arterial blockages that may lead to strokes, tissue death, or multiple organ failure. The conditions are particularly severe in newborns and infants, with mortality rates exceeding 50% in the most serious cases. Currently, there are no FDA-approved treatments for these conditions.

INZ-701, an enzyme replacement therapy, aims to restore the body’s ability to produce inorganic pyrophosphate. Clinical trials are progressing, with results from a pivotal late-stage study in children with ENPP1 deficiency anticipated next year, potentially leading to FDA approval by 2027. The company has already completed a Phase 2 study for ABCC6 deficiency and a Phase 1 trial for a related blood vessel calcification condition associated with kidney failure.

Industry analysts have responded positively to the announcement. Leerink Partners analyst Joseph Schwartz described the acquisition as fitting “like a glove” with BioMarin’s existing operations, which already include several enzyme replacement therapies such as Vimizim, Naglazyme, and Palynziq. The deal comes at a strategic time for BioMarin, following recent company restructuring that included workforce reductions and program cuts.

While ENPP1 deficiency affects only approximately 10,000 patients globally, analysts believe the treatment area could develop into a valuable market opportunity. Paul Matteis of Stifel noted BioMarin’s strong commercial infrastructure makes it well-positioned to maximize the potential of this late-stage asset.

However, not all analysts share the same level of enthusiasm. RBC Capital Markets’ Luca Issi, while acknowledging the deal’s strategic sense, suggested it might not significantly impact BioMarin’s overall performance.

Under the agreement terms, BioMarin will purchase Inozyme shares at $4 each, representing a substantial 180% premium over the company’s May 15 closing price of $1.42. BioMarin appears well-positioned
financially to execute the deal, with approximately $1.3 billion in cash and equivalents reported at the end of March 2025.

BioMarin CEO Alexander Hardy emphasized the company’s commitment to expanding its portfolio through both internal and external innovation, citing their strong financial position to acquire additional assets while advancing treatments for patients with unmet medical needs.

The acquisition aligns with BioMarin’s broader growth strategy, which includes plans to achieve $4 billion in annual revenue by 2027. Following the announcement, BioMarin’s stock showed modest gains, trading up slightly more than 1% to nearly $60 per share late Friday morning.