BioMarin expands enzyme offerings with $270M Inozyme acquisition

20 May 2025
BioMarin Pharmaceutical has announced its plans to acquire Inozyme Pharma, a Boston-based company, in a deal valued at approximately $270 million. The acquisition will be executed through an all-cash transaction, with BioMarin offering $4.00 per share. This strategic move aims to enhance BioMarin's portfolio by incorporating a promising late-stage enzyme replacement therapy designed for the treatment of rare genetic disorders.

The focus of this acquisition is on Inozyme's leading investigational therapy, INZ-701. This therapy is currently undergoing Phase III clinical trials for the treatment of ENPP1 deficiency, a rare genetic disorder. ENPP1 deficiency is known to affect blood vessels, soft tissues, and bones, leading to increased cardiovascular mortality and severe skeletal issues. If INZ-701 receives approval, it would be the first available treatment option for individuals suffering from this progressive and debilitating condition.

The timeline for the acquisition's completion is set for the third quarter of the year, pending regulatory approvals and customary closing conditions. BioMarin's interest in Inozyme's therapy underscores its commitment to addressing unmet medical needs in the realm of genetic disorders. With INZ-701, BioMarin hopes to provide a novel treatment avenue for patients who currently have no available therapies.

Looking ahead, BioMarin anticipates receiving initial pivotal data from pediatric testing of INZ-701 by early 2026. This data will be crucial in evaluating the therapy's efficacy and safety, ultimately determining its potential approval and subsequent commercial launch. Should the therapy meet the necessary criteria, BioMarin aims to make it available to patients by 2027. This acquisition not only fortifies BioMarin's position in the market for treatments of rare diseases but also exemplifies its proactive approach in advancing therapeutic options for underserved patient populations.

BioMarin's acquisition of Inozyme Pharma marks a significant step in the biopharmaceutical industry's efforts to innovate and expand treatment options for rare genetic disorders. The integration of INZ-701 into BioMarin's portfolio could potentially transform the standard of care for individuals with ENPP1 deficiency, offering hope and improved quality of life for patients and their families. As the company prepares for the acquisition's closure and the subsequent development of INZ-701, the medical community and stakeholders eagerly anticipate the potential impact of this new therapy on the treatment landscape for rare genetic conditions.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!