BioMarin Files BLA for Gene Therapy to Treat Hemophilia A

BioMarin Pharmaceutical Inc. BMRN announced that it has submitted a biologics license application (“BLA”) to the FDA seeking approval for valoctocogene roxaparvovec, an investigational gene therapy, developed for treating adult patients with severe hemophilia A.

The company anticipates that the review of the BLA will be initiated by the regulatory authority in February 2020. The FDA will provide an update on whether it will accept the BLA or not after completing the filing review.

The marketing application was based on interim data from an ongoing phase III study and the updated three-year results from a long-term phase I/II study. Previously announced interim data from the phase III study showed that eight out of 20 patients treated with the gene therapy candidate achieved factor VIII levels of 40 international units per deciliter. Patients also achieved significant reduction in annual bleed rate from baseline.

Per the company, this is the first BLA submission in the United States for a gene therapy product for any type of hemophilia.

Shares of BioMarin have gained 1% so far this year compared with the industry’s increase of 9.1%.

We remind investors that BioMarin was conducting two separate phase III studies, namely GENEr8–1 (6e13 vg/kg dose) and GENEr8–2 (4e13 vg/kg dose) on valoctocogene roxaparvovec for the treatment of patients without the pre-existing AAV5 antibodies.

However, the company stopped developing 4e13 vg/kg dose (GENEr8–2 study) of valoctocogene roxaparvovec due to patient preference for the 6e13 vg/kg dose. The GENEr8-1 study, however, continued to enroll for achieving its planned completion target of 130 total patients.

In a separate press release, BioMarin announced that its marketing authorization application seeking approval for valoctocogene roxaparvovec as a treatment for severe hemophilia A has been validated by the European Medicines Agency. The review of the regulatory application will start in January 2020 under accelerated assessment.

Hemophilia A is a genetic disorder caused by missing or defective factor VIII, a clotting protein. Despite being prescribed the current standard of care medicines, patients with severe hemophilia A persistently experience painful bleeds, thereby creating significant need for medicines that can improve patients' quality of life. Several companies are developing gene therapy products to treat severe hemophilia A.

Notably, Sangamo Therapeutics, Inc. SGMO and partner Pfizer, Inc. PFE are evaluating their gene therapy candidate SB-525 in a phase III study for addressing patients with severe hemophilia A. Other companies developing gene therapy candidates to treat hemophilia A include Roche and uniQure QURE.

Zacks Rank

BioMarin currently sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

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