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BioMarin Files MAA to EMA for Gene Therapy of Hemophilia A

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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that it has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval for valoctocogene roxaparvovec, an investigational gene therapy, developed for treating adult patients with severe hemophilia A.

BioMarin expects the MAA review process to begin under accelerated assessment in January 2020, subject to the completion of the EMA’s validation check.

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. Per the company, this is the first marketing application submission for a gene therapy product for any type of hemophilia.

Shares of BioMarin have declined 9.5% so far this year against the industry’s increase of 4.7%.


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.

BioMarin is on track to submit a biologics license application for valoctocogene roxaparvovec to the FDA by this year-end. The candidate already enjoys the FDA’s Breakthrough Therapy Designation for severe hemophilia A.

Hemophilia A is a genetic disorder caused by missing or defective factor VIII, a clotting protein. Despite being prescribed as the current standard of care medicines, severe hemophilia A patients 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 - Free Report) and partner Pfizer, Inc. (PFE - Free Report) 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 Spark Therapeutics and uniQure.

Zacks Rank

BioMarin currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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