BioMarin Receives European Commission's Conditional Marketing Authorization For Gene Therapy Candidate

The European Commission (EC) has granted conditional marketing authorization (CMA) to BioMarin Pharmaceutical’s BMRN lead asset ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A in adult patients.

The regulatory decision is supported by significant body of data from the Roctavian clinical development program, the most extensively studied gene therapy for hemophilia A, including two-year outcomes from the global GENEr8-1 Phase 3 study. 

The Hemophilia A patients have a mutation in the gene responsible for producing Factor VIII, a protein necessary for blood clotting.

ROCTAVIAN (valoctocogene roxaparvovec) is a gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).

Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, commented: "Roctavian approval in Europe is a historic milestone in medicine and is built upon almost four decades of scientific discovery, innovation, and perseverance. We thank the European Commission for recognizing Roctavian's value as the first gene therapy for hemophilia A, a feat that we believe will transform how healthcare professionals and the patient community think about caring for bleeding disorders."

The European Commission also endorsed European Medicines Agency (EMA) recommendation for Roctavian to maintain orphan drug designation, by-way-of providing 10-years of Market Exclusivity. 

A Conditional Marketing Authorization (CMA) recognizes that the medicine fulfils an unmet medical need based on a positive benefit-risk assessment, and that the benefit to public health of the immediate availability on the market outweighs the uncertainties inherent to the fact that additional data are still required.

Price Action : BioMarin shares are trading around 3 percent higher at $95.80 on Wednesday during after-hours session