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Positive CHMP Opinion For BioMarin's Gene Therapy To Treat Severe Hemophilia A

BioMarin Pharmaceutical Inc. (BMRN) on Friday said it received positive opinion from European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) for conditional marketing authorization of its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A.

The CHMP based its positive opinion on the totality of data from the valoctocogene roxaparvovec clinical development program, including two-year outcomes from the global GENEr8-1 Phase 3 study, supported by five and four years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts respectively, in the ongoing Phase 1/2 dose escalation study, BioMarin said.

The company plans to market the drug under the brand name Roctavian.

Roctavian is the first gene therapy to be recommended for approval in Europe for hemophilia A. A final decision is expected in the third quarter of 2022.

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