Research & Development
BioMarin Pharmaceutical's valoctocogene roxaparvovec recommended for conditional marketing authorisation by European regulator
27 June 2022 -

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a US-based company involved in developing and commercialising innovative biopharmaceuticals for rare diseases driven by genetic causes, announced on Friday that it has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), the European regulator, recommending conditional marketing authorisation (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, intended to treat adults with severe haemophilia A.

The European Commission is likely to reveal a final approval decision, typically consistent with the CHMP recommendation in the third quarter of 2022. The one-time infusion is proposed to be marketed under the brand name ROCTAVIAN (valoctocogene roxaparvovec), intended to treat severe haemophilia 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). The product is the first gene therapy to be recommended for approval in Europe for haemophilia A.

The positive opinion is based CHMP's positive opinion on the totality of data from the valoctocogene roxaparvovec clinical development program, the most extensively studied gene therapy for haemophilia A, including two-year results from the global GENEr8-1 Phase three 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 one /two dose escalation study.



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