CHMP recommends Roctavian to treat Haemophilia A- BioMarin.
BioMarin Pharmaceutical Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.
A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2022.
The one-time infusion is planned to be marketed under the brand name Roctavian (valoctocogene roxaparvovec), 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).Roctavian is the first gene therapy to be recommended for approval in Europe for hemophilia A.
It is estimated that more than 20,000 adults across Europe, Middle East, and Africa are affected by severe hemophilia A. BioMarin anticipates additional patient access through named patient sales based on an EMA approval in countries in the Middle East and Africa and expects additional market registrations to be facilitated by an anticipated EMA license.
People with hemophilia A have a mutation or irregularity in the gene responsible for producing Factor VIII (FVIII), a protein necessary for blood clotting. The standard of care for patients with severe hemophilia A is chronic lifelong injectable therapy to maintain enough clotting factor in the bloodstream to prevent bleeds. Investigational valoctocogene roxaparvovec gene therapy works by delivering a functional gene that is designed to enable the body to produce FVIII on its own with the goal of reducing the need for ongoing prophylaxis.
The CHMP based its positive opinion on the totality of data from the valoctocogene roxaparvovec clinical development program, the most extensively studied gene therapy for hemophilia A, including two-year outcomes from the global GENEr8-1 Phase III 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/II dose escalation study.