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.
"Today's positive CHMP opinion for Roctavian addresses the unmet medical needs in severe hemophilia A by providing a treatment option that has been shown in clinical studies can maintain effective levels of endogenously produced coagulation Factor VIII over multiple years with a single intravenous administration. Currently available treatment options require long-term, chronic use with a high degree of compliance to a prescribed schedule to be effective," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We are grateful to the patients, investigators and community who have been an integral part in reaching this important milestone that brings us one step closer to delivering on the promise and ingenuity of gene therapy. We are proud of this scientific accomplishment and committed to the ongoing study of Roctavian."
"The positive CHMP opinion offers hope for a new treatment option for people with severe hemophilia A, who have been bound to lifelong treatment and still experience serious health complications, such as breakthrough bleeding, pain, and joint damage, as well as having to constantly consider their condition in all aspects of their lives," said Professor Johannes Oldenburg, Director of the Institute of Experimental Haematology and Transfusion Medicine and the Haemophilia Centre at the University Clinic in Bonn, Germany. "The robust data set from the clinical trial program underscores the potential impact of gene therapy for patients, including a substantial and sustained reduction in bleeding that would have previously required Factor VIII infusions."
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 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 has committed to continue working with the broader community to monitor the long-term effects of treatment.
The CHMP is a scientific committee composed of representatives from the 27-member states of the EU, and Iceland, Norway, and Liechtenstein. The committee reviews medical product applications on its scientific and clinical merit and provides advice to the European Commission (EC), which has the authority to approve medicines for the EU.
A conditional marketing authorization (CMA) recognizes that benefit to public health of the immediate availability on the market outweighs the uncertainties inherent to the fact that the science is still new, as is the case with any gene therapy, and the fact that additional data are still required. Once a CMA has been granted by EMA, BioMarin will provide further data from ongoing studies within defined timelines to confirm that the benefits continue to outweigh the risks, building on what already constitutes the largest clinical data package for gene therapy in hemophilia A. Conversion to a standard marketing authorization will be contingent on the provision of additional data from currently ongoing Roctavian clinical studies, including longer-term follow up of patients enrolled in the pivotal trial GENEr8-1, as well as a study of corticosteroids for which enrollment is now complete. The final summary of product characteristics will be available when the product is approved by the European Commission.
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