BioMarin’s Biologics Licence Application (BLA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe haemophilia A has been accepted by the US Food and Drug Administration (FDA).
If approved, valoctocogene roxaparvovec would be the first gene therapy in the US for this indication.
People living with haemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk of painful and potentially life-threatening bleeds from even modest injuries.
Patients with the most severe form of the disease, which make up approximately 50% of the haemophilia A population, are currently treated with a prophylactic regimen of intravenous Factor VIII infusions administered two to three times per week or a bispecific monoclonal antibody that mimics the activity of Factor VIII, which is administered one to four times per month.
Despite these regimens, many patients continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.
The BLA includes data from the valoctocogene roxaparvovec clinical development programme, which the company reports is the most extensively studied gene therapy for severe haemophilia A.
This includes two-year outcomes from all study participants in the company’s global GENEr8-1 phase 3 study, which demonstrated stable and durable bleed control, including a reduction in the mean annualised bleeding rate and the mean annualised Factor VIII infusion rate.
“This BLA resubmission is an important step that brings us closer to delivering a gene therapy treatment choice to address the unmet needs of people with severe haemophilia A in the US… We look forward to working closely with the Agency on our application for this potentially transformative therapy,” said Hank Fuchs, president of worldwide research and development at BioMarin.
The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021, complementing the Breakthrough Therapy Designation it received in 2017.
The therapy has also received orphan drug designation from the European Medicines Agency and the FDA for the treatment of severe haemophilia A, as well as conditional marketing authorisation from the European Commission in August this year.