First gene therapy for adults with severe Haemophilia A, BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec), approved by European Commission (EC)

25 August 2022


BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for the treatment of 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 EC also endorsed EMA’s recommendation for Roctavian to maintain orphan drug designation, thereby granting a 10-year period of market exclusivity. The EMA recommendation noted that, even in light of existing treatments, Roctavian may potentially offer a significant benefit to those affected with severe Haemophilia A. The one-time infusion is the first approved gene therapy for haemophilia A and works by delivering a functional gene that is designed to enable the body to produce Factor VIII on its own without the need for continued haemophilia prophylaxis, thus relieving patients of their treatment burden relative to currently available therapies. People with haemophilia A have a mutation in the gene responsible for producing Factor VIII, a protein necessary for blood clotting.

Dr Richard Fagan, Director of BioPharm at UCLB said, “We are delighted to see this success for BioMarin. Their work along with the work of Professor Amit Nathwani at UCL has created a milestone event in the treatment of haemophilia A, offering a potentially one time curative option. We look forward to seeing their success continue, and create even more positive impact.”

Read the full story on BioMarin’s website.