The first gene therapy for haemophilia to be submitted for regulatory review, valoctocogene roxaparvovec, has been given ‘priority review’status by the US Food and Drug Administration. Developed by BioMarin Pharmaceutical Inc, the therapy will be assessed for safety and efficacy for the treatment of patients with haemophilia A. Haemophilia A is the more prevalent of the two disease types, haemophilia A and B. Under a priority review, the FDA aims to issue a decision on marketing in six months rather than the usual 10, which means the outcome of the review should be known by 21 August.
