Posted by Michael Wonder on 19 Aug 2020
BioMarin receives complete response letter from FDA for valoctocogene roxaparvovec gene therapy for severe haemophilia A
19 August 2020 - FDA introduces new recommendation for 2 year annualised bleeding rate as primary outcome for on-going Phase 3 clinical trial 270-301.
BioMarin Pharmaceutical today announced that the U.S. FDA issued a complete response letter to the Company's biologics license application for valoctocogene roxaparvovec gene therapy for severe haemophilia A on 18 August 2020.
The FDA issues a complete response letter to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form.
