Biomarin: Status of hemophilia A med application

 BioMarin plans to include the previously reported results from the two-year follow-up safety and efficacy data from the Phase 3 GENEr8-1 study in a Biologics Licensing Application (BLA) resubmission for valoctocogene roxaparvovec to the Food and Drug Administration (FDA).  Based on recent feedback received from the FDA related to BioMarin's plans for the upcoming BLA, the Agency has requested additional information and analyses of data to be included in the BLA prior to submission.  The FDA has not requested additional pre-clinical or clinical studies.  While at present, no requests have been made concerning evaluation of efficacy and safety three years after dosing from the GENEr8-1 study, BioMarin is aware that such data will become available during the anticipated BLA review.  Based on these new information requests, the BLA resubmission is now expected by the end of September.

https://finance.yahoo.com/news/biomarin-announces-durable-hemostatic-efficacy-123100345.html