Biomarin recently provided an “Update for the Hemophilia Community,” on the ongoing clinical trial program for its investigational hemophilia A gene therapy, valoctocogene roxaparvovec. It comprises a brief overview of current clinical studies, including a phase 1/2 trial in which 15 participants received a one-time administration of valoctocogene roxaparvovec at one of two dose levels – these individuals are in long-term follow up.
The program also encompasses an ongoing phase 3 study (GENEr8-1) which is fully enrolled with 134 participants, all of whom have received a single dose administration of the therapy and have been observed for more than two years post treatment.
On September 29th, the company submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for valoctocogene roxaparvovec. BioMarin is the anticipating the FDA’s review of the BLA to begin this month.
Source: BioMarin Clinical Development Program Update, September 29, 2022