BioMarin announces incremental progress on BLA review for valoctocogene roxaparvovec AAV gene therapy for adults with severe hemophilia A

As part of their review of the BLA, the FDA has scheduled its Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility, located in Novato, CA. As anticipated, the FDA also has requested that the Company submit results from the upcoming three-year data analysis from the ongoing Phase III GENEr8-1 Study.

While the FDA did not communicate a change to the current PDUFA target action date of March 31, 2023, the Agency stated that submission of these results may qualify as a Major Amendment, which would extend the action date by 3 months. FDA will evaluate the additional data prior to making this determination.

"We appreciate the level of engagement from FDA this early in their BLA review cycle and are pleased to share that the inspection of our gene therapy manufacturing facility has now been scheduled. Additionally, with their request related to the upcoming three-year data analysis from our Phase III GENEr8-1 study, FDA stated that these data are expected to provide longer-term efficacy and safety information and would thus be useful to people with Hemophilia A and healthcare providers in making better and more informed decisions when considering valoctocogene roxaparvovec as a treatment choice should it be approved," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We are encouraged by the bleed control results observed in the first cohort of patients who reached three years of observation in the Phase III study as shared in January, and we look forward to reviewing the three-year results from all participants (N=134). We anticipate sharing the three-year results from all 134 participants in our Phase 3 study in early 2023."