BioMarin hemophilia gene therapy further delayed by FDA requests

Nov. 8, 2022

The U.S. FDA has requested more data on BioMarin Pharmaceutical's investigational AAV gene therapy for hemophilia A — further extending a regulatory process that has been strung out by repeated requests from the agency.

According to California-based BioMarin, as part of their review of the BLA, the FDA has scheduled a Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility, located in Novato, CA. The FDA also has requested that the company submit results from the upcoming three-year data analysis from the ongoing phase 3 GENEr8-1 study.

The submission of these results may likely qualify as a 'Major Amendment' to the BLA, which would extend the current PDUFA action date of March 31, 2023 by three months.

The FDA first rejected the company's BLA back in August 2020, noting that results from the phase 1/2 trials and phase 3 trials had conflicting data as to the protective durability of the drug.  Determined, BioMarin pressed forward and was planning to resubmit its BLA in June 2022. But a month prior to that happening, in a press release discussing study results, BioMarin quietly mentioned that the agency has requested additional information and analyses of data to be included in the BLA — pushing the the BLA resubmission to September.

In its Sept. resubmission, BioMarin addressed the concerns raised by an FDA’s complete response letter issued in 2020, including two-year outcome data from its GENEr8-1 phase 3 trial as well as five-year follow-up data from the previous studies. 

BioMarin's therapy — which got the green light by the European Commission back in August under the brand name Roctavian — was granted RMAT, Breakthrough Therapy and Orphan drug designations by the FDA.