On March 6th BioMarin announced that it received a notice from the U.S. Food and Drug Administration (FDA) indicating that the agency has extended their review of the company’s Biologics License Application for ROCTAVIAN™ (valoctocogene roxaparvovec).
ROCTAVIAN is Biomarin’s investigational hemophilia A gene therapy, which is currently in multiple ongoing clinical trials to observe its safety and efficacy at various dose levels in adult patients with severe hemophilia A.
According to a company press release, the FDA determined that the submission of the three-year data analysis from the ongoing Phase 3 GENEr8-1 study constituted a “Major Amendment” due to the substantial amount of additional data and therefore set a new PDUFA* Target Action Date of June 30, 2023 – the company had previously communicated that this data submission could be qualified as a Major Amendment.
“We are continuing to work closely with FDA and appreciate the agency’s active engagement as we seek to deliver this important therapy to patients with severe hemophilia A,” said Hank Fuchs, MD, president of Worldwide Research and Development of BioMarin. “The three-year data enhance our application and further reinforce our belief that ROCTAVIAN has the potential to fundamentally transform care for people with hemophilia A.”
* Created by Congress in 1992, the Prescription Drug User Fee Act (PDUFA) authorizes FDA to collect user fees from persons that submit certain human drug applications for review or that are named in approved applications as the sponsor of certain prescription drug products. Since the passage of PDUFA, user fees have played an important role in expediting the drug review and approval process.
Source: Cision (PR Newswire, March 6, 2023