The Food and Administration (FDA) has extended the review period for the Biologics License Application (BLA) for valoctocogene roxaparvovec, an investigational adeno-associated virus (AAV) gene therapy, for the treatment of hemophilia A in adults.

The new Prescription Drug User Fee Act target date is June 30, 2023. The FDA determined that the 3-year data analysis from the ongoing phase 3 GENEr8-1 study ( Identifier: NCT03370913) constituted a major amendment due to the substantial amount of additional data.

Valoctocogene roxaparvovec is administered as a single infusion to produce clotting factor VIII. In December 2019, the BLA was originally submitted based on interim analysis from the phase 3 GENEr8-1 study ( Identifier: NCT03370913) and 3-year phase 1/2 data ( Identifier: NCT02576795).

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The FDA subsequently issued a Complete Response Letter (CRL) in August 2020, requesting the Company provide substantial evidence from its phase 3 study demonstrating durable effect using annualized bleeding rate (ABR) as the primary endpoint. 

To address the issues raised in the CRL, the BLA was resubmitted in October 2022, which included 2-year outcome data from the GENEr8-1 study and supportive data from 5 years of follow-up from the 6e13 vg/kg dose cohort in the ongoing phase 1/2 dose escalation study. 

“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. 


BioMarin provides update on FDA review of Roctavian (valoctocogene roxaparvovec) gene therapy for adults with severe hemophilia A. News release. BioMarin. March 6, 2023. Accessed March 7, 2023.

This article originally appeared on MPR