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# BioMarin Asked to Submit New Data on Gene Therapy
- URL: https://www.fdaweb.com/biomarin-asked-to-submit-new-data-on-gene-therapy/
- Published: 2022-11-07T12:00:00.000Z
- Updated: 2026-09-14T18:05:21.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5153144

As expected, BioMarin Pharmaceutical says FDA has requested results from an upcoming three-year data analysis from the ongoing Phase 3 GENEr8-1 study on its resubmitted valoctocogene roxaparvovec, a gene therapy for adults with severe Hemophilia A that is currently under BLA review. The agency also told the company that it has scheduled a pre-licensure inspection of BioMarin’s gene therapy manufacturing facility in Novato, CA.

Last month, FDA accepted for review BioMarin’s BLA resubmission and set a user fee review action target date of 3/31\. Previously, BioMarin said it expects an additional three months may be added to the review “based on the number of data read-outs that will emerge during the procedure.” It now notes that the three-year data analysis just requested may result in a three-month review extension.

The resubmission includes the company’s response to a 2020 complete response letter and subsequent agency feedback, including two-year outcomes data from the global GENEr8-1 Phase 3 study and supportive data from five years of follow-up from the ongoing Phase 1/2 dose escalation study, BioMarin says. It also proposes a long-term extension study following all clinical trial participants for up to 15 years, as well as a post-approval registry study to follow patients dosed in a real-world setting.