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BioMarin recently shared a community update on their Hemophilia A Clinical Development Program for valoctocogene roxaparvovec, the company’s investigational gene therapy currently under review by the U.S. Food and Drug Administration (FDA). It included a brief clinical trial overview with the following updates:

  • The Phase 1/2 study was initiated in 2015 and involved 15 individuals and two dose levels. Participants are now in long term follow-up post treatment with valoctocogene roxaparvovec.

  • The Phase 3 study (named GENEr8-1) is currently being conducted in 13 countries. This study has been fully enrolled since November 2019 with 134 participants who received investigational gene therapy.  Participants in the study continue to be closely followed as part of this study.

  • On January 9th, 2022, BioMarin released an update on the Phase 3 study of valoctocogene roxaparvovec, including the most recent topline data based on three years or more of follow up for individuals enrolled in the GENEr8-1 study. BioMarin intends to submit data to an upcoming medical congress and to relevant Health Authorities. BioMarin extends its sincere gratitude to our investigators and the study participants who have helped make this milestone possible.

  • On October 12, 2022, BioMarin’s Biologics License Application for valoctocogene roxaparvovecwas accepted for review by the FDA with a review action date of March 2023. Subject to completion of the filing review, FDA evaluates the data provided by BioMarin and makes its decisions based on their assessment of safety and efficacy.

Source: Biomarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community, January 2023