Regulatory Agency and Partner Announcements

Biomarin - Update on FDA approval for valoctocogene roxaparvovec gene therapy for severe hemophilia A
A statement was released by BioMarin regarding the status of the U.S. Food and Drug Administration (FDA) approval process for valoctocogene roxaparvovec gene therapy for severe hemophilia A. To read the full press release, please click here.
August 2020


Biomarin
Biomarin provides updated 4-year data from their Phase 1/2 Valoctocogene Roxaparvovec gene therapy trial in Late-Breaking Oral Presentation at World Federation of Hemophilia Virtual Summit. To read the full press release, please click here.
June 2020


EHC-EAHAD Joint position statement on Gene Therapy
May 2020