FDA pushes back approval of BioMarin gene therapy for hemophilia

March 8, 2023

BioMarin’s gene therapy for hemophilia is going to have to wait a few more months, after the FDA determined that the submission of the data analysis from an ongoing phase 3 study constituted a Major Amendment.

The phase 3 trial, the GENEr8-1 study, is the largest and longest to date for a gene therapy in hemophilia. Due to the substantial amount of data that needs to be reviewed, the new PDUFA target action date is now June 30, 2023. 

This is the second delay and second major amendment to the BLA, after the initial delay was announced in November of last year. Back then, as a part of their review, the FDA scheduled a Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility, located in Novato, CA, and also requested that the company submit results from the upcoming three-year data analysis from the ongoing phase 3 GENEr8-1 study.

BioMarin's therapy — which got the green light by the European Commission back in August under the brand name Roctavian — was granted RMAT, Breakthrough Therapy and Orphan drug designations by the FDA.