Bluebird Bio Says FDA Accepts BLA For Lovotibeglogene Autotemcel For Priority Review

(RTTNews) - bluebird bio, Inc. (BLUE) announced Wednesday that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) for priority review. The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023.

Lovo-cel is a potentially transformative one-time gene therapy for individuals living with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs).

The BLA for lovo-cel is based on efficacy results from 36 patients in the HGB-206 study Group C cohort with a median 32 months of follow-up and two patients in the HGB-210 study with 18 months of follow-up each.

The BLA submission also includes safety data from 50 patients treated across the entire lovo-cel program, including six patients with six or more years of follow-up, which is the longest follow-up of any gene therapy program for SCD.

If approved, lovo-cel will be bluebird bio's third ex-vivo gene therapy approved by the FDA for a rare genetic disease and its second FDA approval for an inherited hemoglobin disorder, building on more than a decade of leadership in gene therapy.