bluebird bio, Inc.’s BLA for lovotibeglogene autotemcel (lovo-cel) has been accepted for priority review by U.S. FDA. Lovo-cel is a one-time gene therapy for individuals with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs). It’s specifically designed to treat the underlying cause of SCD through the addition of a functional gene that enables production of anti-sickling adult hemoglobin. The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023.
 
The BLA for lovo-cel is based on efficacy results from 36 patients in the HGB-206 study 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.
 
The FDA’s Priority Review designation is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions, and targets a review timeline of six months from the time of filing, compared to a standard review timeline of 10 months.