The US Food and Drug Administration (FDA) will conduct a priority review of the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel), an experimental drug for the treatment of sickle cell disease (SCD), bluebird bio announced.
The Prescription Drug User Fee Act goal date is set for December 20, 2023.
“The FDA’s acceptance of our BLA for lovo-cel moves us one step closer to bringing a potentially transformative therapy to the SCD community that is long overdue, and we are grateful to the patients, caregivers, researchers, clinicians, and community leaders who have enabled this exciting milestone. We look forward to working with the agency on its review,” said Andrew Obenshain, chief executive officer, bluebird bio.
The researchers submitted efficacy results obtained from 36 patients in the HGB-206 study group C cohort with a median of 32 months of follow-up and 2 patients in the HGB-210 study with 18 months of follow-up each, as well as safety data from 50 patients treated across the entire lovo-cel program, including 6 patients with 6 or more years of follow-up, which is the most extensive follow-up of any gene therapy program for SCD.
Read more about SCD experimental therapies
The experimental drug has previously received orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation.
Lovo-cel is a one-time gene therapy for patients with SCD aged 12 years or older with a history of vaso-occlusive events designed to add a functional gene that codes for the production of antisickling adult hemoglobin.
If approved, it 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. At present, lovo-cel is the most intensely studied gene therapy in development for SCD.
bluebird bio announces FDA priority review of the Biologics License Application for lovotibeglogene autotemcel (lovo-cel) for patients with sickle cell disease (SCD) 12 years and older with a history of vaso-occlusive events. News release. bluebird bio, Inc; June 21, 2023.