Bluebird Bio announced their submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy for patients 12 and older with sickle cell disease (SCD) and a history of vaso-occlusive events.
The submission, announced via news release, also includes a request for Priority Review, which would expedite the review process and shorten it to 6 months instead of 10.
“We are pleased to have satisfied the Agency’s questions about comparability to enable our BLA submission, and to take this important step toward making lovo-cel available for individuals living with SCD,” said Andrew Obenshain, chief executive officer, Bluebird Bio.
SCD is a rare, progressive genetic disorder characterized by high concentrations of sickle hemoglobin in the blood. It causes vaso-occlusion, acute pain crises, organ damage, and early death.
Read more about SCD therapies
Lovo-cell is an investigational, one-time therapy that is designed to add functional copies of a version of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic stem cells. Their red blood cells can then produce antisickling hemoglobin, thereby reducing sickled hemoglobin, hemolysis, and other SCD complications.
Lovo-cel has already been granted Orphan Drug Designation, Fast Track Designation, Regenerative Medicine Advanced Therapy Designation, and Rare Pediatric Disease Designation by the FDA. Bluebird Bio’s clinical development program includes a completed phase 1/2 HGB-205 trial, and two ongoing studies, phase 1/2 HGB-206 and phase 3 HGB-210.
LTF-307, a long-term safety and efficacy study of individuals treated with lovo-cel, is also underway.
The BLA is based on results from the HGB-206 group C cohort of 36 patients over a 32-month period and 2 patients in the HGB-210 trial with 18 months of follow-up. Also included are safety data on 50 patients in the lovo-cel clinical development program, 6 of whom have 6 or more years of follow-up.
Bluebird bio submits Biologics License Application (BLA) to FDA 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; April 24, 2023.