Bluebird Bio submits BLA for lovo-cel gene therapy for SCD treatments

bluebird bio announced the submission of its Biologics License Application, BLA ,to the U.S. Food and Drug Administration, FDA ,for lovotibeglogene autotemcel or lovo-cel gene therapy in patients with sickle cell disease SCD ages 12 and older who have a history of vaso-occlusive events. The BLA includes a request for Priority Review, which, if granted, would shorten the FDA’s review of the application to six months from the time of filing, versus a standard review timeline of 10 months. 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 severity of sickle cell disease, and its impact on patients and caregivers, has been underappreciated and overlooked for far too long. Transformative therapies for this community are long overdue," said Andrew Obenshain, chief executive officer, bluebird bio. "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." The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy designation, and rare pediatric disease designation for the treatment of SCD.