BLA is submitted to the FDA for lovotibeglogene autotemcel for patients with sickle cell disease 12 years and older with 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.

Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease. The BLA submission is based on efficacy results from 36 patients in the HGB-206 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.

The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation for the treatment of SCD.