Massachusetts-based biotech bluebird bio announced this week that it had successfully submitted its Biologics License Application to the FDA for lovo-cel, its sickle cell disease gene therapy.
Lovotibeglogene autotemcel, knowns as lovo-cel, is a personalized gene therapy treatment, in which the patents own blood stem cells are modified with a virus to add a new version of a gene, which is designed to help prevent sickle cell anemia.
The BLA is supported with data from efficacy results and safety data from 50 patients treated across the entire lovo-cel program, which includes several years of data. If approved, lovo-cel would be bluebird bio’s third rare disease ex-vivo gene therapy.
Because bluebird bio had refined its manufacturing processes for the drug, the agency recently asked for comparability data from its CMC practices in both commercial manufacturing facilities as well as clinical trials. bluebird said its Q1 BLA submission goal for lovo-cel was delayed while the company awaited FDA feedback on the CMC updates.
The agency has 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.