BLA Submitted for Lovo-cel, a One-Time Gene Therapy for Sickle Cell Disease

The Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel), an investigational gene therapy for sickle cell disease, has been submitted to the Food and Drug Administration (FDA) by bluebird bio. The Company is seeking approval of the treatment for patients 12 years of age and older who have a history of vaso-occlusive events.

Lovo-cel is an investigational one-time gene therapy designed to add functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells. This results in the production of anti-sickling hemoglobin that decreases the proportion of sickle hemoglobin, thereby reducing sickled red blood cells, hemolysis, and other complications.

The BLA submission includes data from the phase 1/2 HGB-205 study (ClinicalTrials.gov Identifier: NCT02151526), the ongoing phase 1/2 HGB-206 study (ClinicalTrials.gov Identifier: NCT02140554) and phase 3 HGB-210 study (ClinicalTrials.gov Identifier: NCT04293185).

Efficacy results were based on 36 patients from the HGB-206 study (Group C cohort) with a median follow-up of 32 months, and 2 patients from the HGB-210 study with 18 months of follow-up each. In the HGB-206 Group C cohort, treatment with lovo-cel led to sustained production of anti-sickling hemoglobin and complete resolution of severe vaso-occlusive events in most of the evaluated patients.

The Company has requested Priority Review of the application, which would shorten the review time from 10 months to 6 months. 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 sickle cell disease.