Source – bluebird bio
On June 21, 2023, the US Food and Drug Administration (FDA) accepted the Biologics Licence Application (BLA) for lovotibeglogene autotemcel (lovo-cell) for priority review. For sickle cell disease (SCD) patients with a history of vaso-occlusive episodes (VOEs), aged 12 and older, Lovo-cell is a potentially game-changing one-time gene therapy. The most extensively researched gene treatment for this condition is especially created to tackle the underlying cause of SCD by introducing a functioning gene that permits the creation of anti-sickling adult hemoglobin. The Prescription Drug User Fee Act (PDUFA) objective date stated by the FDA is December 20, 2023.
“The burden that people living with SCD and their families live with today is staggering. Beyond extreme pain crises that send patients to the hospital, SCD progression is associated with grave long-term consequences. The FDA’s acceptance of our BLA for lovo-cell moves us one step closer in bringing a potentially transformative therapy to the sickle cell disease community that is long overdue, and we are grateful to the patients, caregivers, researchers, clinicians, and community leaders who have enabled this exciting milestone. We look forward to working with the agency on its review.”
– Andrew Obenshain, chief executive officer, bluebird bio
Based on effectiveness data from 36 patients in the Group C cohort of the HGB-206 study with a median follow-up of 32 months and two patients in the HGB-210 study with a median follow-up of 18 months each, the BLA for lovo-cell was developed. The BLA application also included safety information from 50 patients who were treated throughout the whole lovo-cell programme, six of whom had the longest follow-up of any SCD gene therapy programme at six years or more.
If approved, lovo-cel will be bluebird bio’s third ex-vivo gene therapy and its second FDA approval for an inherited hemoglobin deficiency. This builds on the company’s more than ten years of pioneering work in gene therapy.
The FDA’s Priority Review designation, which targets a review timeline of six months from the time of filing as opposed to a standard review timeline of ten months, is given to therapies that have the potential to significantly improve the treatment, diagnosis, or prevention of serious conditions.
Previous FDA designations for lovo-cell included orphan drug, fast track, regenerative medicine advanced treatment (RMAT), and rare pediatric condition.