Bluebird Bio Sickle Cell Therapy Wins FDA Priority Review

Bluebird Bio has announced that the FDA has accepted the Biologics License Application (BLA) for lovo-cel for priority review. Lovo-cel is a one-time gene therapy for individuals living with sickle cell disease (SCD). It is specifically designed to treat the underlying cause of SCD through the addition of a functional gene that enables production of anti-sickling adult hemoglobin and is the most deeply studied gene therapy in development for this disease. The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023. 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.

The FDA’s Priority Review designation is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions, and targets a review timeline of six months from the time of filing, compared to a standard review timeline of 10 months. The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation.

Vertex Pharmaceuticals and CRISPR Therapeutics exa-cel SCD therapy received Priority Review status earlier this month with a target action date of December 8, 2023.

Sickle cell disease is a progressive genetic disease associated with debilitating and unpredictable pain crises, anemia, irreversible damage to vital organs, and early death. In the U.S., there are approximately 100,000 people living with SCD, and the median age of death is 45 years of age. While SCD was the first disease to have a genetic cause identified, treatment advances have lagged. Since that discovery in 1949, only four therapies have been approved, none of which address the underlying genetic cause of disease. (Source: Bluebird Bio Website, 21 June, 2023 and Vertex Website, 08 June 2023)