Vertex Pharmaceuticals, Crispr Therapeutics complete rolling BLAs to FDA

Vertex Pharmaceuticals (VRTX) and Crispr Therapeutics (CRSP) announced the completion of the rolling biologics license applications, or BLAs, to the FDA for the investigational treatment exagamglogene autotemcel for sickle cell disease, or SCD, and transfusion-dependent beta thalassemia, or TDT. The BLAs include requests for priority review, which if granted, would shorten the FDA’s review of the application to eight months from the time of submission versus a standard review timeline of 12 months. These submissions are supported by results from the ongoing Phase 3 studies, CLIMB-111 and CLIMB-121, as well as an ongoing long-term follow-up study, CLIMB-131. Data from the Phase 3 studies were most recently presented at the American Society of Hematology (ASH) Annual Meeting and Exposition in December of 2022.