FDA accepts BLA's for exagamglogene autotemcel for severe sickle cell disease and transfusion-dependent beta thalassemia.- Vertex Pharma + CRISPR Therapeutics
Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that the FDA has accepted the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
The FDA has granted Priority Review for SCD and Standard Review for TDT and assigned Prescription Drug User Fee Act (PDUFA) target action dates of December 8, 2023, and March 30, 2024, respectively.
Updated data from the pivotal trials supporting the regulatory submissions will be presented at the Annual European Hematology Association Congress on June 11, 2023. In the U.S., exa-cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA for both TDT and SCD.
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