FDA Accepts Biologics License Applications for Exagamglogene Autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

- First CRISPR gene-editing filings to be accepted for review by FDA

- FDA grants Priority Review for severe sickle cell disease (SCD) and Standard Review for transfusion-dependent beta thalassemia (TDT)

- PDUFA target action date of December 8, 2023, for SCD and March 30, 2024, for TDT  

On June 8, 2023, Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced that the U.S. Food and Drug Administration (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 was presented at the Annual European Hematology Association Congress on June 11, 2023.

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