Earlier today, UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorization to Vertex Pharmaceuticals and CRISPR Therapeutics for their CRISPR/Cas9 gene therapy, CASGEVY (exagamglogene autotemcel), marking the world’s first regulatory approval for a CRISPR-based gene-edited therapy.  CASGEVY is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR.  CASGEVY is approved for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).  The approval is based on two global clinical trials, demonstrating significant efficacy in reducing severe pain crises and achieving red blood cell transfusion independence.  The therapy utilizes a patient’s own stem cells genetically edited with CRISPR/Cas9 to enhance fetal hemoglobin production.

As we previously reported, Vertex and CRISPR have also submitted Biologics License Applications (BLA) to the Food and Drug Administration (FDA) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe SCD and TDT.  The FDA 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.

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