Vertex Pharmaceuticals, Crispr Therapeutics get MHRA authorization for Casgevy

Vertex Pharmaceuticals (VRTX) and Crispr Therapeutics (CRSP) announced that the United Kingdom Medicines and Healthcare products Regulatory Agency, or MHRA, has granted conditional marketing authorization for Casgevy, a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease, or SCD, and transfusion-dependent beta thalassemia, or TDT. Casgevy has been authorized for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises, or VOCs, or TDT, for whom a human leukocyte antigen, or HLA, matched related hematopoietic stem cell donor is not available. There are an estimated 2,000 patients eligible for Casgevy in the U.K. In two global clinical trials of Casgevy in SCD and TDT, the trials met their respective primary outcome of becoming free from severe VOCs or transfusion independent for at least 12 consecutive months. Once achieved, these benefits are potentially expected to be life-long. The safety profile of 97 SCD and TDT patients treated to date with Casgevy in these ongoing studies is generally consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.