Health Canada has granted Marketing Authorization for CASGEVY (exagamglogene autotemcel), a gene-edited cell therapy. This approval is for patients aged 12 years and older with sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).
The authorization is based on positive interim results from two ongoing clinical trials. These trials met their primary endpoints, demonstrating that users were free from severe VOCs or achieved transfusion independence for at least 12 consecutive months.
An estimated 2,000 patients in Canada are eligible for this treatment. Vertex Pharmaceuticals is working with provincial, territorial, and federal governments to facilitate rapid access to this one-time therapy.
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