Positive results from pivotal trials of Casgevy (exagamglogene autotemcel) highlighted in oral presentations at the American Society of Hematology (ASH) annual meeting
Vertex Pharmaceuticals Incorporated announced two oral presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition from the global pivotal trials of Casgevy (exagamglogene autotemcel [exa-cel])
Data from 96 patients (44 sickle cell disease [SCD], 52 transfusion-dependent beta thalassemia [TDT]) treated with Casgevy in pivotal studies, with the longest follow-up of more than four years, continue to reinforce the consistent and durable response to treatment. In addition, new data illustrating improvements in patient-reported outcomes after treatment with Casgevy were featured in poster presentations at ASH.
Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex stated. “These data demonstrate the consistent and durable efficacy of Casgevy in eliminating severe vaso-occlusive crises in patients with SCD and conferring transfusion independence in patients with TDT.”
Casgevy is approved by the FDA for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs). The use of Casgevy for the treatment of TDT in the U.S. remains investigational. Vertex has submitted a BLA to the FDA for the potential use of Casgevy for patients 12 years and older with TDT and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 30, 2024.
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