The U.S. Food and Drug Administration expanded approval for Casgevy to include children aged two and older. CRISPR Therapeutics and Vertex Pharmaceuticals co-developed the therapy for sickle cell disease with recurrent vaso-occlusive crises and transfusion-dependent beta-thalassemia (TDT). The FDA previously restricted the treatment to patients aged 12 and over.
This decision makes Casgevy the first CRISPR/Cas9 gene-editing therapy available for this younger U.S. age group. Approximately 5,500 additional children in the U.S. are now eligible for the one-time treatment.
The FDA completed the accelerated review in 53 days. The agency processed the application under its priority voucher program.