Vertex and Crispr announce FDA approval of Casgevy for sickle cell disease

Vertex Pharmaceuticals (blue‘>VRTX) and CRISPR Therapeutics (CRSP) announced that the U.S. Food and Drug Administration has approved Casgevy, a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease, or SCD, in patients 12 years and older with recurrent vaso-occlusive crises, or VOCs. “This approval means that for the first time, approximately 16,000 patients with SCD may be eligible for a durable one-time therapy that offers the potential of a functional cure for their disease by eliminating severe VOCs and hospitalizations caused by severe VOCs,” the companies stated. The administration of Casgevy requires specialized experience in stem cell transplantation; therefore, Vertex is engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers, or ATCs, throughout the U.S. to offer Casgevy to patients. Additional ATCs will be activated in the coming weeks, the company noted. The use of Casgevy for the treatment of transfusion-dependent beta thalassemia, or TDT, in the U.S. remains investigational. Vertex has submitted a BLA to the U.S. 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, or PDUFA, target action date of March 30, 2024. The press release contains no mention of a boxed warning for Casgevy; Bluebird Bio (BLUE), which also today received FDA approval for a gene therapy for SCD, says the safety information for Lyfgenia included a boxed warning for hematologic malignancy.