Vertex gets expanded Casgevy label for kids 2+ years

The U.S. FDA has approved expanded use of Vertex Pharmaceuticals’ gene-edited cell therapy, Casgevy, for treatment of people ages 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).

The nod makes Casgevy the first approved genetic therapy indicated for children as young as 2 years for both SCD and TDT.

Last year, Vertex announced data from the CLIMB-141 and CLIMB-151 studies demonstrating the clinical benefits of Casgevy, in people ages 5 years and older living with SCD or TDT. Cohorts of the two phase 3 open-label studies remain ongoing, assessing the safety and efficacy of a single dose of Casgevy in patients ages 2-11 years with TDT or with SCD and recurrent VOCs.

Casgevy, co-developed with CRISPR Therapeutics, was approved by the FDA using the Commissioner’s National Priority Voucher in December 2023, as the first gene-edited cell therapy for patients with sickle cell disease. In early 2024, its approval was expanded to include the treatment of transfusion-dependent beta thalassemia in patients 12 years and older. Vertex has also recently completed regulatory submissions in Saudi Arabia andUK to expand the use of Casgevy to children as young as five.