Vertex and CRISPR Therapeutics Gain FDA Approval for Gene-Editing Therapy for Sickle Cell Disease

Vertex Pharmaceuticals and CRISPR Therapeutics have announced that the FDA has approved their CRISPR/Cas9 genome-edited cell therapy, to be marketed as Casgevy, for the treatment of sickle cell disease (SCD) in patients 12 years and older. 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.

Vertex leads global development, manufacturing and commercialization of Casgevy with support from CRISPR Therapeutics. In conjunction with the FDA approval of Casgevy, Vertex will make a $200 million milestone payment to CRISPR.

SCD is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” red blood cells. The clinical hallmark of SCD is recurrent vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain. In the U.S., the median age of death for patients with SCD is approximately 45 years.

Prior to Casgevy, a cure for SCD would be a stem cell transplant from a matched donor, but this option is only available to a small fraction of patients because of the lack of available donors. In contrast, Casgevy is made individually for each patient, using the patient’s own edited blood stem cells.

Casgevy was granted a conditional marketing authorization in Great Britain by the U.K. Medicines and Healthcare products Regulatory Agency and by the National Health Regulatory Authority in Bahrain for patients 12 years of age and older with SCD characterized by recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia (TDT). Casgevy is currently under review by the European Medicines Agency and the Saudi Food and Drug Agency for both SCD and TDT.

The use of Casgevy for the treatment of 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 (PDUFA) target action date of March 30, 2024. (Source: Vertex Website, 08 December, 2023)