Patients in the U.K. with transfusion-dependent beta-thalassemia will have access to Vertex Pharmaceuticals’ gene editing therapy Casgevy, thanks to an agreement with the National Health Service on the price.
Vertex Pharmaceuticals announced Wednesday that it has inked a reimbursement agreement with the U.K.’s National Health Service on its CRISPR-based gene editing therapy Casgevy (exagamglogene autotemcel) for patients with transfusion-dependent beta-thalassemia.
According to Vertex, the reimbursement deal comes after the National Institute for Health and Care Excellence (NICE) issued a recommendation for Casgevy in this indication. NICE is an executive and non-departmental government agency in the U.K. that issues recommendations and quality standards regarding the health services and drugs. The National Health Service (NHS) uses NICE guidance to determine which treatments to offer patients.
Ludovic Fenaux, senior vice president at Vertex International, in a statement said that the reimbursement deal with NHS is a “historic moment” for patients living with transfusion-dependent beta thalassemia in the U.K., who have long suffered from “limited options for this life-shortening disease.”
“Through collaboration with NHS England and NICE, we have reached this landmark agreement that recognizes the value a one-time treatment can provide to patients, their families and the healthcare system,” Fenaux said.
Casgevy will be offered at seven specialist NHS facilities across England and will be available within weeks, according to the agency’s news release. In total, there are approximately 460 patients in England with transfusion-dependent beta thalassemia who are at least 12 years of age and are potentially eligible for Casgevy treatment.
NHS England is one of the first healthcare systems in the world to provide access to Casgevy, the agency said.
Casgevy is a one-time gene-edited therapy that uses CRISPR technology to modify a patient’s own CD34-positive hematopoietic stem cells to reduce expression levels of the BCL11A gene. The treatment boosts the production of fetal hemoglobin, which is otherwise impaired in sickle cell disease and beta-thalassemia.
In November 2023, the U.K.’s Medicines and Healthcare Products Regulatory Agency became the first health authority to sign off on the use of Casgevy, allowing its use in both blood disorders. The FDA followed suit soon after, granting approval for sickle cell disease in December 2023 and beta-thalassemia in January 2024.
Vertex and its partner CRISPR Therapeutics gave Casgevy a list-price of $2.2 million.
In March 2024, Vertex failed to convince NICE of Casgevy’s value in sickle cell disease (SCD). The agency at the time refused recommend funding the gene-editing therapy on the grounds of cost-effectiveness.
Vertex on Wednesday said it is working with reimbursement authorities in the European Union to bring the therapy to eligible sickle cell disease and beta-thalassemia patients.
