SCD patients can access Casgevy via England’s public health system
Patient access granted in reimbursement deal between marketer, NHS
Vertex Pharmaceuticals has entered a reimbursement agreement with the National Health Service in England that will enable eligible sickle cell disease (SCD) patients in that country to get access to treatment with Casgevy (exagamglogene autotemcel) via the public health system.
“[This] agreement means eligible sickle cell disease … patients in England now have access to Casgevy,” Vertex said in a company press release.
The deal comes after a positive decision from the National Institute for Health and Care Excellence, or NICE — an independent organization providing guidance on health and social care in England — that recommended Casgevy’s reimbursement. It also follows a prior agreement for treatment reimbursement for people with transfusion-dependent beta-thalassemia, known as TDT.
Last year, NICE preliminarily recommended against reimbursement for the approved therapy due to uncertainties regarding its cost-effectiveness. However, following a consultation period in which patients, healthcare professionals, and advocates could respond to the draft decision, NICE ultimately decided to support Casgevy’s reimbursement.
The agreement was heralded by Vertex, which markets the therapy.
“Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease,” said Ludovic Fenaux, senior vice president at Vertex International.
Casgevy shown to reduce, eliminate pain crises in SCD
Both SCD and TDT are marked by a lack of functional hemoglobin, the protein responsible for transporting oxygen in red blood cells.
In SCD, mutations in the HBB gene result in the production of a faulty version of hemoglobin that tends to clump up inside red blood cells, causing them to acquire the sickle-like shape that gives the disease its name. These defective red blood cells tend to die prematurely and may obstruct blood vessels, leading to anemia, vaso-occlusive crises (VOCs), and other SCD symptoms. In TDT, little or no hemoglobin is produced.
Casgevy is a one-time treatment that uses a gene-editing technology called CRISPR-Cas9 to increase the production of fetal hemoglobin. This version of hemoglobin is more effective at carrying oxygen than its adult counterpart. Fetal hemoglobin is normally produced during fetal development, but its production typically switches off sometime after birth.
The treatment involves collecting a patient’s hematopoietic stem cells — stem cells in the bone marrow that can give rise to all types of blood cells — and editing them in a lab to enable the production of fetal hemoglobin. The modified cells are then returned to the patient via a stem cell transplant. Once transplanted, the engineered cells are expected to multiply and give rise to new red blood cells that are capable of producing fetal hemoglobin.
Casgevy was shown to reduce or eliminate VOCs for SCD patients and to reduce the need for blood transfusions for those with TDT.
We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with Casgevy, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system.
In the U.K., the treatment was conditionally approved in 2023 for people ages 12 and older, who have SCD and recurrent VOCs, or TDT, and are eligible for a stem cell transplant, but lack an available donor. Casgevy also is approved in the U.S. for patients with SCD or TDT, ages 12 and older.
Because the administration of Casgevy requires experience in stem cell transplants and management of SCD, the company is engaging with experienced hospitals and establishing a network of certified medical centers with appropriate experience and training, according to the release.
“We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with Casgevy, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system,” Fenaux said.
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