NHS approves gene therapy for sickle cell disease

U.K. – The National Health Service (NHS) in England has approved Vertex Pharmaceuticals’ groundbreaking gene-editing therapy, Casgevy, for sickle cell disease (SCD) under a managed access scheme with a confidential discount.

This therapy, priced at £1.65 million (US $2.05 million), offers hope to patients aged 12 and older who experience at least two vaso-occlusive crises (VOCs) annually. These crises are agonizing episodes caused by misshapen red blood cells blocking blood vessels.

Casgevy, also known as exagamglogene autotemcel (exa-cel), uses CRISPR-based gene-editing technology.

The treatment involves collecting a patient’s stem cells, modifying them to produce healthy blood cells, and reinfusing them into the patient.

Unlike traditional bone marrow transplants, this therapy uses the patient’s own cells, eliminating the risk of immune rejection. It is especially valuable for those who lack suitable bone marrow donors.

The approval means that patients with SCD, alongside those with transfusion-dependent beta-thalassemia (TDT), will now have access to this life-changing therapy.

Casgevy was approved for TDT in 2023 and for SCD the following year. NICE (National Institute for Health and Care Excellence) made the decision after hearing testimonials from patients who experienced remarkable improvements.

NICE recognized that sickle cell disease disproportionately impacts people of African, Caribbean, Middle Eastern, and South Asian heritage and factored these health inequalities into its cost-effectiveness assessment of Casgevy.

The approval was granted with plans to gather additional evidence on the therapy’s long-term effectiveness over time.

Data collection under the managed-access agreement aims to address uncertainties, particularly regarding the durability of its benefits.

John James, CEO of the Sickle Cell Society, celebrated this milestone, stating, “We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today. The significance of this milestone for the sickle cell community cannot be understated.”

The Sickle Cell Society had been advocating for the inclusion of SCD in Casgevy’s scope since NICE initially declined to recommend it in March 2024.

However, James acknowledged that not all patients with SCD would be eligible for this therapy.

He added, “While today’s news is incredible, we remain acutely aware that not everyone with sickle cell will be eligible for the potentially life-changing benefits of Casgevy. Furthermore, this new therapy joins a disappointingly limited range of treatments currently available for this complex and challenging condition.”

Vertex Pharmaceuticals is working closely with experienced hospitals across England to set up a network of treatment centers capable of delivering Casgevy.