The NHS has approved Casgevy, a groundbreaking gene-editing treatment for sickle cell disease, offering hope to thousands. Expected to help 50 patients annually, the £1.65 million therapy corrects faulty genes, significantly reducing hospital visits. The treatment will be available at NHS centers in London, Manchester, and Birmingham.
The NHS has approved a revolutionary treatment for sickle cell disease, offering new hope to patients suffering from the painful genetic condition. The gene-editing therapy, known as Casgevy (exa-cel), was initially rejected in 2024 but has now been given the green light following further evaluation. The treatment, which won the 2020 Nobel Prize for Chemistry, works by modifying a patient’s own stem cells to correct the faulty gene responsible for sickle cell disease.
The National Institute for Health and Care Excellence (NICE) estimates that approximately 1,700 patients in the UK could be eligible, with around 50 expected to receive the treatment annually. While the listed cost of the drug is £1.65 million per patient, the NHS has secured a confidential pricing agreement with the manufacturer, Vertex Pharmaceuticals.
Sickle cell disease, which primarily affects individuals of African and Caribbean descent, causes red blood cells to become misshapen, leading to severe pain, organ damage, and life-threatening complications. Patients like Lanre Ogundimu, who has endured multiple strokes and prolonged hospital stays, see the new treatment as life-changing.
Clinical trials have shown promising results, with nearly all patients avoiding hospital admissions for over three years post-treatment. NHS England Chief Executive Amanda Pritchard hailed the approval as a “transformative” moment, providing the possibility of a cure for those with severe cases. The treatment will initially be available at specialized NHS centers in London, Manchester, and Birmingham, marking a significant milestone in genetic medicine.
