NICE Endorses Vertex/CRISPR's Casgevy Gene Therapy for Beta Thalassaemia

Vertex Pharmaceuticals and CRISPR Therapeutics have achieved a significant milestone with their gene therapy, Casgevy (exagamglogene autotemcel), which has been recommended by the National Institute for Health and Care Excellence (NICE) for treating transfusion-dependent beta thalassaemia (TDT) within the NHS in England. This recommendation specifically targets patients aged 12 and older who are suitable for a blood and bone marrow transplant but lack a compatible donor.

Beta thalassaemia is a genetic disorder affecting red blood cells, which are vital for oxygen transport throughout the body. Those with severe cases of this condition suffer from life-threatening anaemia and require continual blood transfusions.

An estimated 460 individuals in England with TDT could benefit from Casgevy. This therapy functions by correcting the defective gene within a patient's bone marrow stem cells, enabling the production of healthy haemoglobin. Clinical trials have shown promising results, with 93% of beta thalassaemia patients not needing blood transfusions for at least a year post-treatment, raising hopes for a potential lifetime cure.

Helen Knight, director of medicines evaluation at NICE, acknowledged the potential of Casgevy despite some uncertainties regarding its long-term benefits. She highlighted that the therapy could significantly alleviate the burden and risks associated with regular blood transfusions for TDT patients.

Casgevy will be made available through the Innovative Medicines Fund in England, which aims to expedite access to non-cancer treatments while additional data is gathered. The therapy will be offered at seven specialized NHS centers across the country in the coming weeks.

Ludovic Fenaux, senior vice president of Vertex International, hailed the approval as a historic achievement for TDT patients who have had limited treatment options for this debilitating condition. He emphasized the importance of collaboration with NHS England and NICE in reaching this crucial agreement, which acknowledges the significant value of a one-time treatment for patients, their families, and the healthcare system.

Casgevy stands as the inaugural treatment from the strategic partnership between Vertex and CRISPR, which began in 2015. Vertex now oversees the global development, manufacturing, and commercialization of Casgevy, sharing program costs and profits with CRISPR worldwide.