Vertex Pharmaceuticals and CRISPR Therapeutics’ one-time gene therapy has been recommended by the National Institute for Health and Care Excellence (NICE) to treat transfusion-dependent beta thalassaemia (TDT) on the NHS in England.
NICE’s final draft guidance recommends Casgevy (exagamglogene autotemcel) for patients aged 12 years and older for whom a blood and bone marrow transplant is suitable but no donor is available.
Beta thalassaemia is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body.
Patients with the most severe form of the disease develop life-threatening anaemia and have to undergo regular blood transfusions throughout their lives.
An estimated 460 people in England with TDT could be eligible for Casgevy, which works by editing the faulty gene in a patient’s own bone marrow stem cells so that the body produces functioning haemoglobin.
In clinical trials, 93% of patients with beta thalassaemia did not require a blood transfusion for at least a year after receiving the treatment, and it is hoped that the therapy could offer a lifetime cure.
Helen Knight, director of medicines evaluation at NICE, said: “Although there are some uncertainties in the evidence for its long-term benefits, the committee felt [Casgevy] could represent a potential cure for some people with TDT, freeing them from the burden and risks of needing regular blood transfusions.”
Casgevy will be available through England’s Innovative Medicines Fund, which provides faster patient access to non-cancer medicines while further data can be collected, and will be offered at seven highly specialist NHS centres across the country within weeks.
Ludovic Fenaux, senior vice president, Vertex International, said: “Securing access to Casgevy is a historic moment for people living with TDT who, for too long, have had limited options for this life-shortening disease.
“Through collaboration with NHS England and NICE, we have reached this landmark agreement that recognises the value a one-time treatment can provide to patients, their families and the healthcare system.”
Casgevy is the first treatment to emerge from Vertex and CRISPR’s strategic partnership, which was originally announced in 2015. Vertex now leads the global development, manufacturing and commercialisation of Casgevy and splits programme costs and profits worldwide with CRISPR.