Gene therapy, Casgevy, has been recommended for use on the NHS in England for some people living with a severe form of sickle cell disease.
Casgevy, also called exa-cel, was first approved by the Medicines and Healthcare products Regulatory Agency for the treatment of sickle cell disease in patients 12 years of age and older in November 2024 (see BioNews 1216). However, in March 2024 the National Institute for Health and Care Excellence (NICE) did not approve the gene therapy for widespread NHS use. Their draft guidance indicated that more information was needed before a decision could be made. Now, NICE's independent appraisal committee has approved the use of Casgevy on the NHS in England.
'Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications as well as addressing NICE's aim of reducing health inequalities associated with the condition and getting the best care to patients fast,' said Dr Samantha Roberts, NICE chief executive.
Sickle cell disease is an inherited blood disorder caused by a mutation in a single gene that codes for haemoglobin, an essential blood molecule that helps red blood cells carry oxygen around the body. The disease is more prevalent in people of African descent, and results in the red blood cells of patients adopting a sickle shape, rather than the disc shape of healthy red blood cells. The most common symptom of sickle cell disease is when blood vessels become blocked causing severe pain and organ damage, known as sickle cell crises.
Currently, the main treatment for sickle cell disease is a stem-cell transplant, although it is often difficult to find a donor. Now, Casgevy will be available with managed access – meaning that only people with a severe form of the disease and for whom a stem cell transplant donor can not be found will be eligible for treatment.
Blood stem cells are taken from the patient, which are then edited using CRISPR/Cas9 to precisely cut a strand of DNA at a specific site on the faulty haemoglobin gene. In the edited cells the haemoglobin gene can be transcribed again, enabling the production of a functioning gene. The edited stem cells are then transplanted back into the patients, who are then able to produce functioning haemoglobin, which can diminish the effects of the faulty haemoglobin that causes sickle cell disease symptoms.
Funmi Dasaolu, who has sickle cell disease, told The Times: 'Today is a momentous day for those living with or affected by sickle cell disease. After months of campaigning, I'm overjoyed and so very grateful exa-cel has been finally approved. It will be truly transformative for patients and offers us the chance of a life without this terrible condition; a chance to grow old, to fulfil our dreams and to live a pain-free life.'
Casgevy was recommended for use on the NHS in England for people living with a severe form of beta-thalassaemia in August 2024 (see BioNews 1251).
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