Patients in England with an inherited blood disorder are to become the first in Europe to benefit from a gene therapy costing well over one million pounds a shot.
The treatment, called Casgevy, uses ‘molecular scissors’ to disable a faulty gene that causes the debilitating condition beta-thalassaemia.
Currently people severely affected by the disease need blood transfusions every few weeks because their own red blood cells are unable to carry enough oxygen around the body.
But in trials 90% of people were effectively cured by a single treatment with Casgevy.
The National Institute for Health and Care Excellence (NICE) said in new final draft guidance that Casgevy, also known as exa-cel, will immediately be made available on the NHS to treat severe beta-thalassaemia while further evidence is collected on its cost-effectiveness.
Treatment may be a ‘potential 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 exa-cel could represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions.”
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In the UK beta-thalassaemia mainly affects people of Pakistani, Indian and Bangladeshi ethnic origin.
It can cause delayed growth, bone and hormonal problems, and affect quality and length of life.
Last March NICE ruled in its initial guidance that there wasn’t enough evidence of clinical benefit to justify the cost of Casgevy, which has a list price of £1,651,000.
But the manufacturer Vertex has agreed a confidential discount and the treatment is being made available for up to 460 patients over the age of 12 through the Innovative Medicines Fund to study the benefits further.
Therapy uses gene-editing ‘scissors’
People with beta-thalassaemia are unable to make enough haemoglobin, the protein in red blood cells that carries oxygen.
To correct the problem with gene therapy doctors first remove stem cells that make red blood cells.
In the lab they use a Noble Prize-winning technique called CRISPR to make precise cuts in the faulty gene, then transfuse the edited cells back into patients.
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A clinical study showed that 90% of 42 patients given the treatment didn’t require a red blood cell transfusion for at least 12 months. The rest needed fewer transfusions.
Yasmin Sheikh, from the stem cell charity Anthony Nolan, said: “We’re delighted with this historic decision to approve the UK’s first ever CRISPR-based therapy.
“Casgevy offers an effective cure for transfusion-dependent beta thalassaemia – a debilitating condition that was previously incurable in patients who don’t have a stem cell donor.”
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However, the charity urged NICE and the manufacturer Vertex to agree a deal allowing Casgevy to also be used for sickle cell, another inherited blood disorder.
The UK medicines regulator said it was a safe and effective treatment for the disease last year, but it is awaiting clearance from NICE before being allowed on the NHS.
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“This groundbreaking therapy must also be funded for people with sickle cell, where it has huge promise and is desperately needed,” said Ms Sheikh.
“We hope this approval for thalassaemia demonstrates a solution is possible, and urge NICE and Vertex to work together to deliver this treatment to patients with sickle cell.”
