{"id":6390,"date":"2024-08-08T15:03:57","date_gmt":"2024-08-08T15:03:57","guid":{"rendered":"https:\/\/dailywashingtoninsider.com\/index.php\/2024\/08\/08\/gene-therapy-costing-over-1-6m-is-approved-for-nhs-patients-with-inherited-blood-disorder\/"},"modified":"2024-08-08T15:03:57","modified_gmt":"2024-08-08T15:03:57","slug":"gene-therapy-costing-over-1-6m-is-approved-for-nhs-patients-with-inherited-blood-disorder","status":"publish","type":"post","link":"https:\/\/dailywashingtoninsider.com\/index.php\/2024\/08\/08\/gene-therapy-costing-over-1-6m-is-approved-for-nhs-patients-with-inherited-blood-disorder\/","title":{"rendered":"Gene therapy costing over \u00a31.6m is approved for NHS patients with inherited blood disorder"},"content":{"rendered":"

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.<\/p>\n

The treatment, called Casgevy, uses ‘molecular scissors’ to disable a faulty gene that causes the debilitating condition beta-thalassaemia.<\/p>\n

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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.<\/p>\n

But in trials 90% of people were effectively cured by a single treatment with Casgevy.<\/p>\n

The National Institute for Health and Care Excellence (NICE) said in new final draft guidance that Casgevy, also known as exa-cel, will immediately be made available on the NHS<\/strong> to treat severe beta-thalassaemia while further evidence is collected on its cost-effectiveness.<\/p>\n

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Treatment may be a ‘potential cure’<\/strong><\/p>\n

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.”<\/p>\n

In the UK beta-thalassaemia mainly affects people of Pakistani, Indian and Bangladeshi ethnic origin.<\/p>\n

It can cause delayed growth, bone and hormonal problems, and affect quality and length of life.<\/p>\n

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 \u00a31,651,000.<\/p>\n

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.<\/p>\n

Therapy uses gene-editing ‘scissors’<\/strong><\/p>\n

People with beta-thalassaemia are unable to make enough haemoglobin, the protein in red blood cells that carries oxygen.<\/p>\n

To correct the problem with gene therapy doctors first remove stem cells that make red blood cells.<\/p>\n

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.<\/p>\n

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.<\/p>\n

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.<\/p>\n

“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.”<\/p>\n

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.<\/p>\n

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.<\/p>\n

“This groundbreaking therapy must also be funded for people with sickle cell, where it has huge promise and is desperately needed,” said Ms Sheikh.<\/p>\n

“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.”<\/p>\n<\/p>\n

This post appeared first on sky.com<\/div>\n

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