Friday, 19 April, 2024
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UK regulators scrap new sickle cell drug

The decision by British regulators to end the roll-out of a medication launched in 2022 – the first new drug in 20 years to treat sickle cell disease – has been slammed by campaigners.

The Independent reports that the decision to scrap the use of Adakveo (crizanlizumab) was made after an internal review, according to the Medicines and Healthcare Products Regulatory Agency (MHRA), an agency within the Department of Health and Social Care, which said that although no safety concerns were identified, it had concluded the drug was “not sufficiently effective”, despite evidence suggesting that it eases symptoms for those living with the illness.

John James, from the Sickle Cell Society, said: “Given that sickle cell disorder is the fastest growing genetic disorder in the UK, with nearly 300 babies born with the condition each year, it is appalling that we have gone backwards to a reliance on only one licensed NHS treatment.”

At least 5 000 eligible sickle cell patients would now be without this treatment: the MHRA said that about 200 patients were already being treated with the drug, but that with immediate effect, no new patients would be prescribed crizanlizumab. Those already on the drug would need to discuss treatment options with their doctors, it added.

Sickle cell disease is a lifelong condition that often requires hospital admissions. It can damage organs and cause intense pain, as well as anaemia, because the blood cells cannot carry oxygen effectively around the body, leaving sufferers with tiredness and shortness of breath.

Guidance from the National Institute for Health and Care Excellence (NICE) recommended crizanlizumab as an effective treatment for reducing chronic pain while improving patients’ quality of life.

NHS England declined to comment.

NICE is currently deciding whether to approve the only other new therapies for NHS roll-out – Casgevy and Voxelotor.


The Independent article – Landmark sickle cell drug axed by regulators (Open access)


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