New hope for sickle cell disease as first new drug in 20 years is approved

A “revolutionary” life-changing drug treatment for sickle cell disease is to be made available on the NHS – the first new treatment for the disease in 20 years.

It will initially only be made available to around 300 patients but will be extended to 450 in future years after the decision by the National Institute for Health and Care Excellence (Nice), which recommended the drug be available on the NHS.

Sickle cell disease is a serious and lifelong health condition causing severe pain and organ failure often requiring hospital admissions.

The new drug, known as crizanlizumab, will be delivered by a transfusion drip and works by binding to a protein in the blood cells to prevent the restriction of blood and oxygen supply.

It could help as many as 5,000 people over the next three years, the NHS has said. It is hoped the drug will reduce the number of times a sickle cell patient needs to go to A&E by two fifths.