A new drug to treat sickle cell disease has been unveiled in the United Kingdom, the first of such drug in 20 years.
The drug, crizanlizumab, made by Novartis, has been described as “an innovative treatment”
It is injected into a vein and can be taken on its own or alongside standard treatment and regular blood transfusions, the BBC reports.
In a trial, patients taking the crizanlizumab had a sickle-cell crisis 1.6 times a year on average, compared with nearly three times a year normally.These painful episodes, which can require hospital treatment and lead to other health complications, are caused by by sickle-shaped red blood cells blocking the small blood vessels .
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But because the trial was small and lasted only a year, it remains unknown how long the benefits last for – and that makes it difficult to judge how cost-effective crizanlizumab is.
Nevertheless, NICE, which recommends treatments in England and Wales, is recommending its use for over-16s, albeit under a special arrangement rather than routinely, on the NHS.
The UK charity Sickle Cell Society said the new treatment brought “new hope” for people living with the world’s most common genetic blood condition.