Patients in the United Kingdom will soon be able to receive a novel medication for sickle cell disease.
The treatment is the first in over 20 years, according to the United Kingdom’s National Health Service (NHS).
Sickle cell disease is a disorder that causes red blood cells to harden, cling together, and form a crescent shape. It is particularly frequent in Africans and Caribbeans.
When cells pass through narrow blood channels, they become caught and obstruct the flow, resulting in pain.
In addition, sickle cells die early, resulting in a chronic lack of red blood cells.
Sickle cell patients experience intense pain known as a ‘sickle cell crisis,’ which can happen many times a year and necessitate hospital visits or admission.
Novartis’ new medicine, Crizanlizumab, is injected into a vein and can be used alone or in conjunction with normal treatment and blood transfusions.
It prevents the restriction of blood and oxygen delivery that leads to a sickle cell crisis by attaching to a protein in blood cells.
According to the NHS, the drug will cut the number of times a sickle cell patient needs to go to A&E by “two fifths.”
People aged 16 and over who suffer from multiple sickle cell crises per year will be eligible for the treatment.
UK’s National Institute for Health and Care Excellence (NICE), which recommended the drug be available on the NHS, said it could not recommend the drug for routine use yet owing to high uncertainty about the long-term effectiveness of the treatment and its associated costs.
But the agreement with the NHS will allow patients to access the drug while extra data is collected through clinical trials.
Amanda Pritchard, chief executive of NHS England, said the drug will help as many as 5,000 people over the next three years to have a much better quality of life.
“This is a historic moment for people with sickle cell disease who will be given their first new treatment in over two decades,” she said.
“This revolutionary treatment will help to save lives, allow patients to have a better quality of life and reduce trips to A&E by almost half.”
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