The first new treatment for sickle cell disease in over 20 years is set to be rolled out to thousands of patients, NHS England has announced.
People with the condition endure severe pain during a ‘sickle cell crisis’ that can occur multiple times per year, often requiring hospital admission so they can be given morphine to control the pain and prevent organ failure which can be fatal. The drug crizanlizumab (produced by Novartis under the name Adakveo) 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 that lead to a sickle cell crisis.
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‘This is a historic moment for people with sickle cell disease who will be given their first new treatment in over two decades,’ said NHS chief executive Amanda Pritchard.
‘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. The NHS has agreed a deal for this drug, so we are able to provide the latest and best possible treatments for patients at a price that is affordable for taxpayers.’
This announcement paves the way for the National Institute for Health and Care Excellence (NICE) to publish final guidance on crizanlizumab which will consider the data that will be collected as part of the agreement. People aged 16 and over who suffer from multiple sickle cell crises, or vaso-occlusive crises as they are clinically known, per year will be eligible for the treatment.
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‘A new treatment brings new hope for people living with Sickle Cell Disorder, the world’s most common genetic blood condition,’ said Chair of the Sickle Cell Society, Kye Gbangbola.
‘SCD is a “medical emergency”; it causes excruciating pain, this new treatment will reduce the number of agonising pain episodes we have to endure. The hope is improved quality of life for many living with the condition and their families.’