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Monday, November 29, 2021

Children with rare spinal condition to receive ‘life changing’ treatment on the NHS

For the first time, around 1,500 people with the disorder will be able to access the new treatment after the National Institute for Health and Care Excellence (Nice) recommended the drug in draft guidance.

Risdiplam is licensed to treat people with types 1, 2 and 3 SMA and clinical evidence has shown it improves the ability to sit up, stand or walk. It may also be effective for people before they begin to show symptoms of the disease. 

Some evidence also suggests people with type 1 SMA live for longer when taking the drug.

‘We’re absolutely delighted’

It comes after another SMA treatment, zolgensma, which has a list price of £1.79 million per dose, was made available on the NHS in March. 

In 2019 spinraza, the first drug to help infants with SMA, was made available.

This new treatment has a list price of nearly £8,000 per 80ml vial, but NHS England and manufacturer Roche have struck an access deal that is “fair for taxpayers”. The cost per dose has not been disclosed due to commercial reasons.

Liz Ryburn, from SMA UK,said: “We’re absolutely delighted that this third treatment for spinal muscular atrophy will now be available in England. It offers children, young people and adults who live with this progressive and complex neuromuscular condition, an oral medication that can be taken daily at home to combat the deterioration they otherwise face.

“Many are powerchair users who have shielded throughout the pandemic and face huge challenges with travel to hospital for treatment. Risdiplam offers so much more flexibility in people’s lives.”

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