‘Life-changing’ drug for rare spine condition now on NHS

BABIES and young children with a rare spinal condition can access a “life changing” at-home treatment on the NHS for the first time.

Patients with spinal muscular atrophy (SMA), an often fatal genetic disease that causes paralysis and muscle weakness, will have access to risdiplam, a medicine taken as a syrup once a day.

The conditions affects about one in every 6,000 to 10,000 births. More than two thirds (68 per cent) of children with type 1 SMA die before their second birthday and 82 per cent die before their fourth.

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be able to access the treatment after the National Institute for Health and Care Excellence recommended the drug in draft guidance.

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

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

Another SMA treatment, zolgensma, which has a list price of £1.79million a dose, was made available on the NHS in March. Spinraza, the first drug to help infants with SMA, was made available in 2019.

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

Liz Ryburn, from SMA UK, said that the charity was “absolutely delighted”.

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