Life-changing mobility drug for spinal muscular atrophy patients to be available on NHS

A medicine which aims to boost the mobility of people with spinal muscular atrophy (SMA) will soon be provided on the NHS, it has been announced.

The rare genetic condition, which gets worse over time, inhibits movement by causing muscle weakness.

Risdiplam, whose cost price is almost £8,000 per 80ml bottle, will become available to roughly 1,500 SMA sufferers across the country, after NHS England struck an agreement with its manufacturer, the pharmaceutical giant Roche.

The deal ensures it can be provided “at a price that is fair for taxpayers”, according to the health service.

However, due to its price, the National Institute for Health and Care Excellence (Nice) does not currently recommend it for routine commissioning.

The drug, which is taken as a syrup, is the first non-injectable SMA treatment and can be given to babies as young as two-months-old, enhancing their motor function by increasing protein production in nerve cells that are integral to muscle movement.

It is the third SMA treatment in as many years to be given NHS approval. One of the drugs which also recently got the green light was Zolgensma, which costs £1.79m per patient.

Amanda Pritchard, chief executive of NHS England, hailed the progress that had been made regarding the condition.

“In the last three years the NHS has revolutionised care for people with SMA, by securing access to a trio of innovative treatments - Spinraza, Zolgensma and now risdiplam - where three years ago clinicians had no effective medicines at all,” she said.

Melvil Vedrenne-Cloquet, a 9-year-old Londoner, was the first SMA patient to receive risdiplam in a clinical trial. A total of 215 patients in England have now had the drug as part of an early access scheme.

His mother Eve said it was “absolutely amazing” how much he had improved.

“Now, since taking risdiplam, we have seen really positive results and my son faces a brighter future. It’s fantastic for the other children and their families out there that it will now be available on the NHS, because when it comes to a diagnosis of SMA, time is of the essence.

“Children will never get what they have lost back, so the faster they can get the right medicines the better,” she said.

Liz Ryburn, from SMA UK, also expressed her delight that SMA patients will now have access to a  medication that can be taken at home.

“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,” she said.

Meanwhile, Dr Elizabeth Wraige, consultant paediatric neurologist at Evelina London Children’s Hospital, said the drug would be “especially important” for those who are unable to receive either Spinraza or Zolgensma.

There are four types of SMA, which start at different ages, with the most severe being SMA 1, which develops in babies which are less than six months old.