Around 1,500 people in England with the rare genetic disorder spinal muscular atrophy (SMA) are set to benefit from a new treatment following a commercial deal struck between the NHS England and NHS Improvement and the company Roche.
They entered a managed access agreement (MAA) and the National Institute for Health and Care Excellence (NICE) published a draft guidance for the drug on Friday (November 19).
The life-changing drug Risdiplam will be made available on the NHS to treat hundreds of SMA patients a year, NHS chief executive Amanda Pritchard said.
Risdiplam is the first non-injectable treatment for SMA, a rare and often fatal genetic disease that can cause paralysis, muscle weakness and progressive loss of movement.
The drug improves motor function and can be taken easily at home. it can be used to treat babies as young as two months.
Pritchard said: “It is yet another example of the NHS leading the way in securing access to the newest and most innovative treatments and therapies available for patients, not only for those with rare genetic conditions, but in all areas including fighting cancer and Covid19 in line with the NHS Long Term Plan”.
At least 215 people in England have already had Risdiplam via the early access scheme provided earlier this year.
As part of the deal the drug will be made available to the health service at a fair price, which otherwise has a list price of nearly £8,000 per dose.
Expressing pleasure over reaching the deal, Richard Erwin, general manager, Roche Products Limited said: “We remain committed to ensure that this treatment is made available across the rest of the UK as quickly as possible”.
Over the last 18 months the NHS has sealed a series of ‘smart deals’ to secure innovative medicines for patients.
Last week, the NHS announced new agreements to expand the use of blood-thinning treatments.
Graeme Duncan 
