Oral medications for spinal muscular atrophy are available on the NHS in England | NHS

[ad_1]

The NHS is set to introduce a revolutionary new treatment to tackle the leading genetic cause of death in infants and young children.

About 1,500 patients in England with certain types of spinal muscular atrophy (SMA) are expected to benefit from risdiplam, following a recommendation from the Health Watchdog. The drug, also called Evrysdi and made by Roche, is a syrup that can be taken at home and is the first non-injectable treatment for this condition.

SMA is a progressive neuromuscular condition that affects the nerves in the spinal cord that control movement and can cause paralysis, muscle weakness, and progressive loss of movement.

NHS England chief executive Amanda Pritchard said: “In the past three years, the NHS has revolutionized the care of people with SMA, by securing access to three innovative treatments – Spinraza, Zolgensma and now risdiplam – where doctors were three years ago. There are absolutely no effective drugs.

“Spinal muscular atrophy is a severe disease and a leading genetic cause of death in infants and young children, which is why NHS England has decided to make these treatments available to people as soon as possible to help transform lives for patients and their families.”

Risdiplam was initially recommended for limited use for people with certain types of SMA as part of a deal known as a managed access agreement between NHS England and Roche, according to the National Institute for Health and Care Excellence (NICE). Children under the age of two months will be among the first beneficiaries.

The list price for risdiplam is £7,900 per 80ml vial, but a deal has been struck at a discounted price to make it available to the NHS.

Dr Elizabeth Raig, consultant pediatric neurologist at Evelina Children’s Hospital London, part of Guy’s and St Thomas’ NHS, said the development was “excellent news”.

“This will be especially important for those with SMA who cannot receive either of the two current treatments, Spinraza and Zolgensma. These are very exciting times and I am sure this news will be welcomed by those families and individuals affected by SMA. As well as by their doctors.”

The watchdog is pleased to recommend an “appropriate oral treatment for people with SMA that can be given at home,” said Mendert Boessen, executive vice president at Nice.

He said: “Not only would this be less stressful and therefore would have a positive impact on the lives of both people with SMA and their caregivers, but it would also reduce the requirements for administering treatment for the NHS.

“In practice, the availability of the drug by mouth should increase adherence to treatment, along with providing access to treatment for those who cannot have other currently recommended options.”

The mother of a nine-year-old boy from London who was the first patient from the UK to receive Risedlam through a clinical trial said the change she saw was “absolutely amazing”.

His mother, Yves Cherdatisak, said Melville-Vedrine Cloquet, who started taking the drug when he was five, has shown “really positive results” and could look forward to a “brighter future.”

She added: “It’s great for other children and their families that it will now be available on the NHS, because when it comes to diagnosing SMA, time is of the essence. Children are not going to get back what they have lost, so the sooner they get the right medicines, the better.”

[ad_2]