The National Institute for Health and Care Excellence (NICE) has recommended a limited use of risdiplam for people with specific types of SMA. Initially babies from two months old will benefit from the drug before opening it up to a wider scope.
Spinal muscular atrophy (SMA) is currently the leading genetic cause of death in babies and young children.
The at home medication can be taken orally as it comes in a syrup liquid form making it appropriate for your children.
Over the last three years, three treatments for SMA have been approved, however this will be the first non-injectable treatment for the condition and will help over 1,500 patients across England.
Over 200 patients have already received the treatment through an NHS early access scheme.
Usually, an 80ml vial of risdiplam is £7,900 but a deal to reduce the price of the drug has been made to ensure it is available on the NHS.
The NHS England chief executive, Amanda Pritchard, said: “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.
“Spinal muscular atrophy is a cruel disease and the leading genetic cause of death among babies and young children, which is why NHS England has been determined to make these treatments available to people as soon as possible to help transform the lives of patients and their families.”
Deputy chief executive of Nice, Meindert Boysen, said the watchdog was pleased to recommend a “convenient oral treatment for people with SMA that can be administered at home”.
He said: “This will not only be less burdensome, and therefore have a positive impact on the lives of both people with SMA and their caregivers, but it will also reduce the treatment administration requirements for the NHS.
“In practical terms, the availability of an oral drug should lead to greater adherence to treatment, along with giving access to a treatment to those who aren’t able to have other currently recommended options.”
