Oral drug for spinal muscular atrophy to be available on NHS in England

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

About 1,500 patients in England with certain types of spinal muscular atrophy (SMA) are expected to benefit from risdiplam, after 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 the condition.

SMA is a progressive neuromuscular condition affecting the nerves in the spinal cord controlling movement and can cause paralysis, muscle weakness and progressive loss of mobility.

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.”

Meindert Boysen, the deputy chief executive of NICE, 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.”

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Source: The Guardian, 19 November 2021