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Spinal muscular atrophy treatment approved despite "uncertainties"

Friday November 19th 2021

The NHS today promised a new treatment for hundreds of patients with spinal muscular atrophy after regulators approved the use of a syrup medicine drug, said to improve motor function.

The drug, risdiplam, is the first non-injectable treatment for the disease, although two other drugs have been approved in the last three years.

The drug modifies the SMN2 gene to increase quantities of the SMN protein. It is licensed to treated types 1,2 and 3 SMA or pre-symptomatic disease.

The National Institute for Health and Care Excellence, issuing draft guidance, said there remained “uncertainties” about the treatment – but approved the use of the syrup while more data is collected. NICE said it remained concerned about the cost effectiveness of the treatment.

Its deputy chief executive Meindert Boysen said: “We are pleased that there will now be the option of a convenient oral treatment for people with SMA that can be administered at home. 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.”

NHS chief executive Amanda Pritchard said: “SMA 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 this treatment available to people as soon as possible to help transform the lives of patients and their families.”

[Draft Guidance]

Tags: Brain & Neurology | Child Health | NHS | UK News

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