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Duchenne muscular dystrophy drug ataluren approved on the NHS

Ataluren (also known as Translarna) has become the first drug specifically designed to treat Duchenne muscular dystrophy (DMD) to be approved for routine prescription on the NHS.

What is ataluren?

Ataluren is a treatment for people with DMD with a nonsense mutation – a type of genetic mutation which causes DMD in approximately 13% of patients. It is manufactured by pharmaceutical company PTC Therapeutics. It has previously been available in the UK under a Managed Access Agreement – an agreement which allows new drugs to be prescribed while more data is gathered.

Who can use the drug?

The drug can be prescribed for DMD patients with the nonsense mutation, aged 2 and over, who are able to walk.

What is NICE?

NICE stands for the National Institute for Health and Care Excellence. It decides whether new treatments are cost effective and should be provided by the NHS.

What has NICE decided?

NICE has said ataluren can be prescribed on the NHS to the specific group mentioned above. Justifying its decision, it says:

“The evidence provided, along with feedback from clinicians and people with the condition, suggests that ataluren is likely to slow down disease progression and delay the loss of the ability to walk.”

NICE said any evidence for benefits later in disease progression was inconclusive:

“Evidence for improvements in later stages of the disease and improved survival with ataluren is limited and highly uncertain but ataluren may also improve outcomes once the ability to walk has been lost.”

You can find NICE’s full appraisal here.

Emily Reuben, CEO and co-founder of Duchenne UK, said:

“The landscape for families dealing with DMD looks set to change radically in the next ten years. When my son was diagnosed, there appeared to be no hope. Now clinical trials are taking place all over the world, and negotiations underway with regulators about bring new treatments to the clinic. Duchenne UK will continue to invest in every part of drug development to help bring a new future for patients with DMD.”

Published on 20 January 2023

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