Accelerating drug development

Treatment for Duchenne muscular dystrophy accepted by Scottish Medicines Consortium

A drug that can treat an underlying genetic cause of Duchenne muscular dystrophy, a muscle-wasting condition, has been accepted for use on NHSScotland, it was announced today (12 April).

This will pave the way for the drug to be made available across the next three years, through a system called the ‘ultra-orphan pathway’.

Today’s exciting news comes after Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group and Duchenne UK appeared before the Scottish Medicines Consortium (SMC) last month, following an earlier joint submission to accept Translarna for use on NHSScotland.

The drug can help treat patients with an underlying genetic cause of Duchenne muscular dystrophy (DMD) called nonsense mutations. This condition primarily affects boys and causes muscles to weaken and waste over time, with most of those affected needing to use a wheelchair by the age of 12. The life-limiting disease also causes other severe health problems to the heart and breathing muscles.

In today’s announcement, the SMC said it accepts that Translarna meets the definition of what is known an ultra-orphan medicine, used to treat extremely rare conditions. It means that eligible DMD patients aged two or over should be able to have the treatment on NHSScotland under the ultra-orphan pathway for at least the next three years. This is provided that the company manufacturing Translarna submits a data collection plan to the Scottish Government.

The plan should outline how further data, including evidence outlining the experiences of patients and carers, will be collected over the next three years. At the end of this period, the drug company should provide the SMC with an updated submission for reassessment. The SMC will then review the evidence before deciding if Translarna can be routinely used on NHSScotland.

In today’s announcement, the SMC acknowledged that Translarna “could potentially bring substantial benefits to both the individual and their family. For children with DMD, it could mean greater independence and inclusion in education and social life. It could also mean greater independence for family and carers”.

More information on the SMC’s announcement can be read here.

Provided that the drug company has submitted its plans to the Scottish Government, Translarna will be given in addition to the current standard treatments on NHSScotland. Families and individuals should speak with their clinician to find out more about eligibility. More information will be shared with relevant patient groups accordingly.

A spokesperson on behalf of the four charities said: “Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group and Duchenne UK are delighted the SMC has accepted Translarna for use on the ultra-orphan pathway for the next three years. Today’s announcement is excellent progress in our bid to help those with Duchenne muscular dystrophy access appropriate treatment in Scotland, especially because every day is a race against time to slow down the condition. We believe that the physical and mental health benefits of Translarna have the potential to improve lives for so many families living with Duchenne. The four charities are therefore very much looking forward to hearing the drug company’s plans going forwards, so that patients can start to access treatment as quickly as possible.”

Published on 13 April 2021

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Categories Accelerating drug development Policy and campaigning