Treatment for Duchenne muscular dystrophy accepted by Scottish Medicines Consortium 12 April 2021 A drug called Translarna can help treat an underlying cause of Duchenne muscular dystrophy Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group, and Duchenne UK appeared before the Scottish Medicines Consortium in March The Scottish Medicines Consortium has accepted the drug for use on NHSScotland over the next three years The drug company must now submit a plan to Scottish Government before it can be given on NHSScotland 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.” Notes to editors For more information on Muscular Dystrophy UK please contact: Alice Cachia - Senior Press Officer 07771 374 839 [email protected] For more information on Action Duchenne please contact: Neil Bennett – Head of Research 020 7250 8240 For more information on Duchenne Family Support Group please contact: [email protected] 0800 121 4518 For more information on Duchenne UK please contact: Sarah Johnson – Communications Manager [email protected] About Duchenne muscular dystrophy Duchenne muscular dystrophy is a life-shortening muscle-wasting condition, caused by the lack of a vital muscle protein called dystrophin. The condition causes muscles to weaken and waste over time, leading to increasing and severe disability. Most of those affected will use a wheelchair by the age of 12 and will face life-threatening health problems by their late teens as their heart and breathing muscles weaken. Few of those born with the condition currently live to see their 30th birthday. Previous and next steps This is the second time a submission has been made to the SMC to approve Translarna, following an earlier rejection in 2016. You can read more about this here. Translarna was subsequently made available by Health Boards on an individual patient treatment request basis. Today’s announcement does not mean Translarna will be made routinely available on NHSScotland. Instead, it will be available for the next three years under an ultra-orphan pathway if the drug manufacturer’s plans are accepted by the Scottish Government.