Tag: Translarna

Treatment for Duchenne Muscular Dystrophy accepted by Scottish Medicines Consortium

·       A drug called Translarna can help treat an underlying genetic 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 NHS Scotland

A DRUG that can treat an underlying genetic cause of a muscle-wasting condition has been accepted for use on NHSScotland, it has been announced.

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’.

The 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 yesterday’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 and who are able to walk 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 the announcement, the SMC acknowledged that Translarna “may allow affected children to retain the ability to walk for longer, potentially leading to increased independence”.

More information 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.”

Charities urge SMC to approve treatment for life-limiting condition

A group of charities is urging for the approval of a drug that can treat an underlying genetic cause of a life-limiting muscle-wasting condition.

Duchenne muscular dystrophy (DMD) 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 condition also causes other severe health problems to the heart and breathing muscles.

The drug Translarna, however, can help treat cases of a version of Duchenne that is caused by nonsense mutations.

Today, Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group and Duchenne UK are appearing before the Scottish Medicines Consortium (SMC) to represent patients who could benefit from Translarna and to seek its approval for use on the NHS in Scotland.

In its joint submission to the SMC ahead of today’s meeting, the charities outlined evidence from a survey conducted earlier this year.

They provided evidence that highlights clear improvements to quality of life reported by those receiving the drug. Significantly, many families of children receiving Translarna say they retained the ability to walk for longer than expected, and that this gave their child greater independence to complete daily tasks and take part in activities with friends.

Families also reported huge behaviour improvements at school and home, as well as positive mental health outcomes.

If SMC approves the submission, Translarna would be the first treatment routinely available through the NHS in Scotland that addresses an underlying genetic cause of Duchenne muscular dystrophy.

One parent who took part in the survey, and whose thoughts were included as evidence in the submission, explained: “For our son knowing that he has a medication to help his muscles is a massive boost to him.

“We believe that every time we receive our Translarna it lifts his spirits in this fight. We never for a moment take for granted how lucky we are to be receiving this drug and feel it’s something in our corner as we face this battle head on.”

If approved, Translarna would be given in addition to the current standard treatments on the NHS in Scotland.

A spokesperson for the four charities said: “Every day is a race against time to slow down Duchenne muscular dystrophy, a progressive condition that robs children of their ability to walk and their independence.

“Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group and Duchenne UK are pleased to have represented the voices of patients in our joint submission to the SMC.

“We believe our submission highlights both the physical and mental health benefits of Translarna, a drug that has the potential to improve lives for so many families living with Duchenne. We very much look forward to hearing the SMC committee’s thoughts on our submission.”