Muscular Dystrophy UK celebrates milestone of ten medical treatments for muscle-wasting conditions, as new drug treatment is approved for use on NHS

  • NICE has today approved a new treatment for the progressive, muscle-wasting conditions, Pompe Disease, for routine use on NHS
  • The new drug avalglucosidase alfa (also known as Nexviazym) is one of ten medical treatments for muscle-wasting conditions now either available for NHS usage or undergoing appraisal
  • Muscular Dystrophy UK, which has supported appraisal processes, hails remarkable advancement from zero to ten treatments in just six years

The charity Muscular Dystrophy UK has warmly welcomed the announcement that a new treatment for a muscle-wasting condition has now been permanently approved by the National Institute for Health and Care Excellence (NICE).

Babies, children, young people and adults living with the rare, genetic, muscle-wasting condition, Pompe Disease, will now be able to access the treatment avalglucosidase alfa from the NHS, following the announcement by NICE on Thursday 21 July.

The new drug, also known as Nexviazym, is one of ten treatments for progressive muscle-wasting conditions that have been approved or are currently being appraised for use on the NHS in just six years.

Muscular Dystrophy UK has worked hard with NICE and the Scottish Medicines Consortium (SMC) through its Fast Track to Treatments campaign to speed up the approval process. The charity has also helped secure early access for NHS patients to important new and potentially-life-changing therapies while they are still being appraised for routine use.

Rob Burley is Director of Care, Communications and Support at Muscular Dystrophy UK, which supports people with more than 60 muscle-wasting conditions and funds scientific research to help develop medical treatments – and ultimately cures.

He said,
“This is a remarkable advancement in such a short amount of time. Just three years ago there was only one treatment available for one group of people living with a muscle-wasting condition and one more going through the treatment appraisal process.

“Muscular Dystrophy UK is proud to be engaging with both NICE and the SMC to support the appraisals of these treatments and to identify and remove the barriers that cause delays. We are committed to ensuring that our community can access and benefit from new, safe and effective
treatments as quickly as possible. When you are living with a progressive muscle-wasting condition, every day counts.”

These ten new treatments include Mexiletine, which is now fully available on the NHS for
for myotonia in adults with non-dystrophic myotonic disorders, and Zolgensma, which has been approved by NICE for some babies with spinal muscular atrophy. Others, such as Spinraza and Risdiplam, which also treat spinal muscular atrophy, are available under special Managed
Access Agreements that Muscular Dystrophy UK have helped secure.

People living with Pompe disease have welcomed the approval of avalglucosidase alfa, which has been developed by Sanofi Genzyme. Clinical trials have found that the enzyme-replacement-therapy delayed the development and reduced the progression of symptoms of the condition.

Baroness Thomas of Winchester is a Trustee of Muscular Dystrophy UK and lives with late-onset Pompe disease herself.  The Lib-Dem peer was a Patient Expert during the NICE appraisal of avalglucosidase alfa and she gave evidence to the NICE committee that approved the drug.

“Pompe disease has a huge impact on my life; I now use an electric wheelchair, and a ventilator at night and for a few hours during the day. Speech is difficult and I eat very little, relying on an overnight food supplement through a percutaneous endoscopic (PEG), a procedure in which a flexible feeding tube is placed through the abdominal wall and into the stomach

“I am delighted that some people with Pompe disease will now have access to this new treatment and the opportunity to have a different experience from my own. I am extremely proud of Muscular Dystrophy UK’s role in both supporting people living with muscle-wasting conditions and advocating for them to have access to the growing number of treatments that are becoming available.”

Finley 1Finley Davies, 9, lives with spinal muscular atrophy (SMA) and was one of the first children to benefit from these life-changing treatments. Finley has received two treatments for his condition, Spinraza and Risdiplam.

Finley has been receiving Risdiplam after trying Spinraza at first. Although Spinraza gave him much more strength than he had previously, his mum, Rosie, noticed that the lumbar punctures that are needed for the treatment was taking a toll on his mental wellbeing. Risdiplam has been much more successful for Finley, allowing him to enjoy being a little boy for the first time in his life.

“It’s incredible to think that there are a range of treatments available for SMA when there was nothing at all for years,” said Rosie. “He is finally able to live the life of a 9-year-old schoolboy and we are able to live without so much sadness and worry,” she added.

Thanks to the treatment, Finley now has much more strength and independence. He can now use a powered wheelchair that wouldn’t have been possible without the treatment.

“He is really excited about having a new, faster, more efficient chair, and this would not have been possible without his strength and energy levels that have been maintained by the treatment he has been able to access,” said Rosie.

The ten treatments are:
Nexviazyme (also known as Avalglucosidase alfa)
Zolgensma
Mexiletine
Spinraza
Risdiplam
Translarna
Fordadistrogene movaparvovec
Vyvgart
Ravvlizumab
Cipaglucosidase alfa with miglustat

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