PTC Therapeutics has announced that NICE has issued final guidance recommending Translarna for ambulatory patients aged five and over with nonsense mutation Duchenne muscular dystrophy in connection with a managed access agreement with NHS England.

Duchenne muscular dystrophy, a rare genetic disease that primarily affects males, is a progressive muscular disorder caused by the lack of functional dystrophin protein. Disease sufferers lose the ability to walk from as early as 10 years old, and may begin to experience potentially fatal lung and heart complications within their late teens and early twenties.

Although rejected by the FDA, Translarna has been approved by the EMA. NICE had backed the drug in initial guidance, and this latest guidance will mean Duchenne patients can now access the first approved therapy to treat the underlying causes of the disease.

Paul Lenihan, CEO of Action Duchenne, says: “We are delighted that this positive recommendation from NICE has now been published. This is such an important day for the Duchenne community, which has been working hard to make this ground-breaking drug available to boys with nonsense mutation Duchenne muscular dystrophy.”

Janis Clayon, General Manager for UK and Ireland at PTC Therapeutics, comments: “Translarna is the first and only licensed treatment to address the underlying cause of Duchenne muscular dystrophy. I am very pleased that the completion of this important review process undertaken by NICE and NHS England now ensures that a much-needed therapy is available for the children and young adults with nonsense mutation DMD that meet the requirements for the MAA.”

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