Rare muscular dystrophy drug to be funded in England and Wales

Duchenne muscular dystrophy

The National Institute for Health and Care Excellence (NICE) has published its Final Guidance recommending Translarna (ataluren) for reimbursement and use across the National Health Service (NHS) in England and Wales.  

Translarna is the only approved treatment for patients with nonsense mutation Duchenne muscular dystrophy aged two years and older who can walk. 

The final Guidance allows patients to continue treatment on ataluren and does not include a formal stopping rule, which is a criterion used to identify when someone should stop receiving a treatment.  

“The published final NICE Evaluation and agreement with the NHS provide critical access to Translarna for newly diagnosed and existing patients with nonsense mutation Duchenne in England and Wales,” said Jesse Sibarium, Senior Vice President and General Manager – EMEA, PTC Therapeutics.  

“The decision to not include a formal stopping rule in the Evaluation is positive. It recognises points raised by Action Duchenne, MDUK and other patient and clinical experts, in evidence to NICE, that clinicians would want the option to continue using Translarna after their patients lose the ability to walk because of the potential ongoing therapeutic benefits seen.” 

Duchenne is a severe progressive disease that leads to rapidly worsening muscle function with children often using a wheelchair by early adolescence and eventually requiring artificial ventilation to breathe. 

The NICE recommendation was based on data from clinical trials and real-world evidence demonstrating Translarna’s potential to slow disease progression and improve patient outcomes.

Related Articles

Join FREE today and become a member
of Drug Discovery World

Membership includes:

  • Full access to the website including free and gated premium content in news, articles, business, regulatory, cancer research, intelligence and more.
  • Unlimited App access: current and archived digital issues of DDW magazine with search functionality, special in App only content and links to the latest industry news and information.
  • Weekly e-newsletter, a round-up of the most interesting and pertinent industry news and developments.
  • Whitepapers, eBooks and information from trusted third parties.
Join For Free