Muscular Dystrophy Campaign puts Translarna in the parliamentary spotlight

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Yesterday's All Party Parliamentary Group (APPG) for Muscular Dystrophy meeting organised by the Muscular Dystrophy Campaign gave MPs and Peers the opportunity to discuss last month's announcement that the European Medicines Agency is recommending that Duchenne muscular dystrophy drug Ataluren (now to be known as Translarna) receives conditional approval.

Baroness Thomas of Winchester, a Trustee of the Muscular Dystrophy Campaign, and Mary Glindon MP, jointly chaired the session at which a panel of experts from the Medicines and Healthcare products Regulatory Agency and NHS England were challenged by parliamentarians on ensuring that Translarna is approved and commissioned for availability to patients as soon as possible.

The panel included:

  • Dr Siu Ping Lam, Director of Licensing, Medicines and Healthcare Products Regulatory Agency
  • Greg Markey, UK MHRA Representative on the Committee for Medicinal Products for Human Use (CHMP), European Medicines Agency
  • Paul McManus, Lead Pharmacist - specialised services (South Yorkshire and Bassetlaw), NHS England

On 23 May the European Medicines Agency announced that it will recommend that Duchenne muscular dystrophy drug ataluren receives conditional approval. The next step will be for the European Commission to review this decision. Ataluren has been developed by PTC Therapeutics to overcome a specific change in the DNA called a nonsense mutation which causes 10-15% of cases of Duchenne muscular dystrophy. Translarna, as the drug will now be known will be the first ever drug to treat an underlying genetic cause of Duchenne muscular dystrophy in children and young people affected, outside of a clinical trial.

Translarna may have the potential to treat around 200 individuals with Duchenne muscular dystrophy in the UK (of a total of around 2,400). The next step for the drug will be for the European Commission to decide whether to grant a conditional licence (which normally takes approximately three months).

Find out more in our frequently asked questions section on Translarna.
The topics of discussion at the APPG session included:

  • an overview of the conditional approval announcement for Translarna
  • the extent of the licence for Translarna
  • the process for accelerated review in Europe for drugs proceeding to accelerated approval in the USA
  • the new Early Access to Medicines Scheme and adaptive licensing pilot project
  • cost assessment and funding for the treatment
  • involvement of Clinical Reference Groups

Following the conclusion of the meeting, the Muscular Dystrophy Campaign held a meeting with the National Institute for Care Excellence (NICE) to establish how they might be involved in the assessment for Translarna in providing an evidence summary assessment.

The next stage of the process will be ensuring a 'commissioning policy' is set up by NHS England over the next few months with a health economic assessment carried out, and the Muscular Dystrophy Campaign will be meeting with NHS England on Friday to discuss the timeframes for Translarna and the details for the 'commissioning policy'.  

For more information and to get involved in the Muscular Dystrophy Campaign's faster access to treatments campaign, please get in touch on 020 7803 4838 or by email at

Further information and links

To read frequently asked questions about Translarna

To learn more about Duchenne muscular dystrophy

Find out more about the research we are funding into duchenne muscular dystrophy

Read more about conditional approval in Target Resaerch

To read the press release from PTC in full

It is only through your contributions that we can continue to fund the vital work that takes us closer to finding treatments and cures for muscle-wasting conditions. Donate now and help change the lives of thousands of people living with muscular dystrophy and related neuromuscular conditions.

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