EMA committee supports Translarna for under 5s

Published Date
Jenny Sharpe & Clare Lucas

The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) has recommended that the current licence for Translarna (ataluren) be expanded to include children aged two to five years old.

Translarna is currently licensed for the treatment of children with nonsense mutation Duchenne muscular dystrophy who are aged over five years and are able to walk (ambulatory). Following a review of PTC Therapeutics’ Study 030 results, the CHMP has concluded that the drug is safe and effective in ambulatory children aged between two and five.

The CHMP’s recommendation now needs to be authorised by the European Commission (EC). Once that has occurred, PTC will be able to market Translarna to eligible children aged over two years old. We are currently looking into what this might mean for the Managed Access Agreement in the UK. We’ll keep you posted.

While we welcome this positive news, it also underlines the need for earlier diagnosis of Duchenne. The average age of diagnosis is currently around 4 and a half years old.

Given the progressive nature of Duchenne, it is vital that children are able to access treatments as soon as possible. This highlights the importance of resources such as our GP training module, which helps GPs have a better understanding of their role in the management of neuromuscular conditions. The hope is that the module will help GPs make faster referrals to neuromuscular specialists and secure earlier diagnoses for individuals so that they can access the right support as soon as possible. The GP module has been taken by over 1100 GPs since 2015.

Earlier access to Translarna also raises the question of newborn screening for Duchenne. We believe that screening would play a valuable role in addressing the issue of delayed diagnosis, which results in children not being able to access best standards of care at the optimum time and therefore a faster deterioration in their condition. We have been discussing the future of newborn screening for Duchenne with Public Health England, NHS England and others to identify the best route forward for ensuring that newborn screening for Duchenne can be put in place.

Further information

Find out more about Translarna and the Managed Access Agreement

Read our Parliamentary roundtable report on newborn screening for Duchenne

Find out about the latest efforts to develop a better newborn screening test


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