Translarna (sometimes referred to as ataluren), developed by pharmaceutical company PTC Therapeutics, is designed to treat children whose Duchenne muscular dystrophy is caused by a nonsense mutation, who are aged five and over and who can still walk. Clinical trials of Translarna showed that the drug could help keep children walking for longer, and delay the need for a wheelchair.
Access to Translarna
Translarna was approved by the European Medicines Agency (EMA) in April 2014. It is now available on the NHS throughout the UK.
In July 2016, NICE published its final guidance agreeing NHS England funding for Translarna through a ‘Managed Access Agreement’. This meant that there would be access to Translarna on the NHS in England for five years while further data and evidence is gathered. This decision led to implementation in Wales and Northern Ireland.
In Scotland, despite the Scottish Medicines Consortium not approving Translarna, the treatment is available through a funding request from a patient’s clinician to the relevant NHS Board in Scotland.
Expanding the licence
The EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended that the current licence for Translarna be expanded to include children aged two to five years old. The CHMP’s recommendation now needs to be authorised by the European Commission. We are currently assessing what action will need to be taken to extend access in Scotland, Wales and Northern Ireland, and the implications for the Managed Access Agreement in England.