Translarna trial to come to Australia


Three Australian clinics will take part in the PTC Therapeutics phase 3 trial of Translarna (ataluren) for Duchenne. This will give Australians an opportunity to access Translarna which, although available in Europe, is not available here. Translarna is for around ten percent of boys (and rare girls) who have a certain type of change in their Glossary Link DNA called a nonsense Glossary Link mutation.

The trial will involve an initial 72 week period where half of the participants will receive a placebo and half will receive Translarna, then all participants will receive the drug for a further 72 weeks.

More details about the trial are available on or please contact us for more information.

Meanwhile in the USA, there was an Advisory Committee Meeting at the FDA for Translarna on September 28, 2017. The majority of the committee (10 out of 11) voted that “although it is possible that ataluren may be effective, the data are inconclusive, and more work would be needed to establish whether ataluren is effective”. The remaining committee member voted that Translarna was effective. Discussions from the Advisory Committee Meeting are used to help inform the FDA and on October 25 they issued a letter to say that Translarna had not been approved for sale in the US and further clinical trials are needed. PTC plans to appeal this decision. Read Pat Furlong's blog on the Ataluren Ad Comm: "Coming Full Circle."

Posted 10/25/2017