EMA confirms recommendation not to renew marketing authorisation for Translarna

In September we shared the disappointing news that the European Medicine Agency’s (EMA) human medicines committee (CHMP) had released a recommendation not to renew the marketing authorisation for Translarna (ataluren). Translarna is a medicine for treating individuals who have Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. It received conditional marketing authorisation in Europe in 2014 and families in Ireland fought hard to seek its availability, which was approved in 2019.

The CHMP agreed to re-examine its decision on request of the manufacturer PTC. This took place in January and despite efforts from the manufacturer and patient community the EMA announced on Friday, 26 January 2024 recommendation for non-renewal of authorisation of Translarna.  The process found that while there was evidence of benefit from the treatment, it’s not statistically significant. Importantly there was no concerns about the safety of the treatment.

This news is devastating for the boys and young men who are taking Translarna as well as those who were hoping to start this treatment. Hearing stories from the community we know many have seen benefits from taking this treatment and are concerned about what will happen when they no longer have access and there is no alternative drug available. If you or your child is receiving Translarna, you should speak to your doctor about this decision and what it means for you or your child’s treatment.

You can read EMA’s full press release here including a paragraph on information for patients and carers.