Eteplirsen is designed to treat an estimated 13% of the Duchenne population amenable to the skipping of exon 51. We are disappointed to share the news that the CHMP’s trend vote was negative. This means that efforts to bring eteplirsen to people living with Duchenne in Europe will be delayed.
You can read more in this letter to the European Duchenne Community from Sarepta.
What happens now?
We share the deep disappointment that families will be feeling at this decision, however, there are major steps being planned to engage the relevant regulatory stakeholders and we will all be working to support you in sharing your stories and lived experience.
Sarepta have told us that they remain committed to bringing eteplirsen to people living with Duchenne in Europe. The next step is for the final oral explanation from the CHMP meeting to be published which will be shared and Sarepta have already requested that that CHMP re-examine the eteplirsen submission. The company will be looking to appeal the negative decision, unfortunately this means a delay of several months. We will ensure to share any relevant news and documentation as soon as possible.
We are waiting to hear from Sarepta about what this news means for the highly specialised technology appraisal of the treatment by NICE and will update the community as soon as possible. We know that every day counts for people with Duchenne. Rest assured, that the UK patient organisations are committed to working together, keeping you updated and ensuring the patient voice is at the heart of the process.