Last week, at the European Medicine’s Agency (EMA), the Committee for Medicinal Products for Human Use (CHMP) held a hearing looking at whether to grant a regulatory approval for eteplirsen (also known as Exondys 51), in Europe. 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. We will all be working to support you in sharing your stories and experiences.

Sarepta Therapeutics 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. 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 HST appraisal of the treatment by NICE and will update the community as soon as possible. Rest assured, that the UK patient organisations are committed to working together, keeping you updated and ensuring the patient voice at the heart of the process. 

Emily Crossley, co-founder of Duchenne UK, said:

We understand the frustration and disappointment within the community over this decision. We will work with Sarepta to understand what the next steps in the process will be.