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. (Link to letter attached)
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 the company have already requested that that CHMP re-examine the eteplirsen submission. Sarepta 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 process by The National Institute for Health and Care Excellence (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 is at the heart of the process.
Our Director of Research at Action Duchenne added: “We all share your immediate disappointment with regards to eteplirsen’s European regulatory developments. Since the news yesterday evening, Action Duchenne and the UK patient organisations have been in touch with the company, in addition to their thoughtful communication to the community. Action Duchenne has been at the forefront of these developments from the very start and are here to support you, please feel free to get in touch with any queries or indeed to share your stories and your lived experiences. Together, we can and will make a difference and alongside our key partners be here for you, every step of the way.”
Please feel free to get in touch with Neil Bennett with any immediate or future queries, he will be delighted to hear from you: neil@actionduchenne.org; 0208 556 9955.
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