Policy and campaigning

A call to action - submit evidence for Exondys-51

A guide to submitting patient input for Sarepta Therapeutics’s drug, Exondys 51.

Duchenne UK and their fellow Duchenne charities have sought advice from the European Medicines Agency to seek guidance on patient input for Sarepta Therapeutics’s drug, Exondys 51.

Please read the following carefully, and if you can, make a submission. Every voice counts in this process.

Exondys 51, formerly known as Eteplirsen is a treatment for Duchenne patients amenable to skipping exon 51. It’s currently under review at the European Medicines Agency by the Committee for Human Medicinal Products (CHMP). Exondys 51 has been approved in the United States by the Federal Drug Administration (FDA).

Patients and Families in the United Kingdom have been left feeling frustrated that they cannot access a drug that is now approved for use by families in the USA.

We need your help to try and get access for eligible patients in Europe.

The European Medicines Agency wants to know about your direct experiences with Duchenne, and if applicable, what the impact of having access to Exondys 51, formerly known as Eteplirsen, may have on you and your family.

Testimony, particularly from community members who are awaiting access to these therapies, is important to them.

Testimony from those who have been involved in the clinical trials either as a participant, the parent of a participant, a clinician, a researcher, or a community member and have first-hand knowledge of how Exondys 51 (Eteplirsen) has impacted the daily life of individuals living with Duchenne, is critical.

If you made a written submission ahead of the FDA Advisory Committee on Eteplirsen in the United States, we would suggest you re-review your statement considering the suggested points for consideration below and use that as a starting point for your submission.

What types of information should be included in your written testimony?

Please include your name, address and contact details. Patients and caregivers from outside the UK and EU can make a submission but it would be helpful to clearly state where you are from. 

Points for consideration:

For those with experience on Exondys 51 (Eteplirsen):

  • Describe how Duchenne impacts you/your child, and your family
  • If you took part in the early UK trials when Sarepta was AVI. How did you feel when the trial was stopped and the clinical trials were moved to America?
  • If you took part in the Prosensa/Biomarin/GSK trials for Drisapersen how do you feel that those trials have now been discontinued and you no longer have access to a therapy whilst patients in America do?
  • What physical changes did/have you seen because of Exondys 51? Please include things like what physical changes did/have you seen to both your mobility e.g. walking and changes to the upper body strength such as hand/finger movement
  • What has the emotional impact been because of the physical changes that have taken place? What has this meant to your family?
  • What activities of daily living did you/your child have difficulty performing prior to the trial’s initiation that became easier or possible as the trial went on?
  • Are there people in you/ your child’s daily life (i.e. teachers, family members, etc.) who may have been unaware of the trial and made observations that spoke to increased, endurance or stamina? Or changes in function?
  • If you have any concerns about the treatment being approved or at any point during the trial your child experienced side effects related to the experimental product, describe those – and discuss whether you considered them to be severe and how that played into your decision to continue/discontinue participation in the trial. If he didn’t experience severe side effects, include that in your statement.

For those outside of the trial/experience some things to think about for your submission:

  • What do you want the EMA and the CHMP members to know most about your child and others in the Duchenne community?
  • If your child was not included in the current trial, but is eligible for this therapy if approved – what would you like for the Committee to know for this review?
  • What do you think the emotional impact is for the child/adult and the whole family unit to have access to the drug?
  • How do you think it could change/impact everyday life with real examples related to accessing schooling/employment e.g brushing teeth, use of touch typing/independence in eating etc?
  • If your child is not eligible for this therapy, but would be eligible for a therapy that could soon be in development or is in early testing – what would you like the Committee to know?
  • Describe how Duchenne impacts you/your child, and your family
  • Describe what functions you/your child wishes they could maintain and worries about losing over time
  • Describe what meaningful benefit of a drug would be to you/your child and your family

Once you have compiled your testimony, You can send your submissions to Molly Hunt and Alex Johnson at Duchenne UK, [email protected] and we will pass them onto EMA.


The deadline for sending in your submissions is 10th of March 2017.

Published on 1 February 2017

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