We've just heard the disappointing news that Sarepta has received a negative opinion of eteplirsen following a second review by the European Medicine's Agency Committee for Medicinal Products for Human Use (CHMP).

Eteplirsen (or Exondys 51) is a drug that is designed to induce exon skipping in exon 51 of the dystrophin gene, and Sarepta believes it has the potential to treat 13% of people living with Duchenne.

On hearing the decision this morning, my heart sunk for those who have been waiting for this decision for their loved ones. It is more than disappointing, and being a Duchenne parent myself I understand the impact that this will have to eligible families and the wider Duchenne community. As a charity, we will continue to do all we can to work with Sarepta, the EMA and partner organisations to ensure access to treatments is timely and positive. - Shelley Simmonds, CEO

In June, Sarepta received a negative opinion from the CHMP and requested that the committee reconsider the evidence. That review has now been completed. Because this review didn’t represent a new application, the company was limited in the new data it could provide.

Doug Ingram, president and chief executive officer, Sarepta Therapeutics, said “While largely anticipated, we are disappointed with the outcome of the CHMP re-examination and firmly believe that eteplirsen should be made available to patients in Europe, as it is in the United States,”

Sarepta also said that they were encouraged by the openness of their discussions with the CHMP and will investigate different routes that eteplirsen could use to gain a marketing approval – with a view to gaining further advice in 2019.

The CHMP opinion is a key stage in gaining marketing approval for a new drug in Europe. While we’re disappointed by the news that Eteplirsen hasn’t been given a positive opinion, it’s encouraging to see Sarepta already looking into other options. - Neil Bennett – Director of Research

 Find out more