NICE confirm delays in evaluating Exondys 51

Published Date
Clare Lucas

Back in May, we shared the disappointing news that Exondys 51 had received a negative trend vote from the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

Exondys 51 (also known as Eterplirsen) is designed to treat an estimated 13% of the Duchenne population amenable to the skipping of exon 51.

Following this news the pharmaceutical company, Sarepta, have requested a data re-examination by the CHMP. The outcome of this will not be known until the Autumn.

What does this mean for the UK?

The National Institute for Health and Care Excellence (NICE) was scheduled to evaluate Exondys 51 this Summer under the Highly Specialised Technology (HST) programme. However, NICE have now confirmed that they have suspended their appraisals whilst they await a final decision from the CHMP.

What will we be doing?

We know these delays are frustrating and that is why we are organising a roundtable meeting with neuromuscular specialists, pharmaceutical companies, families and regulators to identify a way forward for exon-skipping treatments.

In the meantime, we are continuing to push for quicker access to treatments through our Fast Track campaign and remain committed to working with other charities in the UK and internationally to ensure patients’ voices are heard.

For more information contact our Campaigns and Engagement Manager, Clare Lucas, on or 020 7803 4838

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