About Exondys 51

Exondys 51 (also known as eteplirsen), developed by pharmaceutical company Sarepta Therapeutics, is designed to treat an estimated 13% of the Duchenne muscular dystrophy population amenable to the skipping of exon 51.

Decisions on approval for Exondys 51

The National Institute for Health and Care Excellence (NICE) was scheduled to begin evaluating Exondys 51 in the summer of 2018 under its Highly Specialised Technologies (HST) programme. However, NICE suspended their plans following the European Medicines Agency (EMA) rejecting the treatment in Septmeber 2018 following a re-examination.  

What action are we taking?

We know these delays are frustrating. The UK patient organisations are committed to working together, keeping you updated and ensuring the patient voice is at the heart of the process.

For more information, please contact Clare Lucas on c.lucas@musculardystrophyuk.org or call 020 7803 4838.  

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