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

Access to Exondys 51 has been delayed after the European Medicines Agency (EMA) did not grant a licence in May 2018.

Sarepta has requested that the EMA re-examine the data, and a decision is due in Autumn 2018.

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 have now confirmed that they have postponed their assessment whilst they await a final decision from the EMA.

What action are we taking?

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.

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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