Latest news

• Wave’s challenge to Sarepta in Duchenne falls flat

  Exondys 51 (eteplirsen), which was approved three years ago and made around $287m in sales in the first nine months of the year.

• FDA grants surprise OK to Sarepta’s DMD therapy after earlier rejection

  Vyondys 53 (golodirsen) is Sarepta’s second approved DMD therapy after Exondys 51 (eteplirsen), the first disease-modifying treatment to be introduced in the US for the inherited muscle-wasting disease

• Duchenne UK backs Evox dystrophin delivery project

  At the moment that includes already-market drugs that skip over or ignore the mutation in the dystrophin gene – such as PTC Therapeutics’ Translarna (ataluren)   and Sarepta’s Exondys 51 (eteplirsen) –

• CHMP backs Spark’s Luxturna, but rejects Sarepta again

  Sarepta setback. There was bitter news from the CHMP for Sarepta, as the panel turned down its application for Duchenne muscular dystrophy drug Exondys (eteplirsen) for a second time, setting up ... Eteplirsen was approved in the US as Exondys 51 in 2016,

• Pfizer abandons antibody for Duchenne

  News of the setback spurred modest gains in the share price of Sarepta, which sells exon-skipping therapy Exondys 51 (eteplirsen) in the US and Europe, as well as PTC Therapeutics