Latest news

• 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

• Mallinckrodt doses first patient in muscular dystrophy trial

  For now, the only approved therapies are Sarepta’s exon-skipping therapy Exondys 51 (eteplirsen), which has some controversy over its efficacy, PTC’s Translarna (ataluren), which is available in Europe

• Sarepta soars on positive Duchenne data

  it’s already a player in DMD therapy with its exon-skipping Exondys 51 (eteplirsen) therapy – approved in the US but not the Europe – but that drug is only suitable for

• Daily Brief: ASCO latest, Europe cautions on immunotherapy, rejects Duchenne treatment

  Europe still unconvinced by Duchenne treatment Exondys. Finally, the CHMP has confirmed its earlier decision to refuse marketing authorisation for Sarepta’s Exondys(eteplirsen).