EMA’s CHMP confirms non-renewal of Translarna, denies new medicines Nezglyal and Syfovre

Negative opinions and non-renewals headlined this month’s recommendations from EMA’s CHMP.

After re-examination. EMA’s CHMP has again concluded that the effectiveness of Duchenne muscular dystrophy therapy Translarna ataluren from PTC Therapeutics Inc. (NASDAQ:PTCT) cannot be confirmed from Phase III study 041, which was required to convert the drug’s conditional authorization, granted in 2014, to full approval. The agency noted that the mechanism of action could not be confirmed from additional studies showing a small effect on dystrophin protein production. ...