CHMP ‘negative trend vote’ for Exondys 51

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said late Thursday in its 1Q18 earnings that EMA’s CHMP issued a “negative trend vote” for Exondys 51 eteplirsen, which is under EMA review to treat Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. The company plans to request a reexamination.

Sarepta said CHMP did not conclude Exondys 51 was ineffective in the target patient population, but rather that the use of external controls as comparators in clinical trials in part precluded the product from meeting the requirements for conditional approval. In addition to the reexamination, the company said it will request a scientific advisory group of DMD and neuromuscular specialists to provide the committee with insight on the use of external controls...