CHMP refuses recommendation to approve Exondys 51 for Duchenne muscular dystrophy.- Sarepta Therapeutics.
The EMA's CHMP has taken a negative view on its decision to recommend approval of Exondys 51 (eteplirsen) injection, from Sarepta Therapeutics, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD).
It is reported that the CHMP did not conclude that eteplirsen is ineffective for exon 51 amenable patients, but rather that Sarepta has not yet met the regulatory threshold for conditional approval, in part due to the use of external controls as comparators in the studies. To solve this, Sarepta is planning to file for re-examination. The company will request that a Scientific Advisory Group will be convened.
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