Translarna AdCom Has DMD Experience, But Most Opposed Approval Of Prior Applications

Several members of the Peripheral and Central Nervous System Drugs Advisory Committee meeting looking at PTC' Therapeutics' proposed Duchenne muscular dystrophy treatment also looked at Sarepta's Exondys 51 and BioMarin's Kyndrisa, but most did not vote or comment favorably.

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Advisory committee members will review another proposed Duchenne muscular dystrophy drug on Sept. 28.

More than half of the Peripheral and Central Nervous System Drugs Advisory Committee empaneled to review PTC Therapeutics Inc.'s Translarna (ataluren oral suspension) have looked at other proposed Duchenne muscular dystrophy treatments under review, but that may not prove an advantage for the sponsor.

Five committee members served on it during reviews of BioMarin Pharmaceutical Inc.'s Kyndrisa (drisapersen) and Sarepta Therapeutics Inc.'s...

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