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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Streamlined queries for third advisory committee review of a Duchenne muscular dystrophy drug ask only whether the data suggest ataluren is effective, may be effective but needs more study, or is not effective. In contrast, panelist had to muddle through seven discussion and voting questions for Sarepta's eteplirsen and nine for BioMarin's drisapersen.
US FDA's Peripheral and Central Nervous System Drugs Advisory Committee to consider application, which PTC Therapeutics filed over protest.
In memo to then-Commissioner Califf during review of Sarepta's Exondys 51, Jenkins wrote that he was concerned about leaving OND in hands of CDER director upon his retirement.