Patients Can't Rescue Sarepta's Eteplirsen
This article was originally published in The Pink Sheet Daily
Hours of testimony was clearly compelling, but advisory committee agreed with FDA concerns about the Duchenne muscular dystrophy drug's efficacy.
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Patient advocates asked what they could do after US FDA’s complete response letter for the proposed Duchenne drug, but Sarepta's CEO said the company would get the product back on track to approval itself, a marked contrast to engaged role the community played the last time the firm ran into a roadblock at the agency.
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.
US FDA's Peripheral and Central Nervous System Drugs Advisory Committee to consider application, which PTC Therapeutics filed over protest.