BioMarin May Have To Find Subgroups For Drisapersen Efficacy
This article was originally published in The Pink Sheet Daily
FDA advisory committee has concerns about efficacy, says more work needed to identify responders.
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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.
Trial diversity is among the issues that stakeholders worry could hinder vaccine uptake.
Pink Sheet Podcast: COVID-19 Stresses Manufacturing, Vaccine Placebo Controls, Thorny FDA Precedents
Pink Sheet reporters and editor discuss the pandemic’s effects on non-coronavirus-related product manufacturing, US FDA efforts to ensure vaccine sponsors maintain placebo controls once a product is available, and precedents that burden the agency.