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Advisory Committees
At the advisory committee review of Stealth’s Barth syndrome treatment elamipretide, Office of New Drugs Director Peter Stein clarified the circumstances where clinical data in a related indication could serve as confirmatory evidence for a single adequate and well-controlled study.
Members of the Cardiovascular and Renal Drugs Advisory Committee repeatedly challenged Stealth’s assertion that a new randomized trial in Barth syndrome was not possible, but also said potential functional unblinding and other challenges could compromise a new study.
Pink Sheet reporter and editors discuss experts’ experience helping drug sponsors negotiate prices with CMS and the FDA’s proposal for a new pathway to update vaccines before a pandemic is declared.
US FDA Office of New Drugs Director Peter Stein says review divisions have made the case that a discussion-only meeting would solicit the necessary input.
The FDA’s vaccine advisory committee endorsed a new plan to update pandemic influenza vaccines “inter-pandemic,” but encouraged the agency to consider how to make the approach feasible for novel vaccine technologies.
The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that efficacy had been shown for the ultra-rare disease, but even panelists in the majority questioned whether the product satisfied the threshold requirement for an adequate and well-controlled study.
The threat of a highly pathogenic avian influenza pandemic spurred the FDA to have its vaccine advisory committee comment on a new process to update licensed prototype pandemic flu vaccines.
The Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
US FDA Office of New Drugs Director Peter Stein says review divisions have made the case that a discussion-only meeting would solicit the necessary input.