Pathways & Standards
Approval Standards
Acting Commissioner Sara Brenner rejected the company’s request for a hearing, saying the lack of an adequate and well-controlled trial showing tasimelteon's effectiveness in treating sleep-onset insomnia is “fatal to the application.”
The FY 2024 fourth quarter had the highest number of formal meetings cancelled because written responses were sufficient since the beginning of FY 2018.
The European Medicines Agency has recommended in favor of approving four new products, and has also reaffirmed its positive opinion for the Alzheimer’s disease drug, Leqembi.
HHS Secretary Robert F. Kennedy Jr. is nostalgic for the 1960s. Will that include pushing the agency back to the pre-user-fee-era model of defining success based on blocking unsafe drugs, rather than accelerating access to effective ones?
Review Pathways
The European Medicines Agency is considering whether apitegromab, brensocatib and diazoxide choline, from Scholar, Insmed and Soleno respectively, are drugs that are of potential major public health interest, particularly from the point of view of therapeutic innovation.
The agency sent the neuroscience-focused company a refuse-to-file letter for Wakix as a treatment for Idiopathic Hypersomnia, a disease with only one approved treatment and few others in development.
The US FDA is receiving more requests for regenerative medicine advanced therapy designation and granting more of them, according to recently reported agency data.
Companies that are admitted to the UK’s revised innovative licensing and access scheme will be mandated to work with the regulator on developing a target development profile for their product.
User Fees
Like the layoffs, the reason for the recalls of some laid off CDER and Office of Chief Counsel staff remains unclear.
The US FDA has navigated one round of layoff notices, but more cuts could be coming. Here are three potential scenarios.
Some FDA employees who were recently laid off by the Trump Administration are being called back to work, multiple sources confirmed to the Pink Sheet.
Pink Sheet editors and special guest Michael McCaughan of Prevision Policy discuss Robert F. Kennedy Jr.’s first act as HHS Secretary, former FDA Commissioner Scott Gottlieb’s suggestion that industry extend the existing user fee agreement rather than negotiate a new one, and President Trump’s policies impacting FDA advisory committee schedules and staffing.