Approval Standards
A risk-based approach to human cell therapies and tissue-based products could incentivize development and prevent bad actors from taking advantage of the current FDA system.
US FDA Commissioner nominee Martin Makary is being embraced by industry, and Senate Democrats, as a more traditional pick than other Trump Administration nominees, but the Make America Healthy Again agenda still is clearly coming to the agency.
The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Vimkunya, Bavarian Nordic's chikungunya vaccine.
The FDA generic drugs team’s first public workshop of the second Trump Administration ended with a request that industry amplify the value it finds from public engagement.
Only government staff participated in the meeting defining the next season’s influenza vaccine composition after the annual public FDA vaccines advisory committee meeting was cancelled.
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?
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 European Commission’s new biotech and biomanufacturing hub will make it easier for innovative biotech companies to find legislative documents, funding information and guidance to help them bring new products to the market.
The UK’s drug regulator is developing a “clear and streamlined” regulatory pathway for individualized cancer mRNA immunotherapies.
The US FDA faces many communications challenges with vaccine skepticism rampant. Is doing everything right going to be good enough?
The US FDA wants a smoother transition to newer “low global warming potential” propellants in metered dose inhalers than the prior transition away from CFCs.
Medicines containing a new active substance (NAS) approved for pan-EU marketing last year were again led by cancer drugs, with treatments for blood disorders following closely behind. Meanwhile, three NAS-containing drugs have been approved so far this year, and 12 other products are expected to be authorized shortly, having recently been recommended for approval by the European Medicine Agency.
The MHRA’s new scientific dialog program will offer drug developers “closed-door meetings” that will offer “confidential, commercially sensitive discussion” with its staff to help them “refine” their real-world evidence generation strategies.
Former FDA commissioners Mark McClellan and Scott Gottlieb, former acting commissioner Janet Woodcock and current commissioner Robert Califf offered advice on successfully implementing reforms and preventing a mass exodus of FDA employees as inklings emerge that the Trump team is already engaged on this front.
The US Food and Drug Administration updated obesity drug development draft guidance for the first time in almost 20 years and although a lot has changed in the field, the FDA’s fundamental expectations to support approval did not.
Pink Sheet editors discuss Center for Drug Evaluation and Research Director Patrizia Cavazzoni’s surprising retirement announcement, the importance of the large bolus of guidance documents that the FDA released 6 January, and the FDA’s decision to continue reviewing and granting rare pediatric disease designations even though the program lapsed.
2024 saw important regulatory changes in India including in areas such as GMP, clinical trials and efforts to rein in unethical marketing practices. Further action is expected to play out in the new year as well.