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‘Dangling’ Accelerated Approvals: US FDA Flags Concerns About ‘Marginal’ Response Rates

 
• By Sue Sutter

Failed confirmatory trials for PD-1/L-1 inhibitor indications raise questions about marginal response rates in single-arm trials used to support accelerated approval, Oncology Center of Excellence leaders say in NEJM article that explains why the agency is taking six of these indications to its Oncologic Drugs Advisory Committee.

Dangling mugs
US FDA's Oncology Center of Excellence has coined the term "dangling accelerated approvals" when confirmatory trials fail to verify clinical benefit but indications remain on the label. • Source: Shutterstock

Checkpoint inhibitor indications approved on the basis of marginal response rates in single-arm trials are a key focus of the US Food and Drug Administration’s class-wide evaluation of whether accelerated approvals should remain intact despite the failure of confirmatory trials.

In a New England Journal of Medicine article published online 21 April, Oncology Center of Excellence chief of medical oncology...

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More from US FDA Performance Tracker

Novel US FDA Approvals Could Heat Up November, But Will Annual Count Beat 2024?

 
• By Bridget Silverman

Eight novel agents have user fee goal dates in November, which keeps the possibility of matching or topping the 2024 annual count alive.

Cidara’s Non-Vaccine Flu Preventive Offers Respite From Vaccine Conflicts

 
• By Bridget Silverman

Cidara's CD388 drug-Fc conjugate received a breakthrough designation as the Phase III ANCHOR trial started for once-per-season prevention of influenza in a higher-risk population.

October US FDA User Fee Goals: Few Novel Agents, But Many New Indications As Shutdown Looms

 
• By Bridget Silverman

October is unusually low on user fee goals for novel agents, but more than 20 applications for new indications and formulations will keep FDA staff busy, especially in oncology and ophthalmology.

J&J’s Inlexzo Is A Rare 505(b)(2) NDA To Qualify For US FDA’s Real-Time Oncology Review

 
• By Bridget Silverman

A Johnson & Johnson executive tells the Pink Sheet in an interview how the company developed its pretzel-shaped intravesical drug releasing system for gemcitabine, which used design and materials to improve efficacy and ease bladder cancer treatment.

More from Regulatory Trackers

Global Pharma Guidance Tracker – September 2025

 
• By Vibha Sharma and Anabel Costa-Ferreira

Stay up to date on regulatory guidelines from around the world with the Pink Sheet’s Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.

October US FDA User Fee Goals: Few Novel Agents, But Many New Indications As Shutdown Looms

 
• By Bridget Silverman

October is unusually low on user fee goals for novel agents, but more than 20 applications for new indications and formulations will keep FDA staff busy, especially in oncology and ophthalmology.

EU CHMP Opinions And MAA Updates

 
• By Neena Brizmohun

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.