US FDA’s Breakthrough Designation Pace Slows, But Topics Broaden

The 19 breakthrough therapy designations awarded so far this year run the gamut from areas that traditionally have not fared well with BTDs, like cardiovascular and respiratory disease, alongside the traditional cancer and orphan disease tentpoles.

Breakthrough slow down
The pace of designations for the fastest ways to get through FDA are slowing down. • Source: Nielsen Hobbs; the Pink Sheet| Shutterstock images

US FDA’s flagship expedited review programs – the breakthrough therapy designation and its younger cousin, the biologics-focused regenerative medicine advanced therapy designation – have added new designations at a slower pace than the peak a few years ago.

Those designations that have been announced this year, however, illustrate the widening scope of the programs beyond the oncologics and...

More from US FDA Performance Tracker

Complete Response Letter Trio Raises Prospect Of Tidal Shift In Regenerative Medicine Regulation

 

Replimune's RP1 oncolytic immunotherapy became the third regenerative medicine to receive an FDA complete response letter this month as the agency appears to re-visit previous agreements about accelerated approval.

US FDA’s Second Half 2025 User Fee Goals Swing Toward Respiratory Disease

 

The 43 novel agents with user fee goal dates in the remainder of 2025 have fewer oncologics and infectious disease drugs than the first half of the year.

Slowdown: US FDA’s 2025 Novel Approvals Are On Below-Average Pace

 

US FDA's 25 novel approvals in 2025 fell short of the agency's five-year average count, driven by a drop in drugs center approvals; 45 user fee goals remain in second half of year.

US FDA’s CRL Release More Incremental Than ‘Radical’ Transparency

 

The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.

More from Regulatory Trackers

US FDA’s CRL Release More Incremental Than ‘Radical’ Transparency

 

The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.

America Next: Most Novel Agents With July Goal Dates Have Been Approved Overseas

 

The US FDA will not be in its usual position as the first regulator to approve novel drugs, with only one of the six novel candidates on the July user fee calendar seeking its first approval worldwide in the US

EU CHMP Opinions And MAA Updates

 

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.