US FDA Performance Tracker

The US FDA’s regenerative medicine advanced therapy designation is seeing its biggest year yet after a slower ramp-up than the more established breakthrough therapy pathway.

A stacked user fee goal lineup sets the stage for market showdowns in primary biliary cholangitis and IgA nephropathy, the first psychedelic approval decision, and lots of targeted cancer therapies.

Pink Sheet reporter and editors discuss the impact of a US and EU drug approvals analysis that found the US FDA still clears many novel products first, including most new cell and gene therapies and cancer treatments.

Novel agents in immuno-oncology headline the drug center’s upcoming goal dates, while gene therapies make up most of the biologic center’s workload.

Interactive table from the Pink Sheet breaks down the 40 novel drugs and biologics have user fee goal dates before the new year.

So far this year, when the agency has taken action on NMEs that went before advisory committees, the FDA decision has matched the committee vote.

US FDA would need to approve 44 novel agents by year-end to match 2023’s big total, but only 40 candidates are known to have user fee goals in the second half of 2024.

Half of the FDA’s upcoming 2024 user fee goals target rare diseases, but highly prevalent conditions like uncomplicated urinary tract infections and atopic dermatitis are also up for agency action in the second half of the year.

Sun’s deuruxolitinib and Novo Nordisk’s insulin icodec stand out as new molecular entities among the small cluster of US FDA user fee goal dates in July.

June goal dates include RSV and pneumococcal vaccines, two COPD therapies, Rocket and Sarepta gene therapies, and lots of oncology.

The Center for Drug Evaluation and Research’s novel approvals concentrated in August, November, and December, while January and June were the slowest months, according to a Pink Sheet analysis of 2014-2023 data.

Newly announced US FDA advisory committee meetings on Lilly’s Alzheimer’s antibody, Lykos’ pioneering psychedelic therapy for PTSD relieve drought in non-oncology AdComms as the 2024-‘25 COVID vaccine strain selection meeting is postponed.

Five novel agents are among the user fee goal dates coming up in May 2024.

The US FDA approved Day One’s pediatric brain cancer drug Ojemda, ImmunityBio’s bladder cancer immunotherapy Anktiva, an uncomplicated UTI claim for Utility Therapeutics’ Pivya, which has a long history in Europe, and Pfizer’s hemophilia B gene therapy Beqvez.

The US FDA’s approval of Lumicell’s optical imaging agent Lumisight makes a dozen novel approvals in 2024 for the Center for Drug Evaluation and Research.

Labeling describes early death imbalance that delayed approval of Bristol Myers Squibb’s CAR-T therapy for more than three months after its user fee goal, but does not add to boxed warning; J&J’s Carvykti is due for imminent US FDA action on its own early-stage myeloma bid.

Seven novel agents approved in March include one accelerated approval, two rare pediatric disease priority review vouchers, two breakthrough therapy designations and one regenerative medicine advance therapy designation.

In a year full of regulatory milestones for the firm, PTC Therapeutics hopes to set some approval precedents – and basically hopes its candidates have an easier time of it than they have had before.

US FDA approves the PD-1 inhibitor 20 months after user fee goal, resolving one of the last applications delayed by China’s extended pandemic travel restrictions.

With Wegovy’s new indication for cardiovascular risk reduction in patients with overweight and obesity, the migration of diabetes drugs from a single disease state to address a host of interrelated conditions reaches a new milestone.