FDA delays PDUFA date for small biotech, raising questions about impact of agency cuts
Title
FDA Delays PDUFA Date for Small Biotech, Sparking Concerns Over Impact of Agency Resource Cuts
Keywords
- FDA
- PDUFA delay
- biotech approval
- agency staffing cuts
- drug approval timeline
- Stealth Biotherapeutics
- elamipretide
- rare disease
- regulatory review
- clinical trial data
Key Facts
- The FDA has missed the PDUFA (Prescription Drug User Fee Act) decision date for Stealth Biotherapeutics' candidate for an ultra-rare disease, delaying the approval decision without setting a new date1.
- The delay was triggered by the submission of additional data from Stealth, which the FDA deemed a "major amendment"—this automatically extended the original deadline by three months under standard procedures1.
- Labeling discussions between the FDA and Stealth are ongoing, but there is no clear timeline for a new decision date1.
- The FDA had previously granted orphan drug, fast track, and rare pediatric designations for Stealth’s therapy, elamipretide, targeting Barth syndrome1.
- Advisory committee support for approval was mixed (10-6 vote), and the FDA expressed concerns about trial design, data interpretation, and a potential placebo effect complicating the late-stage results1.
- Stealth’s CEO noted that powering concerns for ultra-rare disease trials are difficult to address due to limited patient populations, making it hard to design studies acceptable to the FDA1.
- The delay raises broader questions in the industry about whether recent or anticipated staffing and resource cuts at the FDA could lead to further delays for other small biotech applications, especially for complex or rare disease cases where regulatory review requires intensive analysis1.
- For standard New Molecular Entity (NME) New Drug Applications, the FDA has committed to communicate anticipated postmarketing requirements to applicants no later than eight weeks before the PDUFA date for 80% of applications by 2025, but major amendments or issues can disrupt these timelines5.
These facts underscore growing industry concern about the ripple effects of FDA resource constraints and process delays for smaller biotech firms and patients awaiting new therapies.
Sources:
1. https://www.fiercebiotech.com/biotech/fda-misses-pdufa-date-stealths-ultra-rare-disease-candidate-delaying-approval-decision