FDA Regulatory Roundup
Leadership + Policy
CDER shake-up: George Tidmarsh resigned as director of CDER amid a federal investigation, creating immediate uncertainty around near-term policy priorities and leadership continuity. Watch for interim stewardship and potential knock-on effects to review culture and external communications. STAT+1
Digital Health Advisory Committee meets Nov 6: FDA’s Digital Health Advisory Committee convenes virtually this Thursday (9 a.m.–6 p.m. ET). Expect discussion to inform future frameworks for AI/ML-enabled devices and SaMD oversight. Public comments are docketed. U.S. Food and Drug Administration
Advisory committee calendar—devices: FDA announced a forthcoming Circulatory System Devices Panel meeting; device developers should track agenda materials for read-throughs on trial design and benefit-risk expectations. federalregister.gov
Approvals + Therapeutics
First-ever therapy for TK2 deficiency: FDA approved Kygevvi (doxecitine/doxribtimine) for thymidine kinase 2 deficiency in adults and pediatric patients with symptom onset ≤12 years—an ultra-rare mitochondrial disease. The decision includes Breakthrough Therapy Designation and addresses a substantial unmet need; sponsors in rare neuromuscular spaces should note precedent on endpoints and benefit documentation. U.S. Food and Drug Administration
Menin inhibitor class expands use: FDA posted a new approval for revumenib (Revuforj) in AML with NPM1 mutation (R/R), following last year’s KMT2A-t approval—solidifying menin inhibition as a cross-genomic strategy in myeloid malignancies. Expect companion-diagnostic momentum and labeling updates across the ecosystem. Dana-Farber Cancer Institute+1
Safety + Compliance
Toxic plant adulteration in supplements—expanded alert: FDA added more products to its safety alert on supplements contaminated with yellow oleander (cardiotoxic). Retailers and marketplaces should intensify surveillance; sponsors in adjacent categories should refresh supplier qualification and post-market monitoring SOPs. U.S. Food and Drug Administration
What to watch next (near-term)
Digital health signals (Nov 6): Look for language hinting at validation expectations, real-world data use, update controls, and transparency for adaptive algorithms. U.S. Food and Drug Administration
Leadership continuity at CDER: Interim assignments and any policy clarifications that could affect timelines for guidances and reviews. STAT+1
Operator takeaways
Plan for reviewer variability: With CDER leadership in flux, build extra margin for clarifying interactions and ensure your meeting packages over-document rationale (benefit–risk, unmet need, totality of evidence). STAT+1
Leverage rare-disease precedent: Kygevvi illustrates FDA’s willingness to move in ultra-rare settings with robust clinical justification; map analogs in your portfolio (natural history data, clinically meaningful function endpoints). U.S. Food and Drug Administration
Oncology diagnostics alignment: Menin-pathway expansion underscores the importance of early CDx strategy and assay performance claims; align analytical validity with enrichment strategies. Dana-Farber Cancer Institute
Tighten supplier controls: Update risk files and ingredient authentication where botanical adulteration is plausible; document CAPAs tied to the yellow-oleander alert. U.S. Food and Drug Administration
