The Top Line Autoimmune CAR-T: Navigating the FDA’s new regulatory playbook
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Feb 27, 2026 Harpreet Singh, Chief Medical Officer at Precision for Medicine and former FDA oncology division director, discusses CAR-T moving into autoimmune diseases. He unpacks the FDA’s cautious, case-by-case regulatory stance. Topics include long-term safety and follow-up, evolving clinical endpoints, reproductive and pediatric concerns, and which autoimmune settings may be best suited for CAR-T.
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FDA Signals Cautious Support For Autoimmune CAR-T
- FDA is supportive but cautious about expanding CAR-T into autoimmune diseases, prioritizing durability and long-term safety.
- The Annals piece by Vinay Prasad and CBER colleagues urges case-by-case development, pediatric/reproductive monitoring, and new endpoints like drug-free remission.
Start With B Cell Driven Diseases And Refractory Patients
- Developers should target autoimmune diseases driven by pathogenic B cells with single or few targets, e.g., CD19 in lupus and myasthenia gravis.
- Start in refractory populations (failure of ≥2 immunosuppressants) then move earlier if efficacy supports it.
Reason Behind Lengthy Post‑Treatment Follow Up
- Long-term follow-up stems from risks tied to viral vectors and delayed toxicities, including secondary malignancies that can appear years later.
- FDA picked ~15 years as a conservative cutoff to capture late events, though the timeframe may be reevaluated with more data.