FDA Unveils Plausible Mechanism Framework to Accelerate Tailored Therapies for Ultra-Rare Diseases
TL;DR Summary
The FDA issued a draft guidance outlining a Plausible Mechanism Framework to accelerate approvals of genome-editing and RNA-based, individualized therapies that target the root causes of ultra-rare diseases, allowing smaller, robust studies and the use of master protocols. Sponsors must demonstrate the therapy addresses the specific disease mechanism, rely on well-characterized natural history data, and show target engagement or editing; public comments are due within 60 days.
Topics:business#fda#genome-editing#health-policy#personalized-therapies#regulatory-guidance#ultra-rare-diseases
- FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases fda.gov
- The FDA creates a quicker path for gene therapies NPR
- FDA illuminates new approval pathway for bespoke gene therapies Fierce Biotech
- FDA proposes new treatment approval pathway for ultra-rare diseases The Hill
- FDA proposes framework to speed rare disease gene therapy approvals The Detroit News
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