FDAsafety guidance72% confidence
Learning and Education to ADvance and Empower Rare Disease Drug Developers (LEADER 3D)
Source: FDA
Why This Matters
Why this matters: sets a safety guidance precedent in the same sub-indication (rare disease) as Prevail Therapeutics.
Regulatory Analysis
As part of the Accelerating Rare disease Cures (ARC) Program, CDER’s Rare Diseases Team inaugurated the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) initiative. Learn more.
Supporting Context
- Therapeutic area
- Genetic Disorders · Gaucher Disease
- Sub-indication
- Rare Disease
Related signal: Phase 1/2 Trial of LY3884961 for Gaucher Disease Initiated by Prevail Therapeutics