FDAsafety guidance72% confidence
Medical Products for Rare Diseases and Conditions
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
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