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 Genzyme.
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 · Pompe Disease
- Sub-indication
- Rare Disease
Related signal: Genzyme Launches Global Registry for Pompe Disease to Enhance Patient Care and Research