FDAsafety guidance66% 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 Casper Pharma; the same agency is already in play for this signal, so precedent weight is higher.
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
- Rare Diseases · Cystinosis
- Sub-indication
- Rare Disease
Related signal: FDA Accepts NDA for Tiocystin by Casper Pharma