FDAsafety guidance72% confidence
Rare Disease Drug Approvals
Source: FDA
Why This Matters
Why this matters: sets a safety guidance precedent in the same sub-indication (rare disease) as Genzyme.
Regulatory Analysis
This page describes recent rare disease drug approvals that received CDER or FDA communication. This is not an exhaustive list of all rare disease drug approvals.
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