2
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
200 recruiting
2
Rare Diseases
across 2 areas
0
News (30d)
Quiet
Repligen Corporation is a company with 2 orphan drug designations across 2 rare diseases.
Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.
Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA
| Disease | Drug(s) | Stage |
|---|---|---|
| Friedreich ataxia | N-(6-2-aminophenylamino)-6-oxohexyl)-4-methylbenzamide | Des.TrialAppr. |
| mitochondrial disease | 2',3',5'-tri-o-acetyluridine | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio