3
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
127 recruiting
3
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Acceleron Pharma Inc. is a company with 3 orphan drug designations across 3 rare diseases. 5 active policy bills affect their portfolio.
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 |
|---|---|---|
| Charcot-Marie-Tooth disease | efmitermant alfa | Des.TrialAppr. |
| Duchenne muscular dystrophy | ActRIIB-IgG1) | Des.TrialAppr. |
| facioscapulohumeral muscular dystrophy | Follistatin291-IgG2 Fc fusion protein | Des.TrialAppr. |
50% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
5
affecting portfolio
50% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
5
affecting portfolio