1
Orphan Designations
1 approved
0
FDA Approvals
0
Active Trials
5
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Biogen U.S. Corporation is a company with 1 orphan drug designation across 5 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 |
|---|---|---|
| Charcot-Marie-Tooth peroneal muscular atrophy and Friedreich ataxia, combined | Skyclarys | Des.TrialAppr. |
| Friedreich ataxia | Skyclarys | Des.TrialAppr. |
| Friedreich ataxia 2 | Skyclarys | Des.TrialAppr. |
| Friedreich ataxia with retained reflexes | Skyclarys | Des.TrialAppr. |
| Friedreich ataxia, so-called, with optic atrophy and sensorineural deafness | Skyclarys | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
7
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
7
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio