1
Orphan Designations
0 approved, 1 designated
1
FDA Approvals
Latest: WAKIX (2020)
0
Active Trials
3
Rare Diseases
across 9 areas
0
News (30d)
Quiet
Harmony Biosciences, LLC is a company with 1 orphan drug designation across 3 rare diseases, including 1 FDA-approved therapy.
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 |
|---|---|---|
| Prader-Willi syndrome | pitolisant | Des.TrialAppr. |
| Prader-Willi-like syndrome | pitolisant | Des.TrialAppr. |
| Schaaf-Yang syndrome | pitolisant | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio