2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 5 areas
0
News (30d)
Quiet
ORPHELIA Pharma 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 |
|---|---|---|
| malignant glioma | ready-to-use temozolomide oral suspension | Des.TrialAppr. |
| neuroblastoma | Temozolomide | 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