2
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
6
Rare Diseases
across 12 areas
0
News (30d)
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EVER Neuro Pharma GmbH is a company with 2 orphan drug designations across 6 rare diseases. gene therapy candidates in 1 disease.
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 |
|---|---|---|
| CARASIL syndrome | peptide fraction derived from porcine brain protein | Des.TrialAppr. |
| HTRA1-related autosomal dominant cerebral small vessel disease | peptide fraction derived from porcine brain protein | Des.TrialAppr. |
| cathepsin a-related arteriopathy-strokes-leukoencephalopathy | peptide fraction derived from porcine brain protein | Des.TrialAppr. |
| cerebral arteriopathy with subcortical infarcts and leukoencephalopathy | peptide fraction derived from porcine brain protein | Des.TrialAppr. |
| frontotemporal dementia | peptide fraction derived from porcine brain protein | Des.TrialAppr. |
| retinal vasculopathy with cerebral leukoencephalopathy and systemic manifestations | peptide fraction derived from porcine brain protein | Des.TrialAppr. |
33% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
1/6
candidate diseases
0
avg importance: 0
0
affecting portfolio
33% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
1/6
candidate diseases
0
avg importance: 0
0
affecting portfolio