2
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Axentis Pharma AG is a company with 2 orphan drug designations across 2 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 |
|---|---|---|
| glycogen storage disease Ib | tobramycin liposomal for inhalation | Des.TrialAppr. |
| granulomatous disease, chronic, autosomal recessive, 5 | tobramycin liposomal for inhalation | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/2
candidate diseases
0
avg importance: 0
0
affecting portfolio