2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
1
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Axovia Therapeutics is a company with 2 orphan drug designations across 1 rare 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 |
|---|---|---|
| Bardet-Biedl syndrome | adeno-associated virus 2/9 expressing human BBS1 geneadeno-associated virus 2/8 expressing human BBS1 gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
0
avg importance: 0
0
affecting portfolio