2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
1
Active Trials
200 recruiting
2
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Arcturus Therapeutics, Inc. is a company with 2 orphan drug designations across 2 rare diseases. Active clinical trials in 1 indication. 1 active policy bill affects their portfolio.
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 |
|---|---|---|
| cystic fibrosis | single-stranded mRNA consisting of a 5 cap, a 5 untranslated region (UTR), reading frame of the transgene encoding for the human CFTR (hCFTR), the 3 UTR, and poly A tail | Des.TrialAppr. |
| ornithine carbamoyltransferase deficiency | Ornithine transcarbamylase messenger RNA | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
1
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
1
affecting portfolio