2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
1
Active Trials
200 recruiting
3
Rare Diseases
across 6 areas
1
News (30d)
Prevail Therapeutics is a company with 2 orphan drug designations across 3 rare diseases. Active clinical trials in 1 indication. 3 active policy bills affect their portfolio. 1 news article in the past 30 days.
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 |
|---|---|---|
| Gaucher disease | recombinant adeno-associated virus serotype 9 constitutively expressing codon optimized coding sequence of human GBA1 | Des.TrialAppr. |
| Parkinson disease | - | Des.TrialAppr. |
| frontotemporal dementia | Non-replicating recombinant adeno-associated virus serotype 9 containing the progranulin gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/3
candidate diseases
1
avg importance: 40
3
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/3
candidate diseases
1
avg importance: 40
3
affecting portfolio