2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
2
Active Trials
200 recruiting
3
Rare Diseases
across 7 areas
1
News (30d)
Denali Therapeutics Inc. is a company with 2 orphan drug designations across 3 rare diseases. Active clinical trials in 2 indications. 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 |
|---|---|---|
| Parkinson disease | - | Des.TrialAppr. |
| mucopolysaccharidosis type 1 | - | Des.TrialAppr. |
| mucopolysaccharidosis type 2 | enzyme transport vehicle specific for the transferrin receptor fused to iduronate 2-sulfatase | Des.TrialAppr. |
33% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/3
candidate diseases
1
avg importance: 70
0
affecting portfolio
33% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/3
candidate diseases
1
avg importance: 70
0
affecting portfolio