3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 5 areas
1
News (30d)
Applied Therapeutics Inc. is a company with 3 orphan drug designations across 3 rare diseases. gene therapy candidates in 1 disease. 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 |
|---|---|---|
| PMM2-congenital disorder of glycosylation | 2-(4-oxo-3-((5-(trifluoromethyl)benzo[d]thiazol-2-yl)methyl)-3,4-dihydrothieno[3,4-d]pyridazin-1-yl)acetic acid | Des.TrialAppr. |
| galactosemia | 2-(4-oxo-3-((5-(trifluoromethyl)benzo[d]thiazol-2-yl)methyl)-3,4-dihydrothieno[3,4-d]pyridazin-1-yl)acetic acid | Des.TrialAppr. |
| neuronopathy, distal hereditary motor, autosomal recessive 8 | 2-(4-oxo-3-((5-(trifluoromethyl)benzo[d]thiazol-2-yl)methyl)-3,4-dihydrothieno[3,4-d]pyridazin-1-yl)acetic acid | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/3
candidate diseases
1
avg importance: 40
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/3
candidate diseases
1
avg importance: 40
0
affecting portfolio