1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 4 areas
0
News (30d)
Quiet
Augustine Therapeutics NV is a company with 1 orphan drug designation across 3 rare diseases.
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 |
|---|---|---|
| Charcot-Marie-Tooth disease | highly selective non-hydroxamate, non-hydrazine producing, small molecule histone deacetylase 6 inhibitor | Des.TrialAppr. |
| Charcot-Marie-Tooth disease recessive intermediate D | highly selective non-hydroxamate, non-hydrazine producing, small molecule histone deacetylase 6 inhibitor | Des.TrialAppr. |
| intermediate Charcot-Marie-Tooth disease | highly selective non-hydroxamate, non-hydrazine producing, small molecule histone deacetylase 6 inhibitor | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio