3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 7 areas
0
News (30d)
Quiet
Neurolixis, Inc. is a company with 3 orphan drug designations 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 |
|---|---|---|
| Rett syndrome | 3-chloro-4-fluorophenyl-[4-fluoro-4-[[(5-methylpyrimidin-2-ylmethyl)amino]methyl]piperidin-1yl]methanone | Des.TrialAppr. |
| cerebellar ataxia | befiradol | Des.TrialAppr. |
| fragile X syndrome | 3-Chloro-4-fluorophenyl-[4-fluoro-4-[[(5-methylpyrimidin-2-ylmethyl) amino]methyl]piperidin-1-yl]methanone | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
4
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
4
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio