1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
1
Active Trials
200 recruiting
2
Rare Diseases
across 6 areas
0
News (30d)
Quiet
AXOLTIS Pharma is a company with 1 orphan drug designation across 2 rare diseases. Active clinical trials in 1 indication.
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 |
|---|---|---|
| amyotrophic lateral sclerosis | H-L-tryphophanyl-L-seryl-glycyl-L-tryptophanyl-L-seryl-L-seryl-L-cysteinyl-L-seryl-L-arginyl-L-seryl-L-cysteinyl-glycyl-OH (disulfide bond) | Des.TrialAppr. |
| multiple sclerosis | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
20
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
20
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio