5
Orphan Designations
0 approved, 5 designated
0
FDA Approvals
0
Active Trials
200 recruiting
5
Rare Diseases
across 10 areas
0
News (30d)
Quiet
Eloxx Pharmaceuticals, Ltd. is a company with 5 orphan drug designations across 5 rare diseases. 1 active policy bill affects their portfolio.
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 |
|---|---|---|
| Alport syndrome | 6'-(R)-Methyl-5-O-(5-amino-5,6-dideoxy-alpha-Ltalofuranosyl)-paromamine sulfate | Des.TrialAppr. |
| Rett syndrome | 6'-(R)-Methyl-5-O-(5-amino-5,6-dideoxy-a-L-talofuranosyl)- paromamine sulfate | Des.TrialAppr. |
| cystic fibrosis | 6'-(R)-Methyl-5-O-(5-amino-5,6-dideoxy-a-L-talofuranosyl)-paromamine sulfate | Des.TrialAppr. |
| cystinosis | 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate | Des.TrialAppr. |
| mucopolysaccharidosis type 1 | 6-(R)-Methyl-5-O-(5-amino-5,6-dideoxy-a-L-talofuranosyl)- paromamine sulfate | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
5
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
1
affecting portfolio
0% of portfolio targets high unmet need diseases
5
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
1
affecting portfolio