1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
1
Active Trials
200 recruiting
2
Rare Diseases
across 2 areas
0
News (30d)
Quiet
Larimar Therapeutics, Inc. 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 |
|---|---|---|
| Friedreich ataxia | Fusion protein linking human frataxin to a cell-penetrant peptide | Des.TrialAppr. |
| mitochondrial disease | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio