1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
1
Active Trials
200 recruiting
4
Rare Diseases
across 9 areas
0
News (30d)
Quiet
Restem LLC is a company with 1 orphan drug designation across 4 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 |
|---|---|---|
| acquired idiopathic inflammatory myopathy | umbilical cord lining stem cell (ULSC) | Des.TrialAppr. |
| idiopathic inflammatory myopathy | umbilical cord lining stem cell (ULSC) | Des.TrialAppr. |
| juvenile dermatomyositis | umbilical cord lining stem cell (ULSC) | Des.TrialAppr. |
| muscular dystrophy | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio