1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
200 recruiting
3
Rare Diseases
across 4 areas
0
News (30d)
Quiet
Nuvamid SA is a company with 1 orphan drug designation across 3 rare diseases. gene therapy candidates in 1 disease. 57 active policy bills affect 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 |
|---|---|---|
| sickle cell disease | Beta-nicotinamide mononucleotide | Des.TrialAppr. |
| sickle cell-hemoglobin c disease syndrome | Beta-nicotinamide mononucleotide | Des.TrialAppr. |
| sickle cell-hemoglobin d disease syndrome | Beta-nicotinamide mononucleotide | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
10
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
57
affecting portfolio
0% of portfolio targets high unmet need diseases
10
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
57
affecting portfolio