2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
1
Active Trials
200 recruiting
5
Rare Diseases
across 8 areas
0
News (30d)
Quiet
Nerviano Medical Sciences S.r.l. is a company with 2 orphan drug designations across 5 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 |
|---|---|---|
| acute myeloid leukemia | 4-(4-Methyl-piperazin-1-yl)-N-{6-[2-(4-trifluoromethyl-benzyloxy)-ethoxy]- 1H-indazol-3-yl}-benzamide hemioxalate | Des.TrialAppr. |
| adult hepatocellular carcinoma | N-(2,6-diethyphenyl)-8-({4-[4-(dimethylamino)piperidin-1-yl]-2- methoxyphenyl}amino)-1-methyl-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide phosphate | Des.TrialAppr. |
| hepatocellular carcinoma | N-(2,6-diethyphenyl)-8-({4-[4-(dimethylamino)piperidin-1-yl]-2- methoxyphenyl}amino)-1-methyl-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide phosphate | Des.TrialAppr. |
| ovarian cancer | - | Des.TrialAppr. |
| pediatric hepatocellular carcinoma | N-(2,6-diethyphenyl)-8-({4-[4-(dimethylamino)piperidin-1-yl]-2- methoxyphenyl}amino)-1-methyl-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide phosphate | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
62
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
62
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio