9
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Chagas disease | fixed dose combination of Pyronaridine and BenznidazolePyronaridine | Orphan Designation | - |
| Ebola hemorrhagic fever | quinacrinepyronaridine tetraphosphatetilorone dihydrochloride | Orphan Designation | - |
| Pitt-Hopkins syndrome | nicardipine | Orphan Designation | - |
| autosomal recessive nonsyndromic hearing loss 70 | recombinant human palmitoyl-protein thioeserase-1 (rhPPT1) | Orphan Designation | - |
| chordoma | afatanib and palbociclib | Orphan Designation | - |
| lethal acantholytic epidermolysis bullosa | quinacrinepyronaridine tetraphosphatetilorone dihydrochloride | Orphan Designation | - |
| malaria | Pyronaridine tetraphosphate | Orphan Designation | - |
| neuronal ceroid lipofuscinosis | recombinant human palmitoyl-protein thioeserase-1 (rhPPT1) | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 1 | recombinant human palmitoyl-protein thioeserase-1 (rhPPT1) | Orphan Designation | - |