This biopharmaceutical company focuses on developing treatments for rare diseases, with 26 orphan drug designations and active involvement in 51 rare conditions, including various cancers and genetic disorders.
26
Orphan Designations
8
FDA Approvals
51
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Alstrom syndrome | lenvatinib | Orphan Designation | - |
| Bicervical bicornuate uterus with patent cervix and vagina | LENVIMA | Orphan Designation | - |
| Char syndrome | LENVIMAHalaven | Orphan Designation | - |
| Dravet syndrome | Lorcaserin | Orphan Designation | - |
| Finnish type amyloidosis | lenvatinibchlorotoxin | Orphan Designation | - |
| H syndrome | Ontak | Orphan Designation | - |
| Lennox-Gastaut syndrome | perampanelBanzel | Orphan Designation | - |
| Ramos-Arroyo syndrome | Ontak | Orphan Designation | - |
| X-linked reticulate pigmentary disorder | Targretin | Orphan Designation | - |
| acute myeloid leukemia | (2S,3S,4E,6S,7R,10R)-7,10-Dihydroxy-3,7-dimethyl-12-oxo-2-[(2E,4E,6R)-6-(pyridin-2-yl)hepta-2,4-dien-2-yl]oxacyclododec-4-en-6-yl 4-methylpiperazine-1-carboxylate | Orphan Designation | - |
| adult hepatocellular carcinoma | (N-{2-[(6-{[(2,6-Dichloro-3,5-dimethoxyphenyl)carbamoyl](methyl)amino}pyrimidin-4- yl)amino]-5-(4-ethylpiperazin-1-yl)phenyl}prop-2-enamide) | Orphan Designation | - |
| alveolar capillary dysplasia with misalignment of pulmonary veins | Ontak | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | LENVIMALenvimaHalaven | Orphan Designation | - |
| childhood myelodysplastic syndrome | (2S,3S,4E,6S,7R,10R)-7,10-Dihydroxy-3,7-dimethyl-12-oxo-2-[(2E,4E,6R)-6-(pyridin-2-yl)hepta-2,4-dien-2-yl]oxacyclododec-4-en-6-yl 4-methylpiperazine-1-carboxylate | Orphan Designation | - |
| chronic myelomonocytic leukemia | (2S,3S,4E,6S,7R,10R)-7,10-Dihydroxy-3,7-dimethyl-12-oxo-2-[(2E,4E,6R)-6-(pyridin-2-yl)hepta-2,4-dien-2-yl]oxacyclododec-4-en-6-yl 4-methylpiperazine-1-carboxylate | Orphan Designation | - |
| congenital heart defects, multiple types, 8, with or without heterotaxy | Ontak | Orphan Designation | - |
| cutaneous larva migrans | Targretin | Orphan Designation | - |
| cutaneous leishmaniasis | Targretin | Orphan Designation | - |
| esophageal cancer | lenvatinib | Orphan Designation | - |
| fanconi anemia, complementation group 10 | Ontak | Orphan Designation | - |
| follicular bronchiolits | Lenvima | Orphan Designation | - |
| follicular dendritic cell sarcoma | Lenvima | Orphan Designation | - |
| follicular lymphoma | Lenvima | Orphan Designation | - |
| gastric cancer | lenvatinib | Orphan Designation | - |
| hepatocellular carcinoma | (N-{2-[(6-{[(2,6-Dichloro-3,5-dimethoxyphenyl)carbamoyl](methyl)amino}pyrimidin-4- yl)amino]-5-(4-ethylpiperazin-1-yl)phenyl}prop-2-enamide)LENVIMA | Orphan Designation | - |
| lysinuric protein intolerance | lenvatinib | Orphan Designation | - |
| malaria | 3S,4R,8R,9S,10S)-10- [(Dimethylamino)methyl]-3,4-dihydroxy-N- (4-methoxyphenyl)-9- [4-(phenylethynyl)phenyl]-1,6-diazabicyclo [6.2.0]decane-6-carboxamide | Orphan Designation | - |
| malignant glioma | ChlorotoxinI(131)-TM-601 (chlorotoxin) | Orphan Designation | - |
| mature T-cell and NK-cell non-Hodgkin lymphoma | denileukin diftitox | Orphan Designation | - |
| mesothelioma | amatuximab | Orphan Designation | - |
| metastatic malignant neoplasm in the colon | Lenvima | Orphan Designation | - |
| metastatic malignant neoplasm in the lymph nodes | Lenvima | Orphan Designation | - |
| metastatic melanoma | Lenvima | Orphan Designation | - |
| metastatic neoplasm | Lenvima | Orphan Designation | - |
| metastatic squamous cell carcinoma | Lenvima | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 9 | Ontak | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | LENVIMAHalaven | Orphan Designation | - |
| myelodysplastic syndrome | (2S,3S,4E,6S,7R,10R)-7,10-Dihydroxy-3,7-dimethyl-12-oxo-2-[(2E,4E,6R)-6-(pyridin-2-yl)hepta-2,4-dien-2-yl]oxacyclododec-4-en-6-yl 4-methylpiperazine-1-carboxylate | Orphan Designation | - |
| myelodysplastic syndrome with excess blasts-2 | (2S,3S,4E,6S,7R,10R)-7,10-Dihydroxy-3,7-dimethyl-12-oxo-2-[(2E,4E,6R)-6-(pyridin-2-yl)hepta-2,4-dien-2-yl]oxacyclododec-4-en-6-yl 4-methylpiperazine-1-carboxylate | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | LENVIMAHalaven | Orphan Designation | - |
| ovarian cancer | farletuzumabirofulven | Orphan Designation | - |
| pediatric hepatocellular carcinoma | (N-{2-[(6-{[(2,6-Dichloro-3,5-dimethoxyphenyl)carbamoyl](methyl)amino}pyrimidin-4- yl)amino]-5-(4-ethylpiperazin-1-yl)phenyl}prop-2-enamide) | Orphan Designation | - |
| pilocytic astrocytoma with histological features of anaplasia | 6-hydroxymethylacylfulvene | Orphan Designation | - |
| primary cutaneous T-cell non-Hodgkin lymphoma | Targretin | Orphan Designation | - |
| primary cutaneous lymphoma | Targretin | Orphan Designation | - |
| renal cell carcinoma | LENVIMAirofulven | Orphan Designation | FDA Approved |
| skin disorder | Targretin | Orphan Designation | - |
| soft tissue sarcoma | HalavenHumanized monoclonal antibody to TumorEndothelial Marker-1 | Orphan Designation | - |
| squamous carcinoma in situ | chlorotoxin | Orphan Designation | - |
| thyroid cancer, nonmedullary, 2 | Lenvima | Orphan Designation | - |
| uterine cervix carcinoma in situ | chlorotoxin | Orphan Designation | - |