This biopharmaceutical company focuses on developing therapies for rare diseases, holding 29 orphan drug designations and six FDA-approved drugs across 57 conditions.
29
Orphan Designations
6
FDA Approvals
57
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | NiktimvoJAKAFI | Orphan Designation | - |
| Char syndrome | Jakafi | Orphan Designation | - |
| Ehlers-Danlos syndrome, kyphoscoliotic type, 2 | Monjuvi | Orphan Designation | - |
| Finnish type amyloidosis | epacadostat | Orphan Designation | - |
| Hodgkins lymphoma | (S)-7-(1-(9H-purin-6-ylamino)ethyl)-6-(3-fluorophenyl)-3-methyl-5H-thiazolo[3,2-a]pyrimidin-5-one | Orphan Designation | - |
| MALT lymphoma | tafasitamab-cxixparsaclisib | Orphan Designation | - |
| Ogden syndrome | Jakafi | Orphan Designation | - |
| acquired polycythemia vera | Jakafi | Orphan Designation | - |
| acute lymphoblastic leukemia | Ruxolitinib | Orphan Designation | - |
| adult embryonal tumor with multilayered rosettes, c19mc-altered | PEMAZYRE | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | ZynyzMONJUVI | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | ZynyzMONJUVI | Orphan Designation | - |
| adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy | PEMAZYRE | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | MonjuviZynyzMONJUVI | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | ZynyzMONJUVI | Orphan Designation | - |
| anal carcinoma | Zynyz | Orphan Designation | - |
| aortic valve stenosis | Zynyz | Orphan Designation | - |
| argininosuccinic aciduria | Zynyz | Orphan Designation | - |
| asthma, nasal polyps, and aspirin intolerance | Zynyz | Orphan Designation | - |
| ataxia, early-onset, with oculomotor apraxia and hypoalbuminemia | Pemazyre | Orphan Designation | - |
| autoimmune hemolytic anemia | parsaclisib | Orphan Designation | - |
| autosomal recessive Parkinson disease 14 | Pemazyre | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | MonjuviJakafi | Orphan Designation | - |
| cataract 13 with adult I phenotype | PEMAZYRE | Orphan Designation | - |
| cholangiocarcinoma | PEMAZYRE | Orphan Designation | - |
| chronic graft versus host disease | NiktimvoJAKAFI | Orphan Designation | - |
| chronic inflammatory demyelinating polyradiculoneuropathy | NiktimvoJAKAFI | Orphan Designation | - |
| chronic primary adrenal insufficiency | NiktimvoJAKAFI | Orphan Designation | - |
| chronic recurrent multifocal osteomyelitis | NiktimvoJAKAFI | Orphan Designation | - |
| diffuse large B-cell lymphoma | MONJUVI | Orphan Designation | - |
| essential thrombocythemia | ruxolitinib phosphate | Orphan Designation | - |
| extranodal nasal NK/T cell lymphoma | tafasitamab-cxixparsaclisib | Orphan Designation | - |
| fibrodysplasia ossificans progressiva | 2-Amino-N-(4-hydroxybicyclo[2.2.2]octan-1-yl)-5-(4-((1R,5S)-3-(tetrahydro-2H-pyran-4-yl)-3-azabicyclo[3.1.0]hexan-1-yl)phenyl)nicotinamide fumarate dihydrate | Orphan Designation | - |
| follicular lymphoma | Parsaclisib | Orphan Designation | - |
| graft versus host disease | itacitinibJAKAFI | Orphan Designation | - |
| intestinal pseudoobstruction, neuronal, chronic idiopathic, X-linked | humanized Fc engineered monoclonal antibody against CD19 | Orphan Designation | - |
| leukoencephalopathy, diffuse hereditary, with spheroids 1 | Pemazyre | Orphan Designation | - |
| malignant pancreatic neoplasm | 2-(3-(4-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)-1H-pyrazol-l-yl)-1-(1-(3-fluoro-2-(trifluoromethyl)isonicotinoyl)piperidin-4-yl)azetidin-3-yl)acetonitrile adipateruxolitinib | Orphan Designation | - |
| mantle cell lymphoma | parsaclisib | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 13 | humanized Fc engineered monoclonal antibody against CD19 | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | MonjuviJakafi | Orphan Designation | - |
| mycosis fungoides | tafasitamab-cxixparsaclisib | Orphan Designation | - |
| myelofibrosis | parsaclisib and ruxolitinibJakafi | Orphan Designation | - |
| myeloid neoplasm associated with FGFR1 rearrangement | Pemazyre | Orphan Designation | - |
| myeloid neoplasm associated with PDGFRA rearrangement | Pemazyre | Orphan Designation | - |
| myeloid neoplasm associated with PDGFRB rearrangement | Pemazyre | Orphan Designation | - |
| myeloid/lymphoid neoplasm associated with JAK2 rearrangement | Pemazyre | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | MonjuviJakafi | Orphan Designation | - |
| neurodevelopmental disorder with dysmorphic facies and thin corpus callosum | humanized Fc engineered monoclonal antibody against CD19 | Orphan Designation | - |
| neuroferritinopathy | Pemazyre | Orphan Designation | - |
| nodal marginal zone B-cell lymphoma | tafasitamab-cxixparsaclisib | Orphan Designation | - |
| nut midline carcinoma | PemazyrePEMAZYRE | Orphan Designation | - |
| primary adult heart tumor | MonjuviZynyzMONJUVI | Orphan Designation | - |
| splenic marginal zone lymphoma | tafasitamab-cxixparsaclisib | Orphan Designation | - |
| stage II endometrioid carcinoma | epacadostat | Orphan Designation | - |
| stage IVb bladder cancer | epacadostat | Orphan Designation | - |
| trabecular adenocarcinoma | Zynyz | Orphan Designation | - |