34
Orphan Designations
0
FDA Approvals
84
Rare Diseases
7
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| ALK+ histiocytosis | LorbrenaXalkori | Orphan Designation | - |
| ALK-negative anaplastic large cell lymphoma | Lorbrena | Orphan Designation | - |
| ALK-positive anaplastic large cell lymphoma | Xalkori | Orphan Designation | - |
| ALK-positive large B-cell lymphoma | LorbrenaXalkori | Orphan Designation | - |
| Bicervical bicornuate uterus with patent cervix and vagina | LorbrenaXalkori | Orphan Designation | - |
| Dubowitz syndrome | Idarubicin HCl for injection | Orphan Designation | - |
| Duchenne muscular dystrophy | recombinant human anti-GDF-8 monoclonal antibody | Orphan Designation | - |
| Finnish type amyloidosis | N-[4-(4-amino-2-ethyl-1H-imidazo[4,5-c]quinolin-1-yl)butyl]methanesulfonamide | Orphan Designation | - |
| Gaucher disease | ELELYSO for injection | Orphan Designation | - |
| Gaucher disease type III | Xalkori | Orphan Designation | - |
| Huntington disease | 2-[4-(1-Methyl-4-pyridin-4-yl-lH-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid | Orphan Designation | - |
| Mycobacterium avium complex disease | Rifabutin | Orphan Designation | - |
| Pneumocystis infectious disease | Clindamycin | Orphan Designation | - |
| Rett syndrome | ELELYSO for injection | Orphan Designation | - |
| Sandhoff disease, adult form | Xalkori | Orphan Designation | - |
| Yunis-Varon syndrome | Lorbrena | Orphan Designation | - |
| achondroplasia | Recombinant human fibroblast growth factor receptor 3 | Orphan Designation | - |
| acquired adult-onset immunodeficiency | Beqvez | Orphan Designation | - |
| acquired hemophilia A | Hympavzi | Orphan Designation | - |
| acute myeloid leukemia | maplirpaceptDAURISMO | Orphan Designation | - |
| adult embryonal tumor with multilayered rosettes, c19mc-altered | Beqvez | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Elrexfio | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Elrexfio | Orphan Designation | - |
| adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy | Beqvez | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Elrexfio | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Elrexfio | Orphan Designation | - |
| alcohol abuse | ELELYSO for injection | Orphan Designation | - |
| alcohol dependence | ELELYSO for injection | Orphan Designation | - |
| anaplastic large cell lymphoma | LorbrenaXalkori | Orphan Designation | - |
| ataxia and polyneuropathy, adult-onset | Xalkori | Orphan Designation | - |
| ataxia telangiectasia | Lorbrena | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | LorbrenaVIZIMPROXalkori | Orphan Designation | - |
| cataract 13 with adult I phenotype | Beqvez | Orphan Designation | - |
| central nervous system anaplastic large cell lymphoma | Lorbrena | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | Xalkori | Orphan Designation | - |
| dermatomyositis | Recombinant humanized monoclonal antibody (immunoglobulin gamma-1 with kappa light chains, IgG1kappa) directed against human soluble cytokine interferon beta. | Orphan Designation | - |
| disseminated | Rifabutin | Orphan Designation | - |
| familial amyloid neuropathy | tafamidis | Orphan Designation | - |
| follicular lymphoma | N-Methyl-2-[3-[((E)-2-pyridin-2-yl-vinyl)-1H-indazol-6-ylsulfanyl]benzamide | Orphan Designation | - |
| glioma susceptibility 3 | Idarubicin HCl for injection | Orphan Designation | - |
| hemophilia A | Recombinant adeno-associated viral vector, AAV2 serotype 6 (rAAv2/6), expressing human factor 8 (hf8) cDNAHympavzi | Orphan Designation | - |
| hemophilia A with vascular abnormality | Hympavzi | Orphan Designation | - |
| hemophilia B | Beqvez | Orphan Designation | - |
| heterotopia, periventricular, X-linked dominant | Monoclonal antiendotoxin antibody XMMEn-0e5 | Orphan Designation | - |
| inflammatory myofibroblastic tumor | Xalkori | Orphan Designation | - |
| intellectual disability-severe speech delay-mild dysmorphism syndrome | Hympavzi | Orphan Designation | - |
| intracerebral hemorrhage | recombinant activated Factor X variant | Orphan Designation | - |
| lung adenocarcinoma | VIZIMPRO | Orphan Designation | - |
| lung cancer | VIZIMPRO | Orphan Designation | - |
| lung lymphangioleiomyomatosis | Rapamune | Orphan Designation | - |
| lymphangioleiomyomatosis | Rapamune | Orphan Designation | - |
| malignant carotid body paraganglioma | 6-{(1S)-1-[(2-Amino-6-fluoroquinolin-3-yl)oxy]ethyl}-5-(1H-pyrazol-1-yl)pyridin-2(1H)-one | Orphan Designation | - |
| malignant pancreatic neoplasm | antagonist of chemokine Ligand-2axitinib | Orphan Designation | - |
| mild hemophilia A | Hympavzi | Orphan Designation | - |
| myelodysplastic syndrome | glasdegib | Orphan Designation | - |
| myoclonic dystonia 11 | ELELYSO for injection | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | LorbrenaXalkori | Orphan Designation | - |
| neuroblastoma | crizotinib | Orphan Designation | - |
| neurodevelopmental disorder with seizures, microcephaly, and brain abnormalities | Monoclonal antiendotoxin antibody XMMEn-0e5 | Orphan Designation | - |
| neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia | Xalkori | Orphan Designation | - |
| nicotine dependence | ELELYSO for injection | Orphan Designation | - |
| non-small cell lung carcinoma | VIZIMPRO | Orphan Designation | - |
| non-small cell squamous lung carcinoma | VIZIMPRO | Orphan Designation | - |
| nut midline carcinoma | Beqvez | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Xalkori | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Xalkori | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Xalkori | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Xalkori | Orphan Designation | - |
| pediatric-onset Graves disease | Xalkori | Orphan Designation | - |
| plasma cell myeloma | Elrexfio | Orphan Designation | - |
| pneumocystosis | Clindamycin | Orphan Designation | - |
| post-treatment Lyme disease syndrome | 6-{(1S)-1-[(2-Amino-6-fluoroquinolin-3-yl)oxy]ethyl}-5-(1H-pyrazol-1-yl)pyridin-2(1H)-one | Orphan Designation | - |
| primary adult heart tumor | Elrexfio | Orphan Designation | - |
| primary cutaneous T-cell non-Hodgkin lymphoma | N-[4-(4-amino-2-ethyl-lH-imidazo[4,5-c]quinolin-1-yl)butyl]methanesulfonamide | Orphan Designation | - |
| pulmonary arterial hypertension | a recombinant human IgG1 with minimized Fc effector function that selectively binds to BMP9 with high affinity and inhibits its interaction with Type II receptors on endothelial cells thereby blocking downstream signaling3-Acetyl-1-cyclopentyl-7-{[(3S,4R)-3-hydroxyoxan-4-yl]amino }-4-methyl-1,6-naphthyridin-2(1H)-one | Orphan Designation | - |
| severe hemophilia A | Hympavzi | Orphan Designation | - |
| sickle cell disease | a recombinant humanized Fc effector function null IgG1 antibody with kappa light chains (IgG1) that selectively binds to E-selectin to inhibit binding to cellular proteins containing carbohydrate structures with a sialyl Lewis X (sLex) deter | Orphan Designation | - |
| stage IVb bladder cancer | N-[4-(4-amino-2-ethyl-1H-imidazo[4,5-c]quinolin-1-yl)butyl]methanesulfonamide | Orphan Designation | - |
| thrombocytopenia 11 with multiple congenital anomalies and dysmorphic facies | Monoclonal antiendotoxin antibody XMMEn-0e5 | Orphan Designation | - |
| thrombocytopenia 5 | Idarubicin HCl for injection | Orphan Designation | - |
| thyroid cancer, nonmedullary, 2 | N-Methyl-2-[3-[((E)-2-pyridin-2-yl-vinyl)-1H-indazol-6-ylsulfanyl]benzamide | Orphan Designation | - |
| thyroid gland mixed medullary and follicular cell-derived carcinoma | N-Methyl-2-[3-[((E)-2-pyridin-2-yl-vinyl)-1H-indazol-6-ylsulfanyl]benzamide | Orphan Designation | - |
| treatment-refractory schizophrenia | 6-{(1S)-1-[(2-Amino-6-fluoroquinolin-3-yl)oxy]ethyl}-5-(1H-pyrazol-1-yl)pyridin-2(1H)-one | Orphan Designation | - |
| tumor suppressor gene on chromosome 11 | VIZIMPRO | Orphan Designation | - |