AstraZeneca Pharmaceuticals LP is engaged in the development of treatments for rare diseases, holding 30 orphan drug designations and addressing 48 conditions, including polyneuropathy and systemic sclerosis.
30
Orphan Designations
32
FDA Approvals
48
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Bicervical bicornuate uterus with patent cervix and vagina | TagrissoIressa | Orphan Designation | - |
| Gaucher disease type III | selumetinib | Orphan Designation | - |
| NPHP3-related Meckel-like syndrome | selumetinib | Orphan Designation | - |
| Sandhoff disease, adult form | selumetinib | Orphan Designation | - |
| Thiel-Behnke corneal dystrophy | moxetumomab pasudotox | Orphan Designation | - |
| Toriello-Lacassie-Droste syndrome | TagrissoIressa | Orphan Designation | - |
| VISS syndrome | Benralizumab | Orphan Designation | - |
| acquired idiopathic inflammatory myopathy | anifrolumab | Orphan Designation | - |
| acute lymphoblastic leukemia | surovatamigmoxetumomab pasudotox | Orphan Designation | - |
| acute myeloid leukemia | 2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | ImfinziImjudoIMFINZIFasenraLynparzaLUMOXITI | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | ImfinziImjudoIMFINZIFasenraLynparzaLUMOXITI | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | ImfinziImjudoIMFINZIFasenraLynparzaLUMOXITI | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | ImfinziImjudoIMFINZIFasenraLynparzaLUMOXITI | Orphan Designation | - |
| ataxia and polyneuropathy, adult-onset | selumetinib | Orphan Designation | - |
| autoimmune lymphoproliferative syndrome | Benralizumab | Orphan Designation | - |
| autoinflammation with arthritis and dyskeratosis | TagrissoIressa | Orphan Designation | - |
| autosomal recessive congenital ichthyosis 2 | TagrissoIressa | Orphan Designation | - |
| biliary tract cancer | Imfinzi | Orphan Designation | - |
| cancer-associated retinopathy | retaspimycin hydrochloride | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | TagrissoIressa | Orphan Designation | - |
| cutaneous melanoma | retaspimycin hydrochloride | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | selumetinib | Orphan Designation | - |
| eosinophilic esophagitis | tezepelumabbenralizumab | Orphan Designation | - |
| eosinophilic gastritis | Benralizumab | Orphan Designation | - |
| eosinophilic gastroenteritis | Benralizumab | Orphan Designation | - |
| eosinophilic granulomatosis with polyangiitis | Fasenra | Orphan Designation | - |
| fallopian tube cancer | Lynparza | Orphan Designation | - |
| hairy cell leukemia | LUMOXITI | Orphan Designation | - |
| hepatocellular carcinoma | ImfinziImjudo | Orphan Designation | - |
| hypereosinophilic syndrome | benralizumab | Orphan Designation | - |
| idiopathic pulmonary fibrosis | saracatinib | Orphan Designation | - |
| linear nevus sebaceous syndrome | TagrissoIressa | Orphan Designation | - |
| lymphoproliferative syndrome 2 | moxetumomab pasudotox | Orphan Designation | - |
| malignant pancreatic neoplasm | Lynparza | Orphan Designation | - |
| melanoma | retaspimycin hydrochloride | Orphan Designation | - |
| neurofibromatosis type 1 | selumetinib | Orphan Designation | - |
| neurooculorenal syndrome | selumetinib | Orphan Designation | - |
| neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia | selumetinib | Orphan Designation | - |
| ovarian cancer | Lynparza | Orphan Designation | - |
| plasma cell myeloma | antibody drug conjugate consisting of a humanized monoclonal antibody targeting GPRC5D conjugated to MC-VC-PAB-monomethyl auristatin E (vcMMAE)Autologous chimeric antigen receptor (CAR)-positive T cells targeting cluster of differentiation 19 (CD19) and B cell maturation antigen (BCMA) | Orphan Designation | - |
| polyneuropathy | WAINUA | - | FDA Approved |
| primary adult heart tumor | ImfinziImjudoIMFINZIFasenraLynparzaLUMOXITI | Orphan Designation | - |
| primary peritoneal carcinoma | Lynparza | Orphan Designation | - |
| rhizomelic limb shortening with dysmorphic features | selumetinib | Orphan Designation | - |
| small cell lung carcinoma | IMFINZI | Orphan Designation | - |
| systemic sclerosis | anifrolumab | Orphan Designation | - |
| uveal melanoma | selumetinib | Orphan Designation | - |