18
Orphan Designations
0
FDA Approvals
46
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Adams-Oliver syndrome 1 | Prograf | Orphan Designation | - |
| Alstrom syndrome | Prograf | Orphan Designation | - |
| Bohring-Opitz syndrome | Prograf | Orphan Designation | - |
| Duchenne muscular dystrophy | (3R)-3-Methyl-6-[2-({5-methyl-2-[4-(trifluoromethyl)phenyl]-1H-imidazol-1-yl}methyl)phenoxy]hexanoic acid hemisulfate | Orphan Designation | - |
| Nezelof syndrome | isavuconazonium sulfate | Orphan Designation | - |
| Stargardt disease | MA09-hRPE cells | Orphan Designation | - |
| Stargardt disease 3 | MA09-hRPE cells | Orphan Designation | - |
| Zygomycosis | Cresemba | Orphan Designation | - |
| adrenal cortex carcinoma | linsitinib | Orphan Designation | - |
| adult embryonal tumor with multilayered rosettes, c19mc-altered | Vyloy | Orphan Designation | - |
| adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy | Vyloy | Orphan Designation | - |
| anti-MDA5 dermatomyositis | Cytomegalovirus DNA vaccine with plasmids expressing pp65 and gB genes | Orphan Designation | - |
| autosomal recessive nonsyndromic hearing loss 29 | bispecific antibody against CLDN18.2 and T cell receptor complex CD3 | Orphan Designation | - |
| brain ischemia | diannexin | Orphan Designation | - |
| candidiasis, invasive | isavuconazonium sulfate | Orphan Designation | - |
| cataract 13 with adult I phenotype | Vyloy | Orphan Designation | - |
| chylomicron retention disease | PrografAlefacept | Orphan Designation | - |
| clinically amyopathic dermatomyositis | Cytomegalovirus DNA vaccine with plasmids expressing pp65 and gB genes | Orphan Designation | - |
| digestive system neuroendocrine neoplasm | Bispecific antibody against CLDN18.2 and T cell receptor complex CD3 | Orphan Designation | - |
| discrimination, Two-point, reduction 1N | PrografAlefacept | Orphan Designation | - |
| disseminated candidiasis | isavuconazonium sulfate | Orphan Designation | - |
| endogenous depression | Cytomegalovirus DNA vaccine with plasmids expressing pp65 and gB genes | Orphan Designation | - |
| fibrous dysplasia | Prograf | Orphan Designation | - |
| gastric cancer | Vyloy | Orphan Designation | - |
| gastric mantle cell lymphoma | Bispecific antibody against CLDN18.2 and T cell receptor complex CD3 | Orphan Designation | - |
| gastroesophageal reflux disease | Bispecific antibody against CLDN18.2 and T cell receptor complex CD3 | Orphan Designation | - |
| invasive aspergillosis | Cresemba | Orphan Designation | - |
| invasive breast carcinoma | Cresemba | Orphan Designation | - |
| invasive hydatidiform mole | Cresemba | Orphan Designation | - |
| invasive lobular breast carcinoma | Cresemba | Orphan Designation | - |
| ischemia reperfusion injury | diannexin | Orphan Designation | - |
| long QT syndrome 13 | Prograf | Orphan Designation | - |
| malignant pancreatic neoplasm | chimeric monoclonal antibody against claudin-18 splice variant 2recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem cell antigen | Orphan Designation | - |
| multiple organ dysfunction syndrome | Prograf | Orphan Designation | - |
| neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome | Alefacept | Orphan Designation | - |
| nut midline carcinoma | Vyloy | Orphan Designation | - |
| ovarian cancer | chimeric monoclonal antibody against Claudin 6 | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Prograf | Orphan Designation | - |
| primary organ-specific lymphoma | Prograf | Orphan Designation | - |
| renal hypomagnesemia 3 | bispecific antibody against CLDN18.2 and T cell receptor complex CD3 | Orphan Designation | - |
| renal tubular transport disease | bispecific antibody against CLDN18.2 and T cell receptor complex CD3 | Orphan Designation | - |
| retinal ischemia | diannexin | Orphan Designation | - |
| sensory organ benign neoplasm | Prograf | Orphan Designation | - |
| severe early-childhood-onset retinal dystrophy | MA09-hRPE cells | Orphan Designation | - |
| sickle cell disease | 2-[(1,3-Benzoxazol-2-yl)amino]-N-[2-(2-hydroxyethoxy)ethyl]-1-methyl-1H-benzimidazole-5- carboxamide monophosphate | Orphan Designation | - |
| sitosterolemia | Prograf | Orphan Designation | - |