5
Orphan Designations
0
FDA Approvals
16
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| AIDS | CD4 human truncated 369 AA polypeptide | Orphan Designation | - |
| COVID-19–associated multisystem inflammatory syndrome in adults | Halfan | Orphan Designation | - |
| Johanson-Blizzard syndrome | N2'-deacetyl-N2'-(3-mercapto-1-oxopropyl)-Maystansine-Conjugated Humanized C242 Monoclonal Antibody | Orphan Designation | - |
| Kocher-debre-Semelaigne syndrome | His-D-Trp-Ala-Trp-D-Phe-Lys NH2 | Orphan Designation | - |
| alopecia-intellectual disability syndrome 2 | Halfan | Orphan Designation | - |
| glioblastoma | Halfan | Orphan Designation | - |
| holoprosencephaly 12 with or without pancreatic agenesis | N2'-deacetyl-N2'-(3-mercapto-1-oxopropyl)-Maystansine-Conjugated Humanized C242 Monoclonal Antibody | Orphan Designation | - |
| laryngo-onycho-cutaneous syndrome | His-D-Trp-Ala-Trp-D-Phe-Lys NH2 | Orphan Designation | - |
| latent autoimmune diabetes in adults | Halfan | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 6 (hepatocerebral type) | Halfan | Orphan Designation | - |
| myxedema | Triostat | Orphan Designation | - |
| myxedema coma | Triostat | Orphan Designation | - |
| myxedema heart disease | Triostat | Orphan Designation | - |
| nonimmune chronic idiopathic neutropenia of adults | Halfan | Orphan Designation | - |
| nut midline carcinoma | His-D-Trp-Ala-Trp-D-Phe-Lys NH2 | Orphan Designation | - |
| thrombocytopenia-absent radius syndrome | N2'-deacetyl-N2'-(3-mercapto-1-oxopropyl)-Maystansine-Conjugated Humanized C242 Monoclonal Antibody | Orphan Designation | - |