7
Orphan Designations
0
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Eisenmenger syndrome | marzeptacog alfa (activated) | Orphan Designation | - |
| Fabry disease | recombinant human alpha-galactosidase A fused in-frame to the aglycosylated human IgG4 Fc mutein | Orphan Designation | - |
| Fanconi anemia complementation group B | marzeptacog alfa (activated) | Orphan Designation | - |
| Kabuki syndrome 1 | marzeptacog alfa (activated) | Orphan Designation | - |
| RFT1-congenital disorder of glycosylation | Marzeptacog alfa (activated) | Orphan Designation | - |
| Stormorken syndrome | Marzeptacog alfa (activated) | Orphan Designation | - |
| autism | marzeptacog alfa (activated) | Orphan Designation | - |
| factor VII deficiency | Marzeptacog alfa (activated) | Orphan Designation | - |
| intellectual developmental disorder, X-linked, syndromic, Pilorge type | marzeptacog alfa (activated) | Orphan Designation | - |
| intellectual disability-severe speech delay-mild dysmorphism syndrome | marzeptacog alfa (activated) | Orphan Designation | - |
| malignant carotid body paraganglioma | Dalcinonacog Alfa | Orphan Designation | - |
| post-treatment Lyme disease syndrome | Dalcinonacog Alfa | Orphan Designation | - |
| sitosterolemia 1 | Marzeptacog alfa (activated) | Orphan Designation | - |
| thrombotic thrombocytopenic purpura | A (recombinant) disintegrin and metalloprotease with thrombospondin type 1 repeats, member 13 (ADAMTS13) protein | Orphan Designation | - |
| treatment-refractory schizophrenia | Dalcinonacog Alfa | Orphan Designation | - |