6
Orphan Designations
0
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult neuronal ceroid lipofuscinosis | Vyvgart Hytrulo | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Vyvgart Hytrulo | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Vyvgart Hytrulo | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Vyvgart Hytrulo | Orphan Designation | - |
| chronic inflammatory demyelinating polyradiculoneuropathy | Vyvgart Hytrulo | Orphan Designation | - |
| congenital myasthenic syndrome | a fully humanized immunoglobulin gamma 1 (IgG1) agonistic monoclonal antibody (mAb) that specifically targets and activates MuSK | Orphan Designation | - |
| congenital myasthenic syndrome 3A | Vyvgart HytruloVyvgart | Orphan Designation | - |
| congenital myasthenic syndrome 4C | Vyvgart HytruloVyvgart | Orphan Designation | - |
| congenital myasthenic syndrome 5 | Vyvgart HytruloVyvgart | Orphan Designation | - |
| congenital myasthenic syndrome 6 | Vyvgart HytruloVyvgart | Orphan Designation | - |
| idiopathic inflammatory myopathy | efgartigimod coformulated with recombinant human hyaluronidase PH20 | Orphan Designation | - |
| multifocal motor neuropathy | empasiprubart | Orphan Designation | - |
| myasthenia gravis | Vyvgart HytruloVyvgart | Orphan Designation | - |
| myasthenic syndrome, congenital, 1B, fast-channel | Vyvgart HytruloVyvgart | Orphan Designation | - |
| primary adult heart tumor | Vyvgart Hytrulo | Orphan Designation | - |