5
Orphan Designations
3
FDA Approvals
13
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| PLG-related hereditary angioedema with normal C1inh | Orladeyo | Orphan Designation | - |
| acquired angioedema | Orladeyo | Orphan Designation | - |
| acute flaccid myelitis | 3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid | Orphan Designation | - |
| congenital lethal erythroderma | Orladeyo | Orphan Designation | - |
| essential thrombocythemia | 3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid | Orphan Designation | - |
| familial thrombocytosis | 3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid | Orphan Designation | - |
| fibrodysplasia ossificans progressiva | (S)-1-(4-(1-(3,4,5-trimethyloxyphenyl)-1H-imidazol-4-ylamino)thieno[2,3-d]pyrimidin-2-yl)pyrrolidine-2-carboxamide citrate | Orphan Designation | - |
| hereditary angioedema with normal C1Inh | Orladeyo | Orphan Designation | - |
| malignant carotid body paraganglioma | Orladeyo | Orphan Designation | - |
| paroxysmal nocturnal hemoglobinuria | BCX9930 is a small molecule inhibitor of human complement factor D. | Orphan Designation | - |
| post-treatment Lyme disease syndrome | Orladeyo | Orphan Designation | - |
| primary cutaneous T-cell non-Hodgkin lymphoma | Peldesine | Orphan Designation | - |
| treatment-refractory schizophrenia | Orladeyo | Orphan Designation | - |