4
Orphan Designations
0
FDA Approvals
22
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell acute lymphoblastic leukemia | inotuzumab ozogamicin | Orphan Designation | - |
| Becker muscular dystrophy | Recombinant human anti-GDF-8 (growth and differentiation factor-8) antibody | Orphan Designation | - |
| Becker nevus syndrome | Recombinant human anti-GDF-8 (growth and differentiation factor-8) antibody | Orphan Designation | - |
| Duchenne muscular dystrophy | Recombinant human anti-GDF-8 (growth and differentiation factor-8) antibody | Orphan Designation | - |
| Erb palsy | Recombinant human anti-GDF-8 (growth and differentiation factor-8) antibody | Orphan Designation | - |
| Stormorken syndrome | ReFacto, Xyntha | Orphan Designation | - |
| Tayoun-Maawali syndrome | Torisel | Orphan Designation | - |
| Thomsen and Becker disease | Recombinant human anti-GDF-8 (growth and differentiation factor-8) antibody | Orphan Designation | - |
| advanced heart failure | Torisel | Orphan Designation | - |
| advanced sleep phase syndrome | Torisel | Orphan Designation | - |
| advanced sleep phase syndrome 3 | Torisel | Orphan Designation | - |
| anosmia for isobutyric acid | ReFacto, Xyntha | Orphan Designation | - |
| ataxia, early-onset, with oculomotor apraxia and hypoalbuminemia | inotuzumab ozogamicin | Orphan Designation | - |
| cancer-related condition | inotuzumab ozogamicin | Orphan Designation | - |
| colorectal cancer | Torisel | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | ReFacto, Xyntha | Orphan Designation | - |
| linear and whorled nevoid hypermelanosis | Recombinant human anti-GDF-8 (growth and differentiation factor-8) antibody | Orphan Designation | - |
| lysinuric protein intolerance | ReFacto, Xyntha | Orphan Designation | - |
| pheochromocytoma | ReFacto, Xyntha | Orphan Designation | - |
| pheochromocytoma-islet cell tumor syndrome | ReFacto, Xyntha | Orphan Designation | - |
| renal cell carcinoma | Torisel | Orphan Designation | - |
| toxic or drug-related embryofetopathy | inotuzumab ozogamicin | Orphan Designation | - |