3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Epstein-Barr virus hepatitis | tabelecleucel | Orphan Designation | - |
| Epstein-Barr virus-related tumor | tabelecleucel | Orphan Designation | - |
| LEOPARD syndrome 3 | N-[3-[2-[(1R)-1-Hydroxypropan-2-ylamino]-6-(4- morpholinyl)-4-pyridinyl]-4-methylphenyl]-(3S)-3-(2,2,2-trifluoroethyl)-1- pyrrolidine carboxamide hydrochloride. | Orphan Designation | - |
| Noonan syndrome 7 | N-[3-[2-[(1R)-1-Hydroxypropan-2-ylamino]-6-(4- morpholinyl)-4-pyridinyl]-4-methylphenyl]-(3S)-3-(2,2,2-trifluoroethyl)-1- pyrrolidine carboxamide hydrochloride. | Orphan Designation | - |
| X-linked hypohidrotic ectodermal dysplasia | Fusion protein consisting of human immunoglobim G1 constant region Fc region fused to the human receptor binding domain of ectodysplasin-A1 | Orphan Designation | - |
| cardiofaciocutaneous syndrome 1 | N-[3-[2-[(1R)-1-Hydroxypropan-2-ylamino]-6-(4- morpholinyl)-4-pyridinyl]-4-methylphenyl]-(3S)-3-(2,2,2-trifluoroethyl)-1- pyrrolidine carboxamide hydrochloride. | Orphan Designation | - |
| systemic Epstein-Barr virus-positive T-cell lymphoproliferative disease of childhood | tabelecleucel | Orphan Designation | - |