15
Orphan Designations
0
FDA Approvals
26
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | obatoclax mesylateArsenic trioxide | Orphan Designation | - |
| B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2) | lestaurtinib | Orphan Designation | - |
| Huntington disease | Austedo | Orphan Designation | - |
| Philadelphia-positive myelogenous leukemia | lestaurtinib | Orphan Designation | - |
| acute myeloblastic leukemia with maturation | arsenic | Orphan Designation | - |
| acute myeloid leukemia | lestaurtinibarsenic | Orphan Designation | - |
| acute myeloid leukemia with minimal differentiation | arsenic | Orphan Designation | - |
| acute promyelocytic leukemia | Trisenox | Orphan Designation | - |
| adult embryonal tumor with multilayered rosettes, c19mc-altered | Trisenox | Orphan Designation | - |
| adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy | Trisenox | Orphan Designation | - |
| amyotrophic lateral sclerosis | mecasermin | Orphan Designation | - |
| cataract 13 with adult I phenotype | Trisenox | Orphan Designation | - |
| chorea gravidarum | Austedo | Orphan Designation | - |
| chorea, benign familial | Austedo | Orphan Designation | - |
| chorea-acanthocytosis | Austedo | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | arsenic trioxide | Orphan Designation | - |
| craniosynostosis, Philadelphia type | lestaurtinib | Orphan Designation | - |
| isolated growth hormone deficiency type IA | recombinant human serum albumin human growth hormone | Orphan Designation | - |
| isolated growth hormone deficiency type IB | recombinant human serum albumin human growth hormone | Orphan Designation | - |
| isolated growth hormone deficiency type II | recombinant human serum albumin human growth hormone | Orphan Designation | - |
| liver cancer | arsenic trioxide | Orphan Designation | - |
| malignant glioma | Arsenic trioxide | Orphan Designation | - |
| multiple system atrophy | 3-(1,3-benzodioxol-5-yl)-5-(3-bromophenyl)-1H-pyrazole | Orphan Designation | - |
| myelodysplastic syndrome | arsenic trioxide | Orphan Designation | - |
| nut midline carcinoma | Trisenox | Orphan Designation | - |
| plasma cell myeloma | Arsenic trioxide | Orphan Designation | - |