17
Orphan Designations
0
FDA Approvals
19
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | [6-(4-Chlorophenyl)-spiro[3.5]non-6-en-7-yl]methyl}-piperazin-1-yl}-N-{{3-nitro-4-[((2S)-1,4-dioxan-2-ylmethyl)amino]phenyl}sulfonyl}-2-(1H- pyrrolo[2,3-b]pyridin-5-yloxy)benzamide | Orphan Designation | - |
| Finnish type amyloidosis | Alrizomadlin | Orphan Designation | - |
| Waldenstrom macroglobulinemia | [6-(4-Chlorophenyl)-spiro[3.5]non-6-en-7-yl]methyl}-piperazin-1-yl}-N-{{3-nitro-4-[((2S)-1,4-dioxan-2-ylmethyl)amino]phenyl}sulfonyl}-2-(1H- pyrrolo[2,3-b]pyridin-5-yloxy)benzamide | Orphan Designation | - |
| acute lymphoblastic leukemia | olverembatinib | Orphan Designation | - |
| acute myeloid leukemia | olverembatinib4-{4-{[6-(4-Chlorophenyl)spiro[3.5]non-6-en-7-yl]methyl}-piperazin-1-yl}-N-{{3-nitro-4-[((2S)-1,4-dioxan-2-ylmethyl)amino]phenyl}sulfonyl}-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamideAPG-115, an orally bioavailable small molecule. | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | olverembatinib | Orphan Designation | - |
| follicular lymphoma | 4-{4-{[6-(4-Chlorophenyl)spiro[3.5]non-6-en-7-yl]methyl}-piperazin-1-yl}-N-{{3-nitro-4-[((2S)-1,4-dioxan-2-ylmethyl)amino]phenyl}sulfonyl}-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide | Orphan Designation | - |
| gastric cancer | APG-115, a novel orally available small molecule MDM2 inhibitor | Orphan Designation | - |
| gastrointestinal stromal tumor | Olverembatinib | Orphan Designation | - |
| malignant carotid body paraganglioma | APG-2575, a novel, orally active, selective and potent BCL-2 inhibitor | Orphan Designation | - |
| myelodysplastic syndrome | lisaftoclax | Orphan Designation | - |
| neuroblastoma | alrizomadlin | Orphan Designation | - |
| post-treatment Lyme disease syndrome | APG-2575, a novel, orally active, selective and potent BCL-2 inhibitor | Orphan Designation | - |
| retinoblastoma | Murine double minute-2 (MDM2) or Human double minute-2 (HDM2) | Orphan Designation | - |
| small cell lung carcinoma | Pelcitoclax | Orphan Designation | - |
| soft tissue sarcoma | 4-((3'R,4'S,5'R)-6''-chloro-4'-(3-chloro-2-fluorophenyl)-1'-ethyl-2''-oxodispiro[cyclohexane-1,2'-pyrrolidine-3',3''-indoline]-5'-carboxamido)bicyclo [2.2.2]octane-1-carboxylic acid | Orphan Designation | - |
| stage II endometrioid carcinoma | Alrizomadlin | Orphan Designation | - |
| stage IVb bladder cancer | Alrizomadlin | Orphan Designation | - |
| treatment-refractory schizophrenia | APG-2575, a novel, orally active, selective and potent BCL-2 inhibitor | Orphan Designation | - |