6
Orphan Designations
0
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Duchenne muscular dystrophy | Glucagon like peptide-1 Elastin like peptide-120 fusion protein | Orphan Designation | - |
| adult dermatomyositis | Glucagon like peptide-1 (GLP-1) Elastin like peptide (ELP)-120 fusion protein | Orphan Designation | - |
| amyopathic dermatomyositis | Glucagon like peptide-1 (GLP-1) Elastin like peptide (ELP)-120 fusion protein | Orphan Designation | - |
| cardiomyopathy | Vasomera | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | N-(3-(2-cyanopropan-2-yl)-5-(4-methyl-1H-imidazol-1-yl)phenyl)-2-fluoro-4-methyl-5-((8-((1-methyl-1H-pyrazol-4-yl)amino)imidazo[1,2-a]pyridin-3-yl)ethynyl)benzamide dihydrochloride | Orphan Designation | - |
| dermatomyositis | Glucagon like peptide-1 (GLP-1) Elastin like peptide (ELP)-120 fusion protein | Orphan Designation | - |
| heritable pulmonary arterial hypertension | vasoactive intestinal peptide (VIP)-elastin-like peptide (ELP) fusion protein | Orphan Designation | - |
| idiopathic pulmonary arterial hypertension | vasoactive intestinal peptide (VIP)-elastin-like peptide (ELP) fusion protein | Orphan Designation | - |
| juvenile myelomonocytic leukemia | N-(3-(2-cyanopropan-2-yl)-5-(4-methyl-1H-imidazol-1-yl)phenyl)-2-fluoro-4-methyl-5-((8-((1-methyl-1H-pyrazol-4-yl)amino)imidazo[1,2-a]pyridin-3-yl)ethynyl)benzamide dihydrochloride | Orphan Designation | - |
| leukemia, myeloid, accelerated-phase | N-(3-(2-cyanopropan-2-yl)-5-(4-methyl-1H-imidazol-1-yl)phenyl)-2-fluoro-4-methyl-5-((8-((1-methyl-1H-pyrazol-4-yl)amino)imidazo[1,2-a]pyridin-3-yl)ethynyl)benzamide dihydrochloride | Orphan Designation | - |
| polymyositis | Glucagon like peptide-1 (GLP-1) Elastin like peptide (ELP)-120 fusion protein | Orphan Designation | - |
| pulmonary arterial hypertension | vasoactive intestinal peptide (VIP)-elastin-like peptide (ELP) fusion protein | Orphan Designation | - |