3
Orphan Designations
2
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| CD4+/CD56+ hematodermic neoplasm | (1-{2-[(3S,4R)-1-[(3R,4R)-1-cyclopentyl-3-fluoro-4-(4-methoxyphenyl)pyrrolidine-3-carbonyl]-4-(methoxymethyl)pyrrolidin-3-yl]-5-(trifluoromethyl)phenyl}piperidine-4-caroxylic acid-phosphoric acid (1/1) | Orphan Designation | - |
| amyotrophic lateral sclerosis | RADICAVA ORSRADICAVAunasnemabRadicava ORS | Orphan Designation | FDA Approved |
| primary cutaneous lymphoma | (1-{2-[(3S,4R)-1-[(3R,4R)-1-cyclopentyl-3-fluoro-4-(4-methoxyphenyl)pyrrolidine-3-carbonyl]-4-(methoxymethyl)pyrrolidin-3-yl]-5-(trifluoromethyl)phenyl}piperidine-4-caroxylic acid-phosphoric acid (1/1) | Orphan Designation | - |
| recessive dystrophic epidermolysis bullosa | (1-{2-[(3S,4R)-1-[(3R,4R)-1-cyclopentyl-3-fluoro-4-(4-methoxyphenyl)pyrrolidine-3-carbonyl]-4-(methoxymethyl)pyrrolidin-3-yl]-5-(trifluoromethyl)phenyl}piperidine-4-caroxylic acid-phosphoric acid (1/1) | Orphan Designation | - |
| variegate porphyria | (1-{2-[(3S,4R)-1-[(3R,4R)-1-cyclopentyl-3-fluoro-4-(4-methoxyphenyl)pyrrolidine-3-carbonyl]-4-(methoxymethyl)pyrrolidin-3-yl]-5-(trifluoromethyl)phenyl}piperidine-4-caroxylic acid-phosphoric acid (1/1) | Orphan Designation | - |
| xeroderma pigmentosum variant type | (1-{2-[(3S,4R)-1-[(3R,4R)-1-cyclopentyl-3-fluoro-4-(4-methoxyphenyl)pyrrolidine-3-carbonyl]-4-(methoxymethyl)pyrrolidin-3-yl]-5-(trifluoromethyl)phenyl}piperidine-4-caroxylic acid-phosphoric acid (1/1) | Orphan Designation | - |