CHEBI:71031 - orphan drug

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ChEBI Name orphan drug
Definition Any drug that has been developed specifically for treatment of a rare medical condition, the condition itself being known as an orphan disease.
Stars This entity has been manually annotated by the ChEBI Team.
Members Of orphan drug Class
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selexipag Mass : 496.624
Formula : C26H32N4O4S
ACT-333679 Mass : 419.517
Formula : C25H29N3O3
macitentan Mass : 588.27300
Formula : C19H20Br2N6O4S
ixazomib citrate Mass : 517.122
Formula : C20H23BCl2N2O9
ixazomib Mass : 361.029
Formula : C14H19BCl2N2O4
isavuconazonium sulfate Mass : 814.83500
Formula : C35H36F2N8O9S2
isavuconazole Mass : 437.46500
Formula : C22H17F2N5OS
lenvatinib Mass : 426.854
Formula : C21H19ClN4O4
lenvatinib mesylate Mass : 522.960
Formula : C22H23ClN4O7S
Definition : A two-drug mixture consisting of lumacaftor and ivacaftor, which is used for the treatment of cystic fibrosis.
lumacaftor Mass : 452.408
Formula : C24H18F2N2O5
rifaximin Mass : 785.87850
Formula : C43H51N3O11
sodium phenylbutyrate Mass : 186.18290
Formula : C10H11NaO2
carglumic acid Mass : 190.15400
Formula : C6H10N2O5
ivacaftor Mass : 392.49070
Formula : C24H28N2O3
uridine triacetate Mass : 370.312
Formula : C15H18N2O9
4-aminopyridine Mass : 94.11462
Formula : C5H6N2
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