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Jazz Pharmaceuticals Ireland Limited — Rare Disease Pipeline | Kisho | Kisho

Jazz Pharmaceuticals Ireland Limited

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Bloom syndrome	Rylaze	Orphan Designation	-
CHIME syndrome	Rylaze	Orphan Designation	-
Dubowitz syndrome	Rylaze	Orphan Designation	-
acute graft versus host disease	defibrotide	Orphan Designation	-
acute lymphoblastic leukemia	Rylaze	Orphan Designation	-
adult embryonal tumor with multilayered rosettes, c19mc-altered	Ziihera	Orphan Designation	-
adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy	Ziihera	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	Zepzelca	Orphan Designation	-
ataxia telangiectasia	Rylaze	Orphan Designation	-
basal ganglia calcification, idiopathic, 8, autosomal recessive	Xywav	Orphan Designation	-
biliary tract cancer	Ziihera	Orphan Designation	-
cataract 13 with adult I phenotype	Ziihera	Orphan Designation	-
combined small cell lung carcinoma	Zepzelca	Orphan Designation	-
early-onset generalized limb-onset dystonia	Xywav	Orphan Designation	-
esophageal cancer	zanidatamab	Orphan Designation	-
gastric cancer	zanidatamab	Orphan Designation	-
idiopathic hypersomnia	Xywav	Orphan Designation	-
idiopathic hypersomnia with long sleep time	Xywav	Orphan Designation	-
idiopathic hypersomnia without long sleep time	Xywav	Orphan Designation	-
lung cancer	Zepzelca	Orphan Designation	-
narcolepsy	Xyrem	Orphan Designation	-
narcolepsy 1	Xyrem	Orphan Designation	-
non-small cell lung carcinoma	Zepzelca	Orphan Designation	-
non-small cell squamous lung carcinoma	Zepzelca	Orphan Designation	-
nut midline carcinoma	Ziihera	Orphan Designation	-
small cell lung carcinoma	Zepzelca	Orphan Designation	-

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