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Jazz Pharmaceuticals, Inc. — Rare Disease Pipeline | Kisho | Kisho

Jazz Pharmaceuticals, Inc.

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This biopharmaceutical company focuses on developing treatments for rare diseases, with 8 FDA-approved drugs and 5 orphan drug designations across 16 conditions, including various hematologic and metabolic disorders.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Bicervical bicornuate uterus with patent cervix and vagina	Erwinase	Orphan Designation	-
Down syndrome	Erwinase	Orphan Designation	-
acute adrenal insufficiency	Clonazepam Intranasal Spray	Orphan Designation	-
acute lymphoblastic leukemia	RYLAZEmPEG-r-crisantaspaseErwinase	Orphan Designation	FDA Approved
adult pleomorphic rhabdomyosarcoma	Defitelio	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	Erwinase	Orphan Designation	-
familial acute necrotizing encephalopathy	Clonazepam Intranasal Spray	Orphan Designation	-
hepatic veno-occlusive disease	defibrotideDefitelio	Orphan Designation	-
multiple congenital anomalies-hypotonia-seizures syndrome 2	Erwinase	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	Erwinase	Orphan Designation	-
pediatric acute-onset neuropsychiatric syndrome	Defitelio	Orphan Designation	-
pediatric hepatocellular carcinoma	Defitelio	Orphan Designation	-
pediatric ovarian dysgerminoma	Defitelio	Orphan Designation	-
pediatric ovarian germ cell tumor	Defitelio	Orphan Designation	-
pediatric-onset Graves disease	Defitelio	Orphan Designation	-
recurrent acute pancreatitis	Clonazepam Intranasal Spray	Orphan Designation	-

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