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Horizon Therapeutics Ireland DAC — Rare Disease Pipeline | Kisho | Kisho

Horizon Therapeutics Ireland DAC

Other

4

Orphan Designations

0

FDA Approvals

15

Rare Diseases

0

News Articles

Disease	Drug(s)	Designation	Approved
Alstrom syndrome	teprotumumab	Orphan Designation	-
active cochlear Meniere disease	teprotumumab	Orphan Designation	-
active cochleovestibular Meniere disease	teprotumumab	Orphan Designation	-
active peptic ulcer disease	teprotumumab	Orphan Designation	-
anophthalmia/microphthalmia-esophageal atresia syndrome	2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid	Orphan Designation	-
arrhythmogenic cardiomyopathy with variable ectodermal abnormalities	2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid	Orphan Designation	-
endocrine exophthalmos	teprotumumab	Orphan Designation	-
glycogen storage disease Ib	Krystexxa	Orphan Designation	-
intestinal pseudoobstruction, neuronal, chronic idiopathic, X-linked	2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid	Orphan Designation	-
microphthalmia with brain and digit anomalies	Krystexxa	Orphan Designation	-
neuromyelitis optica	Uplizna	Orphan Designation	-
neuromyelitis optica spectrum disorder with anti-AQP4 antibodies	Uplizna	Orphan Designation	-
neuromyelitis optica spectrum disorder with anti-MOG antibodies	Uplizna	Orphan Designation	-
polycystic kidney disease 6 with or without polycystic liver disease	Krystexxa	Orphan Designation	-
tubulointerstitial kidney disease, autosomal dominant, 2	Krystexxa	Orphan Designation	-

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