Kisho

Ipsen Biopharmaceuticals, Inc. — Rare Disease Pipeline | Kisho | Kisho

Ipsen Biopharmaceuticals, Inc.

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Specializing in treatments for rare diseases, the company has six FDA-approved drugs and 11 orphan drug designations, actively addressing 34 rare conditions, including primary biliary cholangitis and dermatomyositis.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Alagille syndrome	Bylvay	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	Somatuline Depotirinotecan liposome injection	Orphan Designation	-
Char syndrome	irinotecan liposome injection	Orphan Designation	-
Dowling-Degos disease 4	Bylvay	Orphan Designation	-
Huntington disease	phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis (1,1-dimethyethyl) monohydrochloride	Orphan Designation	-
Pelizaeus-Merzbacher disease in female carriers	Dysport	Orphan Designation	-
Wolf-Hirschhorn syndrome	Sohonos	Orphan Designation	-
adult embryonal tumor with multilayered rosettes, c19mc-altered	lanreotide acetate	Orphan Designation	-
arthrogryposis, renal dysfunction, and cholestasis 2	Bylvay	Orphan Designation	-
ataxia-telangiectasia-like disorder 1	Dysport	Orphan Designation	-
autoimmune disorder of the nervous system	Sohonos	Orphan Designation	-
benign essential blepharospasm	Botulinum toxin type A	Orphan Designation	-
benign recurrent intrahepatic cholestasis	Bylvay	Orphan Designation	-
biliary atresia	odevixibat	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	Somatuline Depotirinotecan liposome injection	Orphan Designation	-
carcinoid syndrome	lanreotide acetate	Orphan Designation	-
cataract 13 with adult I phenotype	lanreotide acetate	Orphan Designation	-
cutaneous neuroendocrine carcinoma	Somatuline Depot	Orphan Designation	-
dermatomyositis	lanreotide acetate	Orphan Designation	-
erythrocytosis, familial, 4	Bylvay	Orphan Designation	-
fibrodysplasia ossificans progressiva	Sohonos	Orphan Designation	-
hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain	Sohonos	Orphan Designation	-
hereditary sensory and autonomic neuropathy type 7	Bylvay	Orphan Designation	-
hereditary spastic paraplegia 30	Dysport	Orphan Designation	-
hereditary spastic paraplegia 46	Dysport	Orphan Designation	-
hypotrichosis 6	Bylvay	Orphan Designation	-
malignant pancreatic neoplasm	irinotecan liposome injection	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	irinotecan liposome injection	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	Somatuline Depotirinotecan liposome injection	Orphan Designation	-
nut midline carcinoma	lanreotide acetate	Orphan Designation	-
pancreatic neuroendocrine neoplasm	Somatuline Depot	Orphan Designation	-
pancreatic neuroendocrine tumor	Somatuline Depot	Orphan Designation	-
primary biliary cholangitis	IQIRVO(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid	Orphan Designation	FDA Approved
progressive familial intrahepatic cholestasis	Bylvay	Orphan Designation	-

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