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UCB, Inc. — Rare Disease Pipeline | Kisho | Kisho

UCB, Inc.

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Specializing in rare diseases, the company has 17 FDA-approved drugs and 13 orphan drug designations, addressing conditions such as childhood epilepsy and neurodegenerative disorders.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Dravet syndrome	Fintepla	Orphan Designation	-
Lennox-Gastaut syndrome	Fintepla	Orphan Designation	-
Pitt-Hopkins syndrome	NAYZILAM	Orphan Designation	-
Spondyloenchondrodysplasia with immune dysregulation	Rozanolixizumab	Orphan Designation	-
acquired purpura fulminans	Rozanolixizumab	Orphan Designation	-
autoimmune thrombocytopenic purpura	Rozanolixizumab	Orphan Designation	-
childhood absence epilepsy	brivaracetam	Orphan Designation	-
chronic primary adrenal insufficiency	NAYZILAM	Orphan Designation	-
congenital myasthenic syndrome 3A	ZilbrysqRystiggo	Orphan Designation	-
congenital myasthenic syndrome 4C	ZilbrysqRystiggo	Orphan Designation	-
congenital myasthenic syndrome 5	ZilbrysqRystiggo	Orphan Designation	-
congenital myasthenic syndrome 6	ZilbrysqRystiggo	Orphan Designation	-
developmental and epileptic encephalopathy 119	NAYZILAM	Orphan Designation	-
dihydropyrimidine dehydrogenase deficiency	Kygevvi	Orphan Designation	-
dihydropyrimidinuria	Kygevvi	Orphan Designation	-
early-onset partial cataract	BRIVIACTKEPPRA	-	FDA Approved
febrile seizures, familial	fenfluramine hydrochlorideFintepla	Orphan Designation	-
immunodeficiency 14	seletalisib	Orphan Designation	-
juvenile absence epilepsy	brivaracetam	Orphan Designation	-
mitochondrial DNA depletion syndrome 1	Kygevvi	Orphan Designation	-
mitochondrial DNA depletion syndrome, myopathic form	Kygevvi	Orphan Designation	-
mitochondrial neurogastrointestinal encephalomyopathy	Kygevvi	Orphan Designation	-
multiple congenital anomalies-hypotonia-seizures syndrome 1	NAYZILAM	Orphan Designation	-
multiple congenital anomalies-hypotonia-seizures syndrome 2	NAYZILAM	Orphan Designation	-
multiple congenital anomalies-hypotonia-seizures syndrome 3	NAYZILAM	Orphan Designation	-
myasthenia gravis	ZilbrysqRystiggo	Orphan Designation	-
myasthenic syndrome, congenital, 1B, fast-channel	ZilbrysqRystiggo	Orphan Designation	-
neurodegeneration with developmental delay, early respiratory failure, myoclonic seizures, and brain abnormalities	KEPPRA	-	FDA Approved
pediatric acute-onset neuropsychiatric syndrome	Kygevvi	Orphan Designation	-
pediatric hepatocellular carcinoma	Kygevvi	Orphan Designation	-
pediatric ovarian dysgerminoma	Kygevvi	Orphan Designation	-
pediatric-onset Graves disease	Kygevvi	Orphan Designation	-
progressive supranuclear palsy	recombinant humanized IgG4P monoclonal antibody with specificity for human tau	Orphan Designation	-
progressive supranuclear palsy-corticobasal syndrome	recombinant humanized IgG4P monoclonal antibody with specificity for human tau	Orphan Designation	-
progressive supranuclear palsy-parkinsonism syndrome	recombinant humanized IgG4P monoclonal antibody with specificity for human tau	Orphan Designation	-

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