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Takeda Pharmaceuticals U.S.A., Inc. — Rare Disease Pipeline | Kisho | Kisho

Takeda Pharmaceuticals U.S.A., Inc.

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
16p13.11 microdeletion syndrome	idursulfase IT	Orphan Designation	-
16p13.11 microduplication syndrome	idursulfase IT	Orphan Designation	-
3-methylglutaconic aciduria type 9	RIXUBIS	Orphan Designation	-
3-methylglutaconic aciduria, type VIIB	RIXUBIS	Orphan Designation	-
B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)	Iclusig	Orphan Designation	-
Eisenmenger syndrome	FEIBA	Orphan Designation	-
Fabry disease	Alpha-galactosidase A	Orphan Designation	-
Fanconi anemia complementation group B	FEIBA	Orphan Designation	-
Hurler syndrome	Elaprase	Orphan Designation	-
Kabuki syndrome 1	FEIBA	Orphan Designation	-
Kostmann syndrome	Vonvendi	Orphan Designation	-
Meacham syndrome	Velcade	Orphan Designation	-
Noonan syndrome 1	Velcade	Orphan Designation	-
PURA-related severe neonatal hypotonia-seizures-encephalopathy syndrome	Natpara	Orphan Designation	-
Pelizaeus-Merzbacher disease in female carriers	maribavir	Orphan Designation	-
Philadelphia-positive myelogenous leukemia	Iclusig	Orphan Designation	-
RFT1-congenital disorder of glycosylation	RIXUBIS	Orphan Designation	-
Stormorken syndrome	RIXUBIS	Orphan Designation	-
VISS syndrome	Eohilia	Orphan Designation	-
acute disseminated encephalomyelitis	Kalbitor	Orphan Designation	-
acute flaccid myelitis	Kalbitor	Orphan Designation	-
acute lymphoblastic leukemia	Kalbitor	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	IclusigGATTEX	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	IclusigGATTEX	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	IclusigGATTEX	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	IclusigGATTEX	Orphan Designation	-
anal canal adenocarcinoma	maribavir	Orphan Designation	-
aneurysm-osteoarthritis syndrome	Eohilia	Orphan Designation	-
angioedema	TakhzyroKalbitor	Orphan Designation	-
anti-MDA5 dermatomyositis	Livtencity	Orphan Designation	-
autism	FEIBA	Orphan Designation	-
autosomal recessive spinocerebellar ataxia 10	Gammagard Liquid	Orphan Designation	-
bleeding disorder, platelet-type, 22	RIXUBIS	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	VelcadeEohilia	Orphan Designation	-
ceroid lipofuscinosis, neuronal, 6A	Livtencity	Orphan Designation	-
chronic myelogenous leukemia, BCR-ABL1 positive	Iclusig	Orphan Designation	-
clinically amyopathic dermatomyositis	Livtencity	Orphan Designation	-
cytomegalovirus pneumonia	maribavir	Orphan Designation	-
cytomegalovirus retinitis	maribavir	Orphan Designation	-
endogenous depression	Livtencity	Orphan Designation	-
epilepsy, familial adult myoclonic, 2	Gammagard Liquid	Orphan Designation	-
essential thrombocythemia	Kalbitor	Orphan Designation	-
extramedullary plasmacytoma	Livtencity	Orphan Designation	-
familial thrombocytosis	Kalbitor	Orphan Designation	-
frontonasal dysplasia - severe microphthalmia - severe facial clefting syndrome	Vonvendi	Orphan Designation	-
has an isolated presentation	Natpara	Orphan Designation	-
hyperlipoproteinemia type 3	Vonvendi	Orphan Designation	-
hypoparathyroidism	Natpara	Orphan Designation	-
idiopathic disseminated cytomegalovirus infection	maribavir	Orphan Designation	-
indolent plasma cell myeloma	Velcade	Orphan Designation	-
intellectual developmental disorder, X-linked, syndromic, Pilorge type	FEIBA	Orphan Designation	-
intellectual disability-severe speech delay-mild dysmorphism syndrome	FEIBA	Orphan Designation	-
malignant carotid body paraganglioma	Eohilia	Orphan Designation	-
mantle cell lymphoma	Velcade	Orphan Designation	-
mitochondrial DNA depletion syndrome 11	Gammagard Liquid	Orphan Designation	-
mitochondrial DNA maintenance syndrome	Gammagard Liquid	Orphan Designation	-
mucopolysaccharidosis	Elaprase	Orphan Designation	-
multifocal motor neuropathy	Gammagard Liquid	Orphan Designation	-
multisystem inflammatory syndrome in children and adults	Livtencity	Orphan Designation	-
neuronal ceroid lipofuscinosis 1	LivtencityGammagard Liquid	Orphan Designation	-
neuronal ceroid lipofuscinosis 10	Gammagard Liquid	Orphan Designation	-
neuronal ceroid lipofuscinosis 2	Livtencity	Orphan Designation	-
neuronal ceroid lipofuscinosis 5	LivtencityGammagard Liquid	Orphan Designation	-
non-secretory plasma cell myeloma	Velcade	Orphan Designation	-
osteogenesis imperfecta type 3	Vonvendi	Orphan Designation	-
osteosclerotic plasma cell myeloma	Velcade	Orphan Designation	-
peripheral primitive neuroectodermal tumor	RIXUBIS	Orphan Designation	-
plasma cell myeloma	Velcade	Orphan Designation	-
polydactyly of an index finger	Natpara	Orphan Designation	-
polydactyly of an index finger, bilateral	Natpara	Orphan Designation	-
primary adult heart tumor	IclusigGATTEX	Orphan Designation	-
refractory malignant neoplasm	Livtencity	Orphan Designation	-
renal hypomagnesemia 3	Vonvendi	Orphan Designation	-
secondary short bowel syndrome	Natpara	Orphan Designation	-
short bowel syndrome	GATTEX	Orphan Designation	-
sickle cell disease	rADAMTS13 / apadamtase alfa (INN)	Orphan Designation	-
sitosterolemia 1	RIXUBIS	Orphan Designation	-
smoldering plasma cell myeloma	Velcade	Orphan Designation	-
spatial visualization, aptitude for	idursulfase IT	Orphan Designation	-
taurodontism	maribavir	Orphan Designation	-
thrombocytopenia-absent radius syndrome	Eohilia	Orphan Designation	-
treatment-refractory schizophrenia	Eohilia	Orphan Designation	-
von Willebrand disease (hereditary or acquired)	Vonvendi	Orphan Designation	-

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