Kisho

Takeda Development Center Americas, Inc. — Rare Disease Pipeline | Kisho | Kisho

Takeda Development Center Americas, Inc.

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
ALK+ histiocytosis	Alunbrig	Orphan Designation	-
ALK-negative anaplastic large cell lymphoma	Alunbrig	Orphan Designation	-
ALK-positive large B-cell lymphoma	Alunbrig	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	Agrylin	Orphan Designation	-
CDKL5 disorder	Balipodect	Orphan Designation	-
CINCA syndrome	HyQvia	Orphan Designation	-
Char syndrome	AgrylinAmatine	Orphan Designation	-
Dravet syndrome	cholesterol 24S-hydroxylase inhibitor	Orphan Designation	-
Finnish type amyloidosis	6-amino-5-chloro-N-[1R)-1-[5-[[[5-hloro-4-(trifluoromethyl)-2pyridinyl]amino]carbonyl]-2-thiazoyl]ethyl]-4-pyrimdinecarboxamide	Orphan Designation	-
Friedreich ataxia	D-amino acid oxidase inhibitor	Orphan Designation	-
Friedreich ataxia 1	D-amino acid oxidase inhibitor	Orphan Designation	-
Friedreich ataxia 2	D-amino acid oxidase inhibitor	Orphan Designation	-
Gaucher disease	VPRIV	Orphan Designation	-
Gaucher disease type I	VPRIV	Orphan Designation	-
Kabuki syndrome	5-{(1R,2R)-2-[(Cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride	Orphan Designation	-
Kabuki syndrome 1	darvadstrocel	Orphan Designation	-
Lennox-Gastaut syndrome	cholesterol 24S-hydroxylase inhibitor	Orphan Designation	-
Opitz G/BBB syndrome	darvadstrocel	Orphan Designation	-
RFT1-congenital disorder of glycosylation	OBIZUR	Orphan Designation	-
Stormorken syndrome	OBIZUR	Orphan Designation	-
Yunis-Varon syndrome	Alunbrig	Orphan Designation	-
acquired polycythemia vera	rusfertide	Orphan Designation	-
acrocardiofacial syndrome	darvadstrocel	Orphan Designation	-
acute disseminated encephalomyelitis	C1 esterase inhibitor (Human)Firazyr	Orphan Designation	-
acute flaccid myelitis	Firazyr	Orphan Designation	-
acute lymphoblastic leukemia	Firazyr	Orphan Designation	-
acute myeloid leukemia	MLN4924-Inhibitor of Nedd8-activating enzyme (NAE)	Orphan Designation	-
adrenocortical insufficiency	hydrocortisone modified release tabs	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	Exkivity	Orphan Designation	-
agammaglobulinemia 8b, autosomal recessive	C1 esterase inhibitor (Human)	Orphan Designation	-
alpha 1-antitrypsin deficiency	double stranded oligomer ADS-001 RNA interference-based liver targeted therapeutic	Orphan Designation	-
anaplastic large cell lymphoma	Alunbrig	Orphan Designation	-
angioedema	CinryzeFirazyr	Orphan Designation	-
autism	Cinryze	Orphan Designation	-
bleeding disorder, platelet-type, 22	OBIZUR	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	AgrylinAmatine	Orphan Designation	-
central nervous system anaplastic large cell lymphoma	Alunbrig	Orphan Designation	-
cholecystitis	resatorvid	Orphan Designation	-
chronic beryllium disease	resatorvid	Orphan Designation	-
chronic inflammatory demyelinating polyradiculoneuropathy	HyQvia	Orphan Designation	-
chronic relapsing inflammatory optic neuropathy	HyQvia	Orphan Designation	-
cleidocranial dysplasia 1	Lialda	Orphan Designation	-
dentin dysplasia type I	VPRIV	Orphan Designation	-
epilepsy, familial adult myoclonic, 2	HyQvia	Orphan Designation	-
esophageal cancer	2-[(2S)-1-azabicyclo[2.2.2]oct-2-yl]-6-(3-methyl-1H-pyrazol-4-yl)thieno[3,2-d]pyrimidin-4(3H)-one hemihydrate	Orphan Designation	-
essential thrombocythemia	FirazyrAgrylin	Orphan Designation	-
familial thrombocytosis	Firazyr	Orphan Designation	-
fragile X syndrome	Balipodect	Orphan Designation	-
frontometaphyseal dysplasia 2	Lialda	Orphan Designation	-
graft versus host disease	Vedolizumabvedolizumab	Orphan Designation	-
hemophilia A	Adeno-associated virus serotype 8 vector expressing B domain deleted codon optimized factor VIII	Orphan Designation	-
hemophilia B	adeno associated virus with modified transthyretin and sequence encoding factor IX variant gene	Orphan Designation	-
hereditary pulmonary alveolar proteinosis	C1 esterase inhibitor (Human)	Orphan Designation	-
heterotaxy, visceral, 1, X-linked	Ninlaro	Orphan Designation	-
inflammatory bowel disease (infantile ulcerative colitis) 31, autosomal recessive	Lialda	Orphan Designation	-
inflammatory bowel disease 1	Lialda	Orphan Designation	-
intellectual developmental disorder, X-linked, syndromic, Pilorge type	Cinryze	Orphan Designation	-
intellectual disability-severe speech delay-mild dysmorphism syndrome	Cinryze	Orphan Designation	-
lung adenocarcinoma	Exkivity	Orphan Designation	-
lung cancer	Exkivity	Orphan Designation	-
metachromatic leukodystrophy	arylsulfatase A (rhASA)	Orphan Designation	-
metaphyseal chondrodysplasia, Jansen type	HyQvia	Orphan Designation	-
mucopolysaccharidosis-plus syndrome	Ninlaro	Orphan Designation	-
multiple congenital anomalies-hypotonia-seizures syndrome 2	Ninlaro	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	AgrylinAmatine	Orphan Designation	-
myasthenia gravis	Mezagitamab	Orphan Designation	-
myelodysplastic syndrome	MLN4924-Inhibitor of Nedd8-activating enzyme (NAE)	Orphan Designation	-
narcolepsy	methyl (2R,3S)-3-[(methylsulfonyl)amino]-2-{[(cis-4-phenylcyclohexyl)oxy]methyl}piperidine-1-carboxylate	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	AgrylinAmatine	Orphan Designation	-
neonatal inflammatory skin and bowel disease	HyQvia	Orphan Designation	-
non-small cell lung carcinoma	Exkivity	Orphan Designation	-
non-small cell squamous lung carcinoma	Exkivity	Orphan Designation	-
osteoporosis-pseudoglioma syndrome	Lialda	Orphan Designation	-
osteosarcoma	mifamuritide	Orphan Designation	-
pediatric acute-onset neuropsychiatric syndrome	Adzynma	Orphan Designation	-
pediatric hepatocellular carcinoma	Adzynma	Orphan Designation	-
pediatric ovarian dysgerminoma	Adzynma	Orphan Designation	-
pediatric ovarian germ cell tumor	Adzynma	Orphan Designation	-
pediatric-onset Graves disease	Adzynma	Orphan Designation	-
peripheral primitive neuroectodermal tumor	OBIZUR	Orphan Designation	-
plasma cell myeloma	Human IgG1 monoclonal antibody to human CD38Ninlaro	Orphan Designation	-
polyneuropathy, inflammatory demyelinating, chronic	HyQvia	Orphan Designation	-
pouchitis	vedolizumab	Orphan Designation	-
respiratory failure	resatorvid	Orphan Designation	-
sitosterolemia 1	OBIZUR	Orphan Designation	-
stage II endometrioid carcinoma	6-amino-5-chloro-N-[1R)-1-[5-[[[5-hloro-4-(trifluoromethyl)-2pyridinyl]amino]carbonyl]-2-thiazoyl]ethyl]-4-pyrimdinecarboxamide	Orphan Designation	-
stage IVb bladder cancer	6-amino-5-chloro-N-[1R)-1-[5-[[[5-hloro-4-(trifluoromethyl)-2pyridinyl]amino]carbonyl]-2-thiazoyl]ethyl]-4-pyrimdinecarboxamide	Orphan Designation	-
symptomatic form of hemophilia A in female carriers	Amatine	Orphan Designation	-
thrombocytopenia due to immune destruction	Mezagitamab	Orphan Designation	-
thrombotic thrombocytopenic purpura	Recombinant disintegrin and metalloprotease with thrombospondin type 1 motifsAdzynma	Orphan Designation	-
tumor suppressor gene on chromosome 11	Exkivity	Orphan Designation	-
tyrosinemia type I	VPRIV	Orphan Designation	-
ulcerative colitis	Lialda	Orphan Designation	-
vitelliform macular dystrophy 3	HyQvia	Orphan Designation	-
xanthinuria type I	VPRIV	Orphan Designation	-

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