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Bristol-Myers Squibb Company — Rare Disease Pipeline | Kisho | Kisho

Bristol-Myers Squibb Company

Other

Orphan Designations

FDA Approvals

105

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
AIDS	Dideoxyinosine	Orphan Designation	-
Alstrom syndrome	OpdualagOpdivo	Orphan Designation	-
B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)	Sprycel	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	OpdivoOPDIVO	Orphan Designation	-
COG7-congenital disorder of glycosylation	Opdivo	Orphan Designation	-
Char syndrome	OpdivoOPDIVO	Orphan Designation	-
Finnish type amyloidosis	OpdualagOpdivoYervoy	Orphan Designation	-
Gaucher disease type III	AugtyroOpdivo	Orphan Designation	-
H syndrome	Yervoy	Orphan Designation	-
Hodgkins lymphoma	OPDIVOHuman anti-CD30 monoclonal antibody	Orphan Designation	-
Kabuki syndrome 1	Opdivo	Orphan Designation	-
Kaposi's sarcoma	Nivolumab and Ipilimumab	Orphan Designation	-
Mallory-Weiss syndrome	Fixed dose combination of relatlimab and nivolumabNivolumab and Ipilimumab	Orphan Designation	-
Moyamoya disease	Opdivo	Orphan Designation	-
NTRK fusion positive cancer	Augtyro	Orphan Designation	-
Niemann-Pick disease type B	Opdivo	Orphan Designation	-
Philadelphia-positive myelogenous leukemia	Sprycel	Orphan Designation	-
Smith-Lemli-Opitz syndrome	Opdivo	Orphan Designation	-
X-linked Alport syndrome	Yervoy	Orphan Designation	-
acute disseminated encephalomyelitis	Orencia	Orphan Designation	-
acute flaccid myelitis	Orencia	Orphan Designation	-
acute graft versus host disease	Orencia	Orphan Designation	-
acute lymphoblastic leukemia	Orencia	Orphan Designation	-
acute myeloid leukemia	OrenciaulocuplumabONUREG	Orphan Designation	-
adult embryonal tumor with multilayered rosettes, c19mc-altered	Sprycel	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	OPDIVO® and YERVOY®OPDIVOEmplicitiONUREG	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	OpdualagOPDIVO® and YERVOY®OPDIVOEmplicitiONUREG	Orphan Designation	-
adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy	Sprycel	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	KrazatiOPDIVO® and YERVOY®AugtyroOPDIVOEmplicitiONUREG	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	OPDIVO® and YERVOY®OPDIVOEmplicitiONUREG	Orphan Designation	-
anal carcinoma	nivolumab	Orphan Designation	-
ataxia and polyneuropathy, adult-onset	Opdivo	Orphan Designation	-
autosomal dominant epilepsy with auditory features	Sprycel	Orphan Designation	-
autosomal dominant progressive nephropathy with hypertension	Sprycel	Orphan Designation	-
benign soft tissue neoplasm	ifosfamide	Orphan Designation	-
bone sarcoma	ifosfamide	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	OpdivoOPDIVOAcetylcysteine	Orphan Designation	-
cholestasis-pigmentary retinopathy-cleft palate syndrome	Nivolumab and Ipilimumab	Orphan Designation	-
chronic myelogenous leukemia, BCR-ABL1 positive	Sprycel17-allylamino-17-demethoxygeldanamycin (17-AGG)	Orphan Designation	-
congenital anomalies of kidney and urinary tract 3	Acetylcysteine	Orphan Designation	-
congenital lethal erythroderma	Krazati	Orphan Designation	-
craniosynostosis, Philadelphia type	Sprycel	Orphan Designation	-
cystic fibrosis	Sprycel	Orphan Designation	-
deafness, congenital, and adult-onset progressive leukoencephalopathy	AugtyroOpdivo	Orphan Designation	-
developmental and epileptic encephalopathy, 58	Augtyro	Orphan Designation	-
esophageal adenocarcinoma	Opdivo	Orphan Designation	-
esophageal cancer	Nivolumab and IpilimumabOpdivo	Orphan Designation	-
extramedullary soft tissue plasmacytoma	ifosfamide	Orphan Designation	-
gastric cancer	(7S)-3-Amino-14-ethyl-11-fluoro-7-methyl-4-oxo-4,5,6,7,13,14-hexahydro-1,15-ethenopyrazolo[4,3-f][1,4,8,10]benzoxatriazacyclotridecine-12-carbonitrileFixed dose combination of relatlimab and nivolumabOpdivoNivolumab and Ipilimumab	Orphan Designation	-
gastric carcinoma	Fixed dose combination of relatlimab and nivolumab	Orphan Designation	-
gastroesophageal junction adenocarcinoma	Fixed dose combination of relatlimab and nivolumab	Orphan Designation	-
geniculate herpes zoster	Sorivudine	Orphan Designation	-
glioblastoma	nivolumab	Orphan Designation	-
graft versus host disease	Orencia	Orphan Designation	-
hepatocellular carcinoma	Opdivobrivanib alaninate	Orphan Designation	-
hereditary diffuse gastric adenocarcinoma	Fixed dose combination of relatlimab and nivolumab	Orphan Designation	-
hereditary sensory and autonomic neuropathy	Augtyro	Orphan Designation	-
herpes zoster	Sorivudine	Orphan Designation	-
herpes zoster with dermatitis of eyelid	Sorivudine	Orphan Designation	-
high anorectal malformation	Yervoy	Orphan Designation	-
high-grade B-cell lymphoma double-hit/triple-hit	Yervoy	Orphan Designation	-
hyperopia, high	Yervoy	Orphan Designation	-
idiopathic pulmonary fibrosis	(1S,3S)-3-((2-methyl-6-(1-methyl-5-(((methyl(propyl)carbamoyl)oxy)methyl)-1H-1,2,3-triazol-4-yl)pyridin-3-yl)oxy)cyclohexane-1-carboxylic acid(R)-1-phenylethyl-5-(4-biphenyl-4-cyclopropanecarboxylic acid)-3-methylisoxazole-4-yl carbamate sodium salt	Orphan Designation	-
lung adenocarcinoma	Augtyro	Orphan Designation	-
lung cancer	Augtyro	Orphan Designation	-
lysinuric protein intolerance	OpdualagOpdivo	Orphan Designation	-
malignant carotid body paraganglioma	Krazati	Orphan Designation	-
malignant pancreatic neoplasm	selective small molecule inhibitor of the PRMT5/MTA complex	Orphan Designation	-
mediastinal soft tissue cancer	ifosfamide	Orphan Designation	-
mesothelioma	OPDIVO® and YERVOY®nivolumab	Orphan Designation	-
microinvasive gastric cancer	Fixed dose combination of relatlimab and nivolumab	Orphan Designation	-
multiple organ dysfunction syndrome	Nulojix	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	OpdivoOPDIVO	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	OpdivoOPDIVO	Orphan Designation	-
neuroacanthocytosis	Acetylcysteine	Orphan Designation	-
non-small cell lung carcinoma	Augtyro	Orphan Designation	-
non-small cell squamous lung carcinoma	Augtyro	Orphan Designation	-
nut midline carcinoma	Sprycel	Orphan Designation	-
obesity, hyperphagia, and developmental delay	Augtyro	Orphan Designation	-
ovarian cancer	Taxol isolated from Taxus brevifolia	Orphan Designation	-
pediatric acute-onset neuropsychiatric syndrome	Opdualag	Orphan Designation	-
pediatric hepatocellular carcinoma	Opdualag	Orphan Designation	-
pediatric ovarian dysgerminoma	Opdualag	Orphan Designation	-
pediatric ovarian germ cell tumor	Opdualag	Orphan Designation	-
pediatric-onset Graves disease	Opdualag	Orphan Designation	-
plasma cell myeloma	EmplicitiTanespimycin	Orphan Designation	-
polycystic kidney disease 2	Sprycel	Orphan Designation	-
post 5-alpha-reductase inhibitors treatment syndrome	Krazati	Orphan Designation	-
post-treatment Lyme disease syndrome	Krazati	Orphan Designation	-
primary adult heart tumor	OPDIVO® and YERVOY®OPDIVOEmplicitiONUREG	Orphan Designation	-
primary mediastinal large B-cell lymphoma	nivolumab	Orphan Designation	-
primary organ-specific lymphoma	Nulojix	Orphan Designation	-
sensory organ benign neoplasm	Nulojix	Orphan Designation	-
severe nonproliferative diabetic retinopathy	Yervoy	Orphan Designation	-
sickle cell disease	fetal hemoglobin activator cereblon (CRBN) E3 ligase-modulating drug	Orphan Designation	-
skeletal dysplasia, mild, with joint laxity and advanced bone age	Opdivo	Orphan Designation	-
small cell lung carcinoma	atigotatug plus nivolumab fixed dose combinationipilimumabOPDIVO	Orphan Designation	-
soft tissue amyloid neoplasm	ifosfamide	Orphan Designation	-
soft tissue chondroma	ifosfamide	Orphan Designation	-
soft tissue neoplasm	ifosfamide	Orphan Designation	-
stage I endometrioid carcinoma	Yervoy	Orphan Designation	-
stage II endometrioid carcinoma	Yervoy	Orphan Designation	-
stage IVb bladder cancer	Yervoy	Orphan Designation	-
treatment-refractory schizophrenia	Krazati	Orphan Designation	-
tumor suppressor gene on chromosome 11	Augtyro	Orphan Designation	-

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