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Bristol-Myers Squibb Pharmaceutical Research Institute — Rare Disease Pipeline | Kisho | Kisho

Bristol-Myers Squibb Pharmaceutical Research Institute

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
15q11q13 microduplication syndrome	Droxia	Orphan Designation	-
AIDS related complex	Taxol	Orphan Designation	-
AIDS-related disorder	Taxol	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	Megace	Orphan Designation	-
Char syndrome	Megace	Orphan Designation	-
Cronkhite-Canada syndrome	Megace	Orphan Designation	-
Kabuki syndrome 1	Droxia	Orphan Designation	-
Kaposi's sarcoma	Taxol	Orphan Designation	-
acute kidney injury	Gusperimus	Orphan Designation	-
acute kidney tubular necrosis	Gusperimus	Orphan Designation	-
acute papillary necrosis	Gusperimus	Orphan Designation	-
alveolar capillary dysplasia with misalignment of pulmonary veins	Droxia	Orphan Designation	-
anorexia nervosa	Megace	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	Megace	Orphan Designation	-
cutis laxa with severe pulmonary, gastrointestinal and urinary anomalies	Droxia	Orphan Designation	-
early response to neural induction gene	Vumon for injection	Orphan Designation	-
facial dysmorphism-anorexia-cachexia-eye and skin anomalies syndrome	Megace	Orphan Designation	-
malignant atrophic papulosis	Blenoxane	Orphan Designation	-
malignant pleural mesothelioma	Blenoxane	Orphan Designation	-
malignant pleural solitary fibrous tumor	Blenoxane	Orphan Designation	-
malignant testicular germ cell tumor	Ifex	Orphan Designation	-
mixed testicular germ cell cancer	Ifex	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	Megace	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	Megace	Orphan Designation	-
neurodevelopmental disorder with or without anomalies of the brain, eye, or heart	Droxia	Orphan Designation	-
neurodevelopmental-craniofacial syndrome with variable renal and cardiac abnormalities	Droxia	Orphan Designation	-
prepubertal anorexia nervosa	Megace	Orphan Designation	-
refractory celiac disease	Vumon for injection	Orphan Designation	-
refractory cytopenia of childhood	Vumon for injection	Orphan Designation	-
refractory hairy cell leukemia	Vumon for injection	Orphan Designation	-
refractory plasma cell neoplasm	Vumon for injection	Orphan Designation	-
spastic paraplegia 93, autosomal recessive	Droxia	Orphan Designation	-
spinocerebellar ataxia, autosomal recessive 33	Droxia	Orphan Designation	-
syndromic X-linked intellectual disability 5	Droxia	Orphan Designation	-
testicular cancer	Ifex	Orphan Designation	-
testicular germ cell tumor	Ifex	Orphan Designation	-
testicular non-seminomatous germ cell cancer	Ifex	Orphan Designation	-
testis refractory cancer	IfexVumon for injection	Orphan Designation	-

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