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Boehringer Ingelheim Pharmaceuticals, Inc. — Rare Disease Pipeline | Kisho | Kisho

Boehringer Ingelheim Pharmaceuticals, Inc.

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This pharmaceutical company is engaged in developing treatments for rare diseases, with 30 orphan drug designations and active involvement in 77 rare conditions, including systemic sclerosis and diabetes.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
B-cell chronic lymphocytic leukemia	mouse-human chimeric antibody of the IgG1 isotype, directed against human CD37	Orphan Designation	-
Becker muscular dystrophy	derivative of 4?methoxy?5?phenoxy?2?(piperidine?1?carbonyl)pyridine	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	Gilotrif	Orphan Designation	-
DYRK1A-related intellectual disability syndrome	idarucizumab	Orphan Designation	-
Duchenne muscular dystrophy	derivative of 4?methoxy?5?phenoxy?2?(piperidine?1?carbonyl)pyridine	Orphan Designation	-
Netherton syndrome	Spesolimab-sbzo	Orphan Designation	-
Toriello-Lacassie-Droste syndrome	Gilotrif	Orphan Designation	-
Tourette syndrome	pramipexole	Orphan Designation	-
acute intermittent porphyria	Mobic	Orphan Designation	-
acute myeloid leukemia	human fully IgG1 antibody specific for CD33volasertib	Orphan Designation	-
alopecia-intellectual disability syndrome 2	Bromhexine	Orphan Designation	-
alveolar soft part sarcoma	Spiro[3H-Indole-3,2¿-pyrrolidin]-2(1H)-one derivative	Orphan Designation	-
anosmia for isobutyric acid	idarucizumabMobic	Orphan Designation	-
atypical teratoid rhabdoid tumor	afatinib	Orphan Designation	-
autoinflammation with arthritis and dyskeratosis	Gilotrif	Orphan Designation	-
autosomal recessive congenital ichthyosis 2	Gilotrif	Orphan Designation	-
biliary tract cancer	Spiro[3H-Indole-3,2¿-pyrrolidin]-2(1H)-one derivative	Orphan Designation	-
brain cancer	afatinib	Orphan Designation	-
brain glioma	afatinib	Orphan Designation	-
brain-lung-thyroid syndrome	Tiotropium bromide	Orphan Designation	-
brainstem cancer	afatinib	Orphan Designation	-
campomelic dysplasia	idarucizumab	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	GilotrifOFEV	Orphan Designation	-
central nervous system cancer	afatinib	Orphan Designation	-
cerebral ventricle cancer	afatinib	Orphan Designation	-
ceroid lipofuscinosis, neuronal, 6B (Kufs type)	Mobic	Orphan Designation	-
cervical cancer	Porfiromycin	Orphan Designation	-
chronic obstructive pulmonary disease	STRIVERDI RESPIMATCOMBIVENT RESPIMATSPIRIVA HANDIHALER	-	FDA Approved
congenital lethal erythroderma	Jascayd	Orphan Designation	-
cystic fibrosis	carbamic acid, [[4-[[3-[[4-[1-(4-hydroxyphenyl)-1-methyl-ethyl]phenoxy]methyl]phenyl]methoxy]-phenyl]iminomethyl]-,ethyl ester	Orphan Designation	-
diffuse cutaneous systemic sclerosis	OFEV	Orphan Designation	-
double nail for fifth toe	idarucizumabMobic	Orphan Designation	-
epidermolysis bullosa simplex 1C, localized	Mobic	Orphan Designation	-
familial partial lipodystrophy, Dunnigan type	idarucizumab	Orphan Designation	-
head injury	Porfiromycin	Orphan Designation	-
heart failure	JARDIANCE	-	FDA Approved
hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain	idarucizumab	Orphan Designation	-
hereditary coproporphyria	Mobic	Orphan Designation	-
idiopathic pulmonary fibrosis	Pyridine derivative containing pyridazine that inhibits lysophospholipaseJascaydOFEV	Orphan Designation	-
intellectual developmental disorder with autism and macrocephaly	idarucizumab	Orphan Designation	-
intellectual developmental disorder, X-linked, syndromic 37	Bromhexine	Orphan Designation	-
intestinal pseudoobstruction, neuronal, chronic idiopathic, X-linked	OFEV	Orphan Designation	-
juvenile idiopathic arthritis	Mobic	Orphan Designation	-
limited cutaneous systemic sclerosis	OFEV	Orphan Designation	-
limited systemic sclerosis	OFEV	Orphan Designation	-
linear nevus sebaceous syndrome	Gilotrif	Orphan Designation	-
lung adenocarcinoma	Gilotrif	Orphan Designation	-
lung cancer	Gilotrif	Orphan Designation	-
lymphedema-distichiasis syndrome	Tiotropium bromide	Orphan Designation	-
major depressive disorder	Jascayd	Orphan Designation	-
malignant pancreatic neoplasm	afatinib	Orphan Designation	-
malignant tumor of neck	Porfiromycin	Orphan Designation	-
mesothelioma	Nintedanib	Orphan Designation	-
modifier, X-linked, for Neurofunctional defects	Mobic	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	OFEV	Orphan Designation	-
myelodysplastic syndrome	Fully human IgG1 antibody specific for CD33	Orphan Designation	-
myelodysplastic/myeloproliferative neoplasm	Fully human IgG1 antibody specific for CD33	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	OFEV	Orphan Designation	-
nephronophthisis-like nephropathy 1	idarucizumab	Orphan Designation	-
neuroacanthocytosis	idarucizumab	Orphan Designation	-
neuroendocrine carcinoma	humanized IgG-like T cell engager (TcE) comprised of 2 polypeptide chains specific for human Delta-like 3 (DLL3) and human CD3	Orphan Designation	-
non-small cell lung carcinoma	Gilotrif	Orphan Designation	-
non-small cell squamous lung carcinoma	Gilotrif	Orphan Designation	-
post 5-alpha-reductase inhibitors treatment syndrome	Jascayd	Orphan Designation	-
post-treatment Lyme disease syndrome	Jascayd	Orphan Designation	-
pulmonary systemic sclerosis	OFEV	Orphan Designation	-
pyoderma gangrenosum	spesolimab-sbzo	Orphan Designation	-
small cell lung carcinoma	humanized IgG-like T cell engager (TcE) comprised of 2 polypeptide chains specific for human Delta-like 3 (DLL3) and human CD3 (BI 764532)	Orphan Designation	-
soft tissue sarcoma	Spiro[3H-Indole-3,2¿-pyrrolidin]-2(1H)-one derivative	Orphan Designation	-
spatial visualization, aptitude for	Mobic	Orphan Designation	-
systemic sclerosis	OFEVpyridine derivative containing pyridazine that inhibits lysophospholipaseDerivative of 6-[2-(pyridin-2-yl)phenoxy]methyl}-1,2,3,4-tetrahydroisoquinoline	Orphan Designation	FDA Approved
therapy-related myeloid neoplasm	Fully human IgG1 antibody specific for CD33	Orphan Designation	-
treatment-refractory schizophrenia	Jascayd	Orphan Designation	-
tumor suppressor gene on chromosome 11	Gilotrif	Orphan Designation	-
type 1 diabetes mellitus	JENTADUETOTRADJENTA	-	FDA Approved
type 2 diabetes mellitus	JENTADUETOTRADJENTA	-	FDA Approved
undifferentiated pleomorphic sarcoma	Spiro[3H-Indole-3,2¿-pyrrolidin]-2(1H)-one derivative	Orphan Designation	-

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