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Amgen Inc — Rare Disease Pipeline | Kisho | Kisho

Amgen Inc

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A biopharmaceutical company focused on developing therapies for rare diseases, with 23 FDA-approved drugs and 67 orphan drug designations across 144 rare conditions.

Orphan Designations

FDA Approvals

144

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
7q11.23 microduplication syndrome	Neupogen	Orphan Designation	-
Adams-Oliver syndrome 1	Neupogen	Orphan Designation	-
Angelman syndrome	Repatha	Orphan Designation	-
B-cell chronic lymphocytic leukemia	Blinatumomab	Orphan Designation	-
B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)	Blincyto	Orphan Designation	-
Behcet disease	Otezla XR	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	XgevaFilgrastim	Orphan Designation	-
Char syndrome	Filgrastim	Orphan Designation	-
DYRK1A-related intellectual disability syndrome	NeupogenAncestim	Orphan Designation	-
Diamond-Blackfan anemia 11	Neupogen	Orphan Designation	-
Down syndrome	Nplate	Orphan Designation	-
Eiken syndrome	Recombinant methionyl human stem cell factor	Orphan Designation	-
Ewing sarcoma	a novel humanized bispecific XmAb T cell recruiting antibody cross-reactive to human and nonhuman primate STEAP1 and CD3	Orphan Designation	-
IgE responsiveness, atopic	Neupogen	Orphan Designation	-
Kleefstra syndrome 1	Neupogen	Orphan Designation	-
Noonan syndrome 2	Nplate	Orphan Designation	-
Noonan syndrome 3	Lumakras	Orphan Designation	-
Philadelphia-positive myelogenous leukemia	Blincyto	Orphan Designation	-
Shashi-Pena syndrome	Neupogen	Orphan Designation	-
Tessadori-Van Haaften neurodevelopmental syndrome 4	Neupogen	Orphan Designation	-
Toriello-Lacassie-Droste syndrome	LumakrasXgeva	Orphan Designation	-
acute flaccid myelitis	Gavilimomab	Orphan Designation	-
acute graft versus host disease	Gavilimomab	Orphan Designation	-
acute lymphoblastic leukemia	blinatumomabcarfilzomibBlincytoGavilimomab	Orphan Designation	-
acute myeloid leukemia	bispecific T cell engager antibody construct with a half-life extension Fc moiety capable of binding to the neonatal Fc receptorbispecific T-cell engager (BiTE) antibody targeting CD33 and CD3adoptive, anti-FLT3 chimeric antigen receptor T cell therapy generated through transduction of autologous T-cells with a self-inactivating human immunodeficiency virus-1 based lentiviral vectorA small molecule inhibitor of the pro-survival protein Myeloid Cell Leukemia sequence 1 (MCL 1)	Orphan Designation	-
acute myeloid leukemia, KRAS gene mutation	Lumakras	Orphan Designation	-
acute sensory ataxic neuropathy	Repatha	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	Otezla XR	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	Otezla XR	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	LumakrasImdelltraOtezla XR	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	Otezla XR	Orphan Designation	-
amyotrophic lateral sclerosis	Recombinant methionyl brain-derived neurotrophic factor	Orphan Designation	-
anemia	Epogen	Orphan Designation	-
anosmia for isobutyric acid	Pegylated recombinant human megakaryocyte growth and development factorNeupogenAncestim	Orphan Designation	-
aortic valve disease 1	Repatha	Orphan Designation	-
aortic valve stenosis	Repatha	Orphan Designation	-
aplastic anemia	Romiplostim	Orphan Designation	-
aromatase excess syndrome	NplateNeulastaNeupogen	Orphan Designation	-
arteriovenous malformations of the brain	Lumakras	Orphan Designation	-
atypical teratoid rhabdoid tumor	Epogen	Orphan Designation	-
autoimmune thrombocytopenic purpura	Nplate	Orphan Designation	-
autosomal recessive osteopetrosis 1	XGEVA	Orphan Designation	-
autosomal recessive osteopetrosis 3	Recombinant methionyl human stem cell factor	Orphan Designation	-
autosomal recessive osteopetrosis 5	XGEVA	Orphan Designation	-
biliary tract cancer	small molecule PRMT5/MTA cooperative inhibitor	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	NplateXgevaFilgrastim	Orphan Designation	-
cardiofaciocutaneous syndrome 2	Lumakras	Orphan Designation	-
cardiomyopathy	CORLANOR	-	FDA Approved
chemotherapy-induced alopecia	romiplostim	Orphan Designation	-
chemotherapy-induced hypertension	romiplostim	Orphan Designation	-
chemotherapy-induced toxicity	romiplostim	Orphan Designation	-
chronic lymphoproliferative disorder of NK-cells	blinatumomab	Orphan Designation	-
chronic myelogenous leukemia, BCR-ABL1 positive	Blincyto	Orphan Designation	-
chronic primary adrenal insufficiency	Sensipar	Orphan Designation	-
combined hepatocellular carcinoma and cholangiocarcinoma	small molecule PRMT5/MTA cooperative inhibitor	Orphan Designation	-
combined small cell lung carcinoma	Imdelltra	Orphan Designation	-
complex cortical dysplasia with other brain malformations	Repatha	Orphan Designation	-
complex cortical dysplasia with other brain malformations 1	Repatha	Orphan Designation	-
complex cortical dysplasia with other brain malformations 2	Repatha	Orphan Designation	-
complex cortical dysplasia with other brain malformations 4	Repatha	Orphan Designation	-
congenital bile acid synthesis defect 1	Recombinant secretory leucocyte protease inhibitor	Orphan Designation	-
congenital disorder of deglycosylation 1	Recombinant secretory leucocyte protease inhibitor	Orphan Designation	-
congenital sucrase-isomaltase deficiency	Recombinant secretory leucocyte protease inhibitor	Orphan Designation	-
cystic fibrosis	Recombinant secretory leucocyte protease inhibitor	Orphan Designation	-
diarrheal disease secondary to increased bowel motility	NeulastaNeupogen	Orphan Designation	-
diffuse large B-cell lymphoma	blinatumomab	Orphan Designation	-
dilated cardiomyopathy	CORLANOR	-	FDA Approved
discrimination, Two-point, reduction 1N	Neupogen	Orphan Designation	-
distal biliary tract carcinoma	small molecule PRMT5/MTA cooperative inhibitor	Orphan Designation	-
double nail for fifth toe	Pegylated recombinant human megakaryocyte growth and development factorNeupogen	Orphan Designation	-
ear antitragus, tag at base of	Epogen	Orphan Designation	-
ectopia pupillae	Filgrastim	Orphan Designation	-
familial hypocalciuric hypercalcemia 1	XGEVASensipar	Orphan Designation	-
familial hypocalciuric hypercalcemia 3	Sensipar	Orphan Designation	-
fibrous dysplasia	Neupogen	Orphan Designation	-
fire ant poisoning	Epogen	Orphan Designation	-
gastric cancer	half-life extended bispecific T cell engager (BiTE®) antibody construct designed to target both CLDN18.2 and CD3half-life extended bispecific T-cell engager antibody construct targeting MUC17 and CD3bemarituzumabrilotumumab	Orphan Designation	-
gastrointestinal stromal tumor	N-(2,3-dihydro-3,3-dimethyl-1H-indol-6-yl)-2-[(4-pyridinylmethyl)amino]-3-pyridinecarboxamide, phosphate (1:2)	Orphan Designation	-
glioblastoma	anti-EGFRvIII x anti-CD3 bispecific T cell engager antibody construct	Orphan Designation	-
graft versus host disease	interleukin mutein fused to the C-terminus of an immunoglobulin G Fc domain by way of a G4S linker	Orphan Designation	-
hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain	Pegylated recombinant human megakaryocyte growth and development factorNeupogen	Orphan Designation	-
homozygous familial hypercholesterolemia	Repatha	Orphan Designation	-
hypercalcemia, infantile, 2	XGEVASensipar	Orphan Designation	-
hyperparathyroidism 4	Sensipar	Orphan Designation	-
inclusion body myopathy and brain white matter abnormalities	Xgeva	Orphan Designation	-
indolent B-cell non-Hodgkin lymphoma	blinatumomab	Orphan Designation	-
indolent plasma cell myeloma	blinatumomab	Orphan Designation	-
indolent primary cutaneous B-cell lymphoma	blinatumomab	Orphan Designation	-
indolent primary cutaneous T-cell lymphoma	blinatumomab	Orphan Designation	-
insensitivity to pain with hyperplastic Myelinopathy	XGEVA	Orphan Designation	-
liver diffuse large B-cell lymphoma	blinatumomab	Orphan Designation	-
lung cancer	Imdelltra	Orphan Designation	-
malignant carotid body paraganglioma	blinatumomab	Orphan Designation	-
malignant pancreatic neoplasm	small molecule MTAP-cooperative PRMT5 inhibitor	Orphan Designation	-
melanoma	Imlygic	-	FDA Approved
metaphyseal chondrodysplasia, Jansen type	Sensipar	Orphan Designation	-
mismatch repair cancer syndrome 1	blinatumomab	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	XgevaFilgrastim	Orphan Designation	-
myelodysplastic syndrome	Filgrastim	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	XgevaFilgrastim	Orphan Designation	-
nephropathic cystinosis	Recombinant methionyl human stem cell factor	Orphan Designation	-
neuroacanthocytosis	NeupogenAncestim	Orphan Designation	-
neurodevelopmental disorder with coarse facies and mild distal skeletal abnormalities	Xgeva	Orphan Designation	-
neurodevelopmental disorder with hypotonia and speech delay, with or without seizures	Neupogen	Orphan Designation	-
neurodevelopmental disorder with hypotonia, dysmorphic facies, and skeletal anomalies, with or without seizures	Xgeva	Orphan Designation	-
neurodevelopmental disorder with intracranial hemorrhage, seizures, and spasticity	Neupogen	Orphan Designation	-
neurodevelopmental disorder with progressive spasticity and brain abnormalities	Neupogen	Orphan Designation	-
neutropenia	Neupogen	Orphan Designation	-
neutropenia, severe congenital, 10, autosomal recessive	Neupogen	Orphan Designation	-
neutropenia, severe congenital, 11, autosomal dominant	Neupogen	Orphan Designation	-
neutropenia, severe congenital, 2, autosomal dominant	Neupogen	Orphan Designation	-
neutropenia, severe congenital, 9, autosomal dominant	Neupogen	Orphan Designation	-
non-renal secondary hyperparathyroidism	cinacalcet	Orphan Designation	-
non-small cell lung carcinoma	Imdelltra	Orphan Designation	-
non-small cell squamous lung carcinoma	Imdelltra	Orphan Designation	-
nut midline carcinoma	Neupogen	Orphan Designation	-
osteogenesis imperfecta	romosozumabdenosumab	Orphan Designation	-
ovarian cancer	N-(4-{3-(2-aminopyrimidin-4-yl)pyridin-2-yl]oxy}phenyl)-4-(4-methylthiophen-2-yl)phthalazin-1-amine bis-mesylate dihydratetrebananib	Orphan Designation	-
pediatric acute-onset neuropsychiatric syndrome	Nplate	Orphan Designation	-
pediatric hepatocellular carcinoma	Nplateapremilastetelcalcetide	Orphan Designation	-
pediatric ovarian dysgerminoma	Nplateapremilastetelcalcetide	Orphan Designation	-
pediatric-onset Graves disease	Nplateapremilastetelcalcetide	Orphan Designation	-
plasma cell myeloma	small molecule inhibitor of the pro-survival protein Myeloid Cell Leukemia sequence 1 (MCL1)small molecule inhibitor of the pro-survival protein Myeloid Cell Leukemia sequence 1 (MCL 1)Bispecific T-cell engager (BiTE) containing two single-chain variable fragment (scFv) domains directed against the B cell maturation antigen (BCMA) surface receptor on tumor cells and the cluster of differentiation 3 (CD3) receptor on T-cellsbispecific T-cell engager antibody with a single chain Fc moiety that binds to B cell maturation antigen surface receptor and CD3	Orphan Designation	-
platelet-type bleeding disorder 20	Nplate	Orphan Designation	-
post-treatment Lyme disease syndrome	blinatumomab	Orphan Designation	-
primary adult heart tumor	Otezla XR	Orphan Designation	-
prolymphocytic leukemia	blinatumomab	Orphan Designation	-
pseudo-TORCH syndrome 2	Nplate	Orphan Designation	-
radioulnar synostosis with amegakaryocytic thrombocytopenia 1	Nplate	Orphan Designation	-
radioulnar synostosis with amegakaryocytic thrombocytopenia 2	Nplate	Orphan Designation	-
refractory celiac disease	human immunoglobulin (IgG1K) monoclonal antibody that binds to interleukin 15	Orphan Designation	-
retinitis pigmentosa 94, variable age at onset	Epogen	Orphan Designation	-
secondary hyperparathyroidism	cinacalcet	Orphan Designation	-
secondary hyperparathyroidism of renal origin	cinacalcet	Orphan Designation	-
skeletal overgrowth-craniofacial dysmorphism-hyperelastic skin-white matter lesions syndrome	Xgeva	Orphan Designation	-
small cell lung carcinoma	autologous T cells expressing a transmembrane chimeric antigen receptor (CAR) to target anti-delta-like ligand 3Imdelltra	Orphan Designation	-
systemic sclerosis	blinatumomab	Orphan Designation	-
teeth present at birth	Epogen	Orphan Designation	-
thrombocytopenia	romiplostim	Orphan Designation	-
thrombocytopenia 4	Nplate	Orphan Designation	-
thrombocytopenia 5	Nplate	Orphan Designation	-
thyroid gland diffuse large B-cell lymphoma	blinatumomab	Orphan Designation	-
treatment-refractory schizophrenia	blinatumomab	Orphan Designation	-
twin to twin transfusion syndrome	XGEVA	Orphan Designation	-

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