inotersen

E561115

Inotersen is an antisense oligonucleotide drug used to treat hereditary transthyretin-mediated amyloidosis by reducing the production of transthyretin protein.

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Statements (48)

Predicate Object
instanceOf antisense oligonucleotide drug
pharmacologic substance
transthyretin synthesis inhibitor
approvedBy European Medicines Agency NERFINISHED
U.S. Food and Drug Administration NERFINISHED
belongsToTherapeuticArea neurology
rare diseases
bindsTo TTR mRNA in the liver
contraindication history of serious hypersensitivity to inotersen
severe thrombocytopenia
developedBy Ionis Pharmaceuticals NERFINISHED
EMAApprovalYear 2018
FDAApprovalYear 2018
hasAdministrationFrequency once weekly
hasATCCode N07XX14
hasBackboneModification phosphorothioate
hasBlackBoxWarningFor glomerulonephritis
thrombocytopenia
hasChemicalNature single-stranded antisense oligonucleotide
hasCommonAdverseEffect fatigue
headache
injection site reactions
nausea
hasDosingForm pre-filled syringe
hasINN inotersen
hasMolecularType 2′-O-methoxyethyl–modified antisense oligonucleotide
hasPregnancyCategory not recommended in pregnancy unless clearly needed
hasRouteOfAdministration subcutaneous injection
hasSeriousAdverseEffect glomerulonephritis
immune-mediated reactions
severe thrombocytopenia
hasTradeName Tegsedi NERFINISHED
indication hereditary transthyretin-mediated amyloidosis
polyneuropathy of hereditary transthyretin-mediated amyloidosis
legalStatusInEuropeanUnion prescription only
legalStatusInUnitedStates prescription only
marketedBy Akcea Therapeutics NERFINISHED
mechanismOfAction RNase H–mediated degradation of TTR mRNA
inhibition of transthyretin protein synthesis
primarySiteOfAction liver
reduces serum transthyretin concentration
reducesLevelOf transthyretin protein
requiresMonitoringOf platelet count
renal function
requiresRiskManagementProgram yes
targets transthyretin mRNA
treatsSymptom autonomic neuropathy in hATTR
sensorimotor polyneuropathy in hATTR

Referenced by (2)

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