Onpattro

E446845

Onpattro is an RNA interference (RNAi) therapeutic used to treat polyneuropathy in adults with hereditary transthyretin-mediated (hATTR) amyloidosis.

All labels observed (3)

Label Occurrences
patisiran 3
ONPATTRO (patisiran) 1
Onpattro canonical 1

How this entity was disambiguated

Statements (49)

Predicate Object
instanceOf RNA interference therapeutic
drug
orphan drug
prescription medicine
small interfering RNA
actsOn transthyretin mRNA
approvedBy European Medicines Agency
U.S. Food and Drug Administration
commonAdverseEffect back pain
fatigue
flushing
headache
infusion-related reaction
nausea
containsExcipient DLin-MC3-DMA
PEGylated lipid
cholesterol
lipid excipients
countryOfFirstApproval United States of America
developedBy Alnylam Pharmaceuticals
dosageForm concentrate for solution for infusion
dosingInterval once every three weeks
hasActiveIngredient patisiran
linked to: Onpattro
hasApprovalYear 2018
hasATCCode N07XX13
hasDrugClass gene-silencing therapy
transthyretin expression inhibitor
hasIndication polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis
hasInternationalNonproprietaryName patisiran
linked to: Onpattro
hasLegalStatus prescription only
hasPregnancyCategory not recommended during pregnancy unless clearly needed
hasTherapeuticArea neurology
rare diseases
improves neuropathy impairment scores in hATTR patients
isAdministeredBy healthcare professional
isFormulatedAs lipid nanoparticle
marketingAuthorizationHolder Alnylam Pharmaceuticals
mayCause vitamin A deficiency
mechanismOfAction RNA interference
silencing of transthyretin gene expression in the liver
reduces serum transthyretin levels
reducesLevelOf transthyretin protein
requires premedication to reduce infusion-related reactions
requiresMonitoringOf vitamin A levels
requiresSupplementationOf vitamin A
routeOfAdministration intravenous infusion
targetOrgan liver
treats hATTR amyloidosis with polyneuropathy
hereditary transthyretin-mediated amyloidosis

How these facts were elicited

Referenced by (5)

Full triples — surface form annotated when it differs from this entity's canonical label.

Alnylam Pharmaceuticals, Inc. develops ONPATTRO (patisiran)
subject linked to: ALNY
linked to: Onpattro
Onpattro hasInternationalNonproprietaryName patisiran
linked to: Onpattro
Onpattro hasActiveIngredient patisiran
linked to: Onpattro
RNA therapeutics regulatoryApprovedExample patisiran
linked to: Onpattro