Onpattro
E446845
Onpattro is an RNA interference (RNAi) therapeutic used to treat polyneuropathy in adults with hereditary transthyretin-mediated (hATTR) amyloidosis.
Statements (49)
| Predicate | Object |
|---|---|
| instanceOf |
RNA interference therapeutic
ⓘ
drug ⓘ orphan drug ⓘ prescription medicine ⓘ small interfering RNA ⓘ |
| actsOn | transthyretin mRNA ⓘ |
| approvedBy |
European Medicines Agency
NERFINISHED
ⓘ
U.S. Food and Drug Administration NERFINISHED ⓘ |
| commonAdverseEffect |
back pain
ⓘ
fatigue ⓘ flushing ⓘ headache ⓘ infusion-related reaction ⓘ nausea ⓘ |
| containsExcipient |
DLin-MC3-DMA
ⓘ
PEGylated lipid ⓘ cholesterol ⓘ lipid excipients ⓘ |
| countryOfFirstApproval | United States of America NERFINISHED ⓘ |
| developedBy | Alnylam Pharmaceuticals NERFINISHED ⓘ |
| dosageForm | concentrate for solution for infusion ⓘ |
| dosingInterval | once every three weeks ⓘ |
| hasActiveIngredient | patisiran NERFINISHED ⓘ |
| hasApprovalYear | 2018 ⓘ |
| hasATCCode | N07XX13 ⓘ |
| hasDrugClass |
gene-silencing therapy
ⓘ
transthyretin expression inhibitor ⓘ |
| hasIndication | polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis ⓘ |
| hasInternationalNonproprietaryName | patisiran NERFINISHED ⓘ |
| 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 NERFINISHED ⓘ |
| 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 ⓘ |
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.