Givlaari

E446846

Givlaari is an RNA interference-based therapy used to treat acute hepatic porphyria by reducing the production of toxic heme intermediates in the liver.

All labels observed (1)

Label Occurrences
Givlaari canonical 1

How this entity was disambiguated

Statements (49)

Predicate Object
instanceOf RNA interference-based therapy ⓘ
drug ⓘ
small interfering RNA therapeutic ⓘ
hasAdverseEffect anaphylaxis ⓘ
elevated liver enzymes ⓘ
fatigue ⓘ
injection site reactions ⓘ
nausea ⓘ
renal function impairment ⓘ
hasApprovalYear 2019 ⓘ
hasATCCode B06AX06 ⓘ
hasBlackBoxWarning risk of anaphylactic reaction ⓘ
hasConjugate N-acetylgalactosamine (GalNAc) ⓘ
hasDeveloper Alnylam Pharmaceuticals ⓘ
hasDosageForm solution for injection ⓘ
hasEffect reduces accumulation of toxic heme intermediates ⓘ
reduces hepatic ALAS1 expression ⓘ
reduces need for hemin treatment ⓘ
reduces production of aminolevulinic acid ⓘ
reduces production of porphobilinogen ⓘ
reduces rate of porphyria attacks ⓘ
hasFrequencyOfAdministration once monthly ⓘ
hasGenericName givosiran ⓘ
hasIndication ALA dehydratase-deficiency porphyria ⓘ
acute hepatic porphyria ⓘ
acute intermittent porphyria ⓘ
hereditary coproporphyria ⓘ
variegate porphyria ⓘ
hasMechanismOfAction RNA interference ⓘ
inhibition of ALAS1 mRNA in hepatocytes ⓘ
hasMolecularTarget ALAS1 mRNA ⓘ
linked to: RNA

aminolevulinate synthase 1 ⓘ
hasPharmacologicalClass gene silencing agent ⓘ
siRNA conjugate ⓘ
hasPregnancyCategory not recommended unless clearly needed ⓘ
hasRegulatoryApproval EMA ⓘ
FDA ⓘ
hasRouteOfAdministration subcutaneous injection ⓘ
hasTargetCellType hepatocyte ⓘ
hasTherapeuticArea hematology ⓘ
hepatology ⓘ
metabolic disorders ⓘ
rare diseases ⓘ
isAdministeredBy healthcare professional ⓘ
requiresMonitoring homocysteine levels ⓘ
liver function tests ⓘ
renal function ⓘ
treats acute hepatic porphyria attacks ⓘ
neurovisceral symptoms of acute hepatic porphyria ⓘ

How these facts were elicited

Referenced by (1)

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