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.
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 NERFINISHED ⓘ |
| 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 NERFINISHED ⓘ |
| hasIndication |
ALA dehydratase-deficiency porphyria
NERFINISHED
ⓘ
acute hepatic porphyria ⓘ acute intermittent porphyria ⓘ hereditary coproporphyria ⓘ variegate porphyria ⓘ |
| hasMechanismOfAction |
RNA interference
ⓘ
inhibition of ALAS1 mRNA in hepatocytes ⓘ |
| hasMolecularTarget |
ALAS1 mRNA
NERFINISHED
ⓘ
aminolevulinate synthase 1 NERFINISHED ⓘ |
| hasPharmacologicalClass |
gene silencing agent
ⓘ
siRNA conjugate ⓘ |
| hasPregnancyCategory | not recommended unless clearly needed ⓘ |
| hasRegulatoryApproval |
EMA
NERFINISHED
ⓘ
FDA NERFINISHED ⓘ |
| 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 ⓘ |
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.