Lumasiran
Mechanism :
Lumasiran is a HAO1-directed small interfering dsRNA. It reduces levels of glycolate oxidase (GO) enzyme by targeting the hydroxy acid oxidase 1 mRNA in hepatocytes via RNA interference. A decrease in GO enzyme levels reduce the total amount of glycolate which is a subtracts for production of oxalate to lower urinary oxalate levels.
Indication :
- Primary hyperoxaluria type 1(PH1)
Dosing :
Available as a single dose vial of 94.5 mg/0.5 ml for subcutaneous injection. Rotate injection sites.
Loading dose:
<20 kg: 6 mg/kg subcutaneously once a month; three doses.
≥20 kg: 3 mg/kg subcutaneously once a month; three doses.
Maintainance dose:
Begin one month after the last loading dose.
<10 kg: 3 mg/kg subcutaneously once a month.
10 to <20 kg: 6 mg/kg subcutaneously once every 3 months.
≥20 kg: 3 mg/kg subcutaneously once every 3 months.
Adverse Effect :
Pain in the abdomen, reaction at injection site.
Lactation :
Limited data available regarding use during lactation.
Hepatic Dose :
o Dose adjustment not required for mild to moderate hepatic impairment (total bilirubin <= 3 times the ULN with any AST)
o Limited data available regarding drug usage with severe hepatic impairment (total bilirubin > 3 times the ULN with any AST)
Pregnanacy :
Limited data available regarding use during pregnancy.