奥卢莫 (Oxlumo)

概览

Lumasiran is lumasiran is an interfering RNA that silences hydroxyacid oxidase 1 for the treatment of primary hypoxaluria type 1.

作用机制

Patients with primary hyperoxaluria type 1 produce an excess of oxalate due to a deficiency in the enzyme alanine-glyoxylate aminotransferase. Lumasiran is a small interfering RNA that silences the gene hydroxyacid oxidase 1 (HOA1). Lumasiran targets HOA1 mRNA, preventing translation to the enzyme glycolate oxidase (GO). Reduced levels of GO, reduce levels of glyoxylate, leaving less reactants available for metabolism to oxalate. In the ILLUMINATE trials, lumasiran reduced oxalate levels in 84% of adults and children over 6 years to at or below 1.5 times the upper limits of normal.

适应症

Lumasiran is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients.

• Primary Hyperoxaluria Type 1 (PH1)