Lumasiran Sodium Suppliers & Bulk Manufacturers

Available Forms: Injection

Available Strengths: 94.5 mg/0.5 mL

Reference Brands: Oxlumo (USA)

Category: Orphan Drugs

Product Description:

Lumasiran is a double-stranded small interfering ribonucleic acid (siRNA) therapeutic developed to treat primary hyperoxaluria type 1 (PH1) by directly addressing the metabolic pathway responsible for excessive oxalate production. It works through RNA interference by specifically targeting the hydroxyacid oxidase 1 (HAO1) messenger RNA in hepatocytes. This action leads to a reduction in the production of the glycolate oxidase (GO) enzyme in the liver.

Lower levels of glycolate oxidase result in decreased conversion of glycolate to glyoxylate, which is a key precursor in the formation of oxalate. By reducing the availability of glyoxylate, lumasiran significantly lowers urinary and plasma oxalate levels, thereby addressing the underlying cause of disease manifestations in patients with PH1, including kidney stone formation and progressive renal damage.

Importantly, the mechanism of action of lumasiran is independent of the specific AGXT gene mutation responsible for PH1. Because glycolate oxidase functions upstream of the deficient alanine:glyoxylate aminotransferase (AGT) enzyme, lumasiran is effective across different genetic variants of the disease. This targeted, gene-silencing approach represents a disease-modifying strategy and a major advancement in the management of primary hyperoxaluria type 1.