lumasiran

Lumasiran is an RNA interference therapy developed for the treatment of primary hyperoxaluria type 1 (PH1). It is a small interfering RNA (siRNA) that targets the messenger RNA of hepatic glycolate oxidase (GO), an enzyme involved in glyoxylate metabolism. By reducing GO expression, lumasiran decreases production of oxalate, thereby lowering urinary oxalate levels in PH1 patients.

Administration and dosing: Lumasiran is given by subcutaneous injection. Dosing is weight-based and typically initiated with monthly injections for the first 3 months, followed by injections every 3 months thereafter. A common starting dose is 3 mg/kg, with adjustments based on clinical guidance.

Indications: It is approved for the treatment of primary hyperoxaluria type 1 in pediatric and adult patients to reduce urinary oxalate. It is intended to be used as part of a comprehensive management plan for PH1, including hydration and monitoring for kidney stone risk.

Safety: The most frequently reported adverse events in trials were injection-site reactions, upper respiratory tract infections, and nausea. Serious adverse events were rare. Monitoring for liver and kidney function, and for injection-related reactions, is recommended as per prescribing information.

Regulatory status: Lumasiran was approved by the U.S. Food and Drug Administration in 2020 and marketed under the brand name Oxlumo. Regulatory approvals in other regions followed. See also primary hyperoxaluria type 1 and RNA interference therapy.