OXLUMO

What it is

Oxlumo is lumasiran injection solution. Each vial contains 0.5 mL of lumasiran sodium solution equivalent to 94.5 mg lumasiran. Oxlumo is for subcutaneous use only. Lumasiran is a double-stranded small interfering ribonucleic acid (siRNA) that reduces levels of glycolate oxidase (GO) enzyme by targeting the hydroxyacid oxidase 1 (HAO1) gene messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. Decreased GO enzyme levels reduce the amount of available glyoxylate, a substrate for oxalate production. This results in reduction of urinary and plasma oxalate levels, the underlying cause of disease manifestations in patients with primary hyperoxaluria type 1 (PH1).

Approved indications

— Primary hyperoxaluria type 1 (PH1) in all age groups.

Dosing overview

The recommended dose of Oxlumo consists of loading doses given once a month for 3 doses, followed by maintenance doses beginning one month after the last loading dose. Dosing is based on body weight. For patients weighing less than 10 kg, the loading dose is 6 mg/kg once monthly for 3 doses, followed by a maintenance dose of 3 mg/kg once monthly. For patients weighing 10 kg to less than 20 kg, the loading dose is 6 mg/kg once monthly for 3 doses, followed by a maintenance dose of 6 mg/kg once every 3 months (quarterly). For patients weighing 20 kg and above, the loading dose is 3 mg/kg once monthly for 3 doses, followed by a maintenance dose of 3 mg/kg once every 3 months (quarterly).

Key safety warnings

Treatment with lumasiran increases plasma glycolate levels, which may increase the risk of metabolic acidosis or worsening of pre-existing metabolic acidosis in patients with severe or end-stage renal disease. These patients should therefore be monitored for signs and symptoms of metabolic acidosis. In patients with moderate or severe hepatic impairment there is a potential for decreased efficacy. Therefore, efficacy should be monitored in these patients. The most common adverse reaction reported was injection site reaction (35%). In placebo-controlled and open-label clinical studies, injection site reactions were reported in 34 out of 98 patients (34.7%). The most commonly reported symptoms were erythema, swelling, pain, haematoma, pruritus, and discolouration. The majority of injection site reactions started on the day of administration, with less than 2% of injection site reactions occurring 5 or more days after administration. Injection site reactions were generally mild, resolved within two days, and did not result in interruption or discontinuation of treatment.

Contraindications

Severe hypersensitivity to the active substance or any of the excipients.

PBS listing

Oxlumo solution for subcutaneous injection 94.5 mg (as sodium) in 0.5 mL is listed on the PBS with 2 items. The restriction type is authority required. The ex-manufacturer price is A$97986.57.

Regulatory history

Oxlumo was first listed on the Australian Register of Therapeutic Goods on 24 June 2024. In March 2025, the Pharmaceutical Benefits Scheme Advisory Committee recommended Oxlumo for primary hyperoxaluria type 1 (PH1). Oxlumo is subject to additional monitoring in Australia. This will allow quick identification of new safety information.