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Oxlumo (lumasiran)
Australian Prescription Medicine Decision Summary
Published
Product name
Oxlumo
Active ingredient
lumasiran
Submission type
Type A (New chemical entity)
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Oxlumo was approved for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups.
How this medicine works
Oxlumo (lumasiran) is a double-stranded small interfering ribonucleic acid (siRNA). Lumasiran reduces levels of glycolate oxidase (GO) enzyme by targeting the hydroxyacid oxidase 1 (HAO1) gene messenger ribonucleic acid (mRNA) in liver cells through siRNA-mediated RNA interference (a biochemical pathway in cells that can silence gene expression). Decreased GO enzyme levels reduce the amount of available glyoxylate, a chemical which liver cells use to produce oxalate. This results in the reduction of oxalate levels in the blood and urine. Excess oxalate is the cause of disease in patients with PH1 so reducing oxalate levels reduces disease symptoms.
PH1 is a genetic disease caused by a change in the AGXT (alanine-glyoxylate aminotransferase) gene. This gene is a set of instructions for synthesising the protein alanine-glyoxylate aminotransferase (AGT) which breaks down oxalate in the liver. People living with PH1 either make less AGT or don’t make it at all. Since GO activity occurs before AGT activity in this sequence of chemical reactions, the ability of lumasiran to reduce oxalate levels can proceed despite the presence of the altered AGXT gene.
Why the TGA approved or did not approve this medicine
The approval was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan data submitted to the TGA by the Sponsor.
The effectiveness and safety of Oxlumo was demonstrated in a Phase 1/2 study, an open-label extension study and three Phase 3 studies, including:
- a randomized, double-blind, placebo-controlled study in adults and children greater than 6 years of age with PH1
- a single-arm study in infants and children less than 6 years of age with PH1 and
- a single arm study in patients of all ages with advanced PH1 with or without dialysis.
Consumer Medicine Information (CMI)
The CMI leaflet offers guidance for consumers to support safe and effective use of the medicine. The CMI includes information on dose, how to use the medicine properly, potential side effects, safety precautions, storage instructions and more.
The Australian CMIs can be accessed through the ARTG details hyperlinks below.
Product Information (PI)
The PI document provides essential prescribing information for health professionals, including details on dosage recommendations, pregnancy category, contraindications, precautions and potential side effects.
The Australian PIs can be accessed through the ARTG details hyperlinks below.
Sponsor
Other resources
For health advice and information, including a symptom checker and service finder refer to the healthdirect website.
For advice on prescription medicines, over the counter medicines and other medicines (including complementary medicines) call Medicines Line.
For information on medicines subsidised by the Australian Government refer to the Pharmaceutical Benefits Scheme (PBS) website.
For data and reports on health and welfare topics in Australia refer to the Australian Institute of Health and Welfare website.