Lumasiran is lumasiran is an interfering RNA that silences hydroxyacid oxidase 1 for the treatment of primary hypoxaluria type 1. This condition, caused by a deficiency in the enzyme alanine-glyoxylate aminotransferase, leads to an accumulation of oxalate, causing calcium crystal formation.
- Mechanism
- Curator reviewed · 6 references
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.
- Primary indication
- Lumasiran is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients.Curator reviewed · 1 structured indication
- First approval
- Canada, 2022 · United States, 2020 · European Union, 2020
- Code names
- AD-65585 · ALN-65585 · ALN-G01 · ALN-GO1 · WHO 10684
- Brand names
Resolves to
What you can answer from here — as of September 12, 2026
Which other approved drugs treat the same conditions as Lumasiran, and which companies have late-stage candidates in those indications?
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