Oligonucleotide (siRNA) · Gene silencing: hydroxyacid oxidase 1 (glycolate oxidase) (HAO1) · developed by Alnylam Pharms
Lumasiran is a small interfering RNA that acts in hepatocytes through RNA interference to degrade HAO1 messenger RNA, lowering glycolate oxidase enzyme levels and thereby reducing glyoxylate available for oxalate production. It is studied in primary hyperoxaluria type 1 and primary hyperoxaluria.
Summary written with AI from the sources listed at the end of this page.
From the indications section of the FDA label (version of Dec 1, 2025). Read the full label for the exact wording, limitations and safety information.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT04152200 | Phase 3 | Primary Hyperoxaluria Type 1 | Completed | ALNY |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
Primary HyperoxaluriaPrimary Hyperoxaluria Type 1
Updated Oct 11, 2026. Something wrong or missing? Report it on the ALNY page.
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