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Lumasiran · Oxlumo, OXLUMO

Oligonucleotide (siRNA) · Gene silencing: hydroxyacid oxidase 1 (glycolate oxidase) (HAO1) · developed by Alnylam Pharms

Approved FDA, Nov 2020
ALNY Developer
1 Registered trials

What is Lumasiran?

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.

At a glance

Also known as
Oxlumo, OXLUMO, ALN-GO1
Drug type
Oligonucleotide (siRNA)
Targets
hydroxyacid oxidase 1 (glycolate oxidase) (HAO1)
Mechanism
Gene silencing: hydroxyacid oxidase 1 (glycolate oxidase) (HAO1)
Pathways
glyoxylate metabolism, oxalate production
Therapy area
Metabolic, Renal & urology
Developer
Alnylam Pharms Inc (ALNY)
FDA status
Approved as Oxlumo (November 23, 2020), NDA214103 · FDA label
UNII
67P6XH37HD

✅ FDA-approved uses

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.

🧪 Clinical trials

TrialPhaseConditionStatusSponsor
NCT04152200 Phase 3 Primary Hyperoxaluria Type 1 Completed ALNY

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Primary HyperoxaluriaPrimary Hyperoxaluria Type 1

More from Alnylam Pharms

Sources

Updated Oct 11, 2026. Something wrong or missing? Report it on the ALNY page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z

This data is for informational purposes only, not investment advice. BioRadar does not provide buy/sell recommendations. Past performance does not guarantee future results. Always do your own due diligence.