NDASUBCUTANEOUSSOLUTIONPriority Review
Approved
Nov 2020
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
8
Data completeness
4/8 — Partial
Priority Review
OXLUMO (lumasiran) by Alnylam Pharmaceuticals is (go) enzyme by targeting the hydroxyacid oxidase 1 ( hao1 ) messenger ribonucleic acid (mrna) in hepatocytes through rna interference. First approved in 2020.
Drug data last refreshed 18h ago · AI intelligence enriched 2d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
OXLUMO (lumasiran) is an RNAi therapeutic that reduces oxalate production by silencing the HAO1 gene in hepatocytes, addressing the metabolic imbalance upstream of AGT deficiency in primary hyperoxaluria type 1 (PH1). Administered subcutaneously, it is specifically effective for PH1 regardless of underlying AGXT mutation status. The mechanism is mutation-agnostic and does not address PH2 or PH3 pathways.
Peak12.2 years to LOE
Product is at peak commercial maturity with stable market presence; teams focused on market penetration and patient access in rare disease segment.
Mechanism of Action
(GO) enzyme by targeting the hydroxyacid oxidase 1 ( HAO1 ) messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. Decreased GO enzyme levels reduce the amount of available glyoxylate, a substrate for oxalate production. As the GO enzyme is upstream of the deficient alanine:…
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (8)
Lumasiran in Hyperoxalaemic Patients on Haemodialysis
Started Apr 2024
50 enrolled
HaemodialysisChronic Kidney Disease Requiring Chronic DialysisCardiovascular Disease+2 more
A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels
Started Jan 2022
2 enrolled
Recurrent Calcium Oxalate Kidney Stone DiseaseElevated Urinary Oxalate Levels
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
Started Jan 2020
21 enrolled
Primary Hyperoxaluria Type 1Primary Hyperoxaluria
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Started Apr 2019
18 enrolled
Primary HyperoxaluriaPrimary Hyperoxaluria Type 1 (PH1)
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
Started Nov 2018
39 enrolled
Primary Hyperoxaluria Type 1 (PH1)
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.