Lumasiran

Lumasiran reduces levels of glycolate oxidase (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.

• OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients [see Clinical Pharmacology (12.1) , Clinical Studies (14.1 , 14.2 , 14.3) ] . OXLUMO is a HAO1 -directed small interfering ribonucleic acid (siRNA) indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients.

• None. None.contraindicated

The recommended dose of OXLUMO by subcutaneous injection is based on body weight. ( 2.1 ) Body Weight Loading Dose Maintenance Dose less than 10 kg 6 mg/kg once monthly for 3 doses 3 mg/kg once monthly, beginning 1 month after the last loading dose 10 kg to less than 20 kg 6 mg/kg once monthly for 3 doses 6 mg/kg once every 3 months (quarterly), beginning 1 month after the last loading dose 20 kg and above 3 mg/kg once monthly for 3 doses 3 mg/kg once every 3 months (quarterly), beginning 1 month after the last loading dose See Full Prescribing Information for important preparation and administration instructions. ( 2.2 ) 2.1 Recommended Dosage The recommended dosing regimen of OXLUMO consists of loading doses (monthly for 3 doses) followed by maintenance doses (beginning 1 month after the last loading dose) administered subcutaneously as shown in Table 1. Dosing is based on actual body weight. Table 1. OXLUMO Weight-Based Dosing Regimen Body Weight Loading Dose Maintenance Dose Less than 10 kg 6 mg/kg once monthly for 3 doses 3 mg/kg once monthly, beginning 1 month after the last loading dose 10 kg to less than 20 kg 6 mg/kg once monthly for 3 doses 6 mg/kg once every 3 months (quarterly), beginning 1 month after the last loading dose 20 kg and above 3 mg/kg once monthly for 3 doses 3 mg/kg once every 3 months (quarterly), beginning 1 month after the last loading dose For Patients on Hemodialysis Administer OXLUMO after hemodialysis if administered on dialysis days. Missed Dose If a dose is delayed or missed, administer OXLUMO as soon as possible.

The most common adverse reaction (reported in ≥20% of patients) is injection site reactions. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Alnylam Pharmaceuticals at 1-877-256-9526 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of OXLUMO has been evaluated in a placebo-controlled trial and two single-arm clinical trials. Across these trials, 98 patients with PH1 have been treated with OXLUMO, including 71 pediatric patients and 15 patients on hemodialysis. Overall, 92 patients were treated for at least 6 months, 78 patients for at least 12 months, and 29 patients for at least 24 months. In the randomized, placebo-controlled, double-blind study ILLUMINATE-A in pediatric and adult patients with PH1 aged 6 to 61 years, 26 patients received OXLUMO, and 13 patients received placebo. Of these, 25 patients received ≥5 months of treatment.

8.1 Pregnancy Risk Summary There are no available data with the use of OXLUMO in pregnant women to evaluate a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. No adverse effects on pregnancy or embryo-fetal development related to OXLUMO were observed in rats at 45 times and in rabbits at 90 times the maximum recommended human dose in women (see Data ). The estimated background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data In an embryo-fetal development study in pregnant rats, lumasiran was administered subcutaneously at doses of 3, 10, and 30 mg/kg/day during organogenesis (gestational days 6-17). Administration of lumasiran resulted in no effects on embryo-fetal survival or fetal body weights and no lumasiran-related fetal malformations were observed. The 30 mg/kg/day dose in rats is 45 times the maximum recommended human dose (MRHD) for women of 3 mg/kg/month normalized to 0.1 mg/kg/day, based on body surface area.

Metabolism

The pharmacokinetic (PK) properties of OXLUMO were evaluated following administration of single and multiple dosages in patients with PH1 as summarized in Table 3. Table 3.

• Nov 23, 2020NDANDA214103Alnylam Pharms Inc

1. 1Renal Transplant4714%
2. 2Nephrolithiasis349.8%
3. 3Hospitalisation205.8%
4. 4Laboratory Test Abnormal164.6%
5. 5Urine Oxalate Increased164.6%
6. 6Drug Ineffective154.3%
7. 7Acute Kidney Injury133.8%
8. 8Death123.5%
9. 9Intentional Dose Omission123.5%
10. 10Liver Transplant123.5%
11. 11Nephrocalcinosis102.9%
12. 12Renal And Liver Transplant102.9%
13. 13Dialysis92.6%
14. 14Product Dose Omission Issue92.6%
15. 15End Stage Renal Disease82.3%

The 10 most recently updated of 10 ClinicalTrials.gov registrations naming Lumasiran as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
  Active not recruiting · Observational · 207 enrolled · Alnylam Pharmaceuticals
  NCT04982393updated 2026-04-24
• A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
  Completed · Phase 3 · Interventional · 21 enrolled · Alnylam Pharmaceuticals
  NCT04152200updated 2025-07-18
• A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
  Completed · Phase 3 · Interventional · 18 enrolled · Alnylam Pharmaceuticals
  NCT03905694updated 2025-02-14
• Lumasiran in Hyperoxalaemic Patients on Haemodialysis
  Recruiting · Phase 2 · Interventional · 50 enrolled · Charite University, Berlin, Germany
  NCT06225544updated 2024-08-21
• A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
  Completed · Phase 3 · Interventional · 39 enrolled · Alnylam Pharmaceuticals
  NCT03681184updated 2024-08-12
• A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels
  Terminated · Phase 2 · Interventional · 2 enrolled · Alnylam Pharmaceuticals
  NCT05161936updated 2024-05-22
• An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
  Completed · Phase 2 · Interventional · 20 enrolled · Alnylam Pharmaceuticals
  NCT03350451updated 2024-04-25
• Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
  Approved for marketing · Expanded access · Alnylam Pharmaceuticals
  NCT04125472updated 2024-02-16
• Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.
  Recruiting · Observational · 100 enrolled · Hospices Civils de Lyon
  NCT06225882updated 2024-01-26
• Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
  Completed · Phase 1 · Phase 2 · Interventional · 52 enrolled · Alnylam Pharmaceuticals
  NCT02706886updated 2020-01-30

Frequently asked questions

How does Lumasiran work?

Lumasiran reduces levels of glycolate oxidase (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.

What is Lumasiran used for?

According to FDA labeling, Lumasiran carries indications including: OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients [see Clinical Pharmacology (12.1) , Clinical Studies (14.1 , 14.2 , 14.3) ] . OXLUMO is a HAO1 -directed small interfering ribonucleic acid (siRNA) indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Lumasiran?

Lumasiran is classified as Various alimentary tract and metabolism products.

What are the brand names for Lumasiran?

Lumasiran is marketed under brand names including Oxlumo.

What are the contraindications for Lumasiran?

Lumasiran labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.