Drug updated on 9/4/2024
Dosage Form | Injection (subcutaneous; 94.5 mg/0.5 mL) |
Drug Class | HAO1-directed small interfering ribonucleic acid |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
Latest News
Summary
- Oxlumo (lumasiran) is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
- This summary is based on the review of four randomized controlled trial(s). [1-4]
- Urinary Oxalate Reduction: Lumasiran reduced the spot urinary oxalate ratio by 72% at month 6 and 12 in ILLUMINATE-B, with reductions varying by weight: 89% (<10 kg), 68% (10 to <20 kg), and 71% (≥20 kg). In the Double-Blind Phase 3 Trial, a 65.4% reduction in 24-hour urinary oxalate excretion was observed, with a mean difference of -53.5 percentage points compared to placebo (P<0.001).
- Plasma Oxalate Reduction: In ILLUMINATE-B, plasma oxalate levels were reduced by 32% at month 6 and 47% at month 12. The Double-Blind Phase 3 Trial reported a 39.5 percentage point reduction in plasma oxalate compared to placebo (P<0.001).
- Proportion of Patients with Normal Urinary Oxalate: In the Double-Blind Phase 3 Trial, 84% of lumasiran-treated patients had 24-hour urinary oxalate excretion within 1.5 times the upper limit of normal at month 6, compared to 0% in the placebo group (P<0.001).
- The most common lumasiran-related adverse events were mild, transient injection-site reactions, occurring in 17% of patients in ILLUMINATE-B and 38% of lumasiran-treated patients in the Double-Blind Phase 3 Trial. Similar mild, transient injection-site reactions were reported in ILLUMINATE-C and the Single-Arm Phase 3 Study.
- No serious adverse events related to lumasiran were reported, and there were no discontinuations due to adverse events in ILLUMINATE-C. The safety profile was acceptable across different populations, including infants, young children, and patients with advanced kidney disease, including those undergoing hemodialysis.
- There is no population type or subgroup information available in the reviewed studies.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Oxlumo (lumasiran) Prescribing Information. | 2023 | Alnylam Pharmaceuticals, Inc., Cambridge, MA |
Randomized Controlled Trials
Document Title | Sex Distribution | Year | Source |
---|---|---|---|
Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial. | 18Subjects F: 56% M: 44% | 2023 | Pediatric Nephrology |
Lumasiran for advanced primary hyperoxaluria type 1: Phase 3 ILLUMINATE-C trial. | 21Subjects F: 43% M: 57% | 2023 | American Journal of Kidney Diseases |
Phase 3 trial of lumasiran for primary hyperoxaluria type 1: a new RNAi therapeutic in infants and young children. | 18Subjects F: 56% M: 44% | 2022 | Genetics in Medicine |
Lumasiran, an RNAi therapeutic for primary hyperoxaluria type 1. | 39Subjects F: 33% M: 67% | 2021 | The New England Journal of Medicine |
Sex Distribution:
F:56%
M:44%
18Subjects
Year:
2023
Source:Pediatric Nephrology
Sex Distribution:
F:43%
M:57%
21Subjects
Year:
2023
Source:American Journal of Kidney Diseases
Document Title
Sex Distribution:
F:56%
M:44%
18Subjects
Year:
2022
Source:Genetics in Medicine
Sex Distribution:
F:33%
M:67%
39Subjects
Year:
2021
Source:The New England Journal of Medicine
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Clinical practice recommendations for primary hyperoxaluria: an expert consensus statement from ERKNet and OxalEurope. | 2023 | Nature Reviews Nephrology |