Summary

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• 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.