Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1
RCT (n=39) found that lumasiran reduced urinary oxalate excretion, the cause of progressive kidney failure in this population, with the majority of patients who received lumasiran, having normal or near-normal levels after 6 months of treatment.
Source:
New England Journal of Medicine
SPS commentary:
Primary hyperoxaluria type 1 is a rare genetic disease caused by hepatic overproduction of oxalate that leads to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. Lumasiran, an investigational RNA interference (RNAi) therapeutic agent, reduces hepatic oxalate production by targeting glycolate oxidase. The least-squares mean difference in the change in 24-hour urinary oxalate excretion (lumasiran minus placebo) was −53.5% (p<0.001), with a reduction in the lumasiran group of 65.4%