Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1
Garrelfs SF, Frishberg Y, Hulton SA, Koren MJ, O'Riordan WD, Cochat P, et al.
Published in N Engl J Med, Vol. 384 (13), pp. 1216-1226
Methodology
Phase 3, double-blind trial in patients with PH1 aged 6 years and over, randomised 2:1 to subcutaneous lumasiran or placebo for 6 months (doses at baseline and months 1, 2, 3 and 6). Primary end point: percent change in 24-hour urine oxalate, averaged over months 3-6.
Key Findings
Of 39 patients, 26 received lumasiran and 13 placebo. Urine oxalate fell 65.4% with lumasiran; the difference from placebo was -53.5 percentage points (P<0.001), with an effect from month 1. Plasma oxalate difference was -39.5 percentage points (P<0.001). 84% on lumasiran reached urine oxalate no higher than 1.5 times the upper limit of normal at month 6, versus 0% on placebo. Mild, transient injection-site reactions occurred in 38%.
Conclusions
Lumasiran reduced urine oxalate, the driver of kidney failure in PH1, to normal or near-normal levels in most patients within 6 months.
Limitations
Small trial (39 patients) of 6 months; outcomes are laboratory markers rather than stones or kidney function. Funded by the manufacturer.
Related Health Concerns
Study Details
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Disclosures
Funding
Alnylam Pharmaceuticals
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