NEJM publishes Phase III ILLUMINATE-A trial of lumasiran to treat primary hyperoxaluria type 1.- Alnylam Pharma

Alnylam Pharmaceuticals, Inc. has announced that pivotal trial results from the ILLUMINATE-A Phase III study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1 (PH1), were published online in The New England Journal of Medicine (NEJM). In November 2020, Oxlumo (lumasiran) was approved by the FDA for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients, and received marketing authorization from the European Commission for the treatment of PH1 in all age groups. Oxlumo is the first-ever treatment approved for PH1 and the first RNAi therapeutic evaluated in both children and adults. The data reported in the ILLUMINATE-A Phase III study publication demonstrated that RNAi-mediated targeting of liver GO by lumasiran led to substantial and sustained reductions in urinary oxalate—the toxic metabolite responsible for the debilitating and life-threatening clinical manifestations of PH1. Relative to placebo, treatment with lumasiran resulted in a clinically significant (53.5 percent) reduction in 24-hour urinary oxalate excretion from baseline to month 6 – the primary endpoint of the study. See- "Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1".- Sander F. Garrelfs, M.D., Yaacov Frishberg, M.D., Sally A. Hulton, M.D., Michael J. Koren, M.D., et al., for the ILLUMINATE-A Collaborators-April 1, 2021 .N Engl J Med 2021; 384:1216-1226. DOI: 10.1056/NEJMoa2021712.