Lumasiran shows efficacy in adults with reduced GFR and primary hyperoxaluria type 1 (PH1)

In patients with PH1 and advanced kidney disease, including patients on hemodialysis, use of Lumasiran shows substantial reductions in plasma oxalate levels with acceptable safety. 

Primary hyperoxaluria type 1 (PH1) is a rare, genetic disease characterized by excessive hepatic oxalate production that frequently causes end-stage kidney disease (ESKD). Lumasiran is an RNA interference therapeutic that is administered subcutaneously for the treatment of PH1. Lumasiran has been shown to reduce oxalate levels in the urine and plasma of patients with PH1 who have relatively preserved kidney function. In the ILLUMINATE-C study, the efficacy and safety of lumasiran were evaluated in patients with PH1 and advanced kidney disease, including a cohort of patients on hemodialysis. During the 6-month primary analysis period, lumasiran resulted in substantial reductions in plasma oxalate with acceptable safety in patients with PH1 complicated by advanced kidney disease. The most commonly reported lumasiran-related adverse events were injection-site reactions. These results were recently published in the American Journal of Kidney Diseases (AJKD).

Article Title: Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial

Authors: Mini Michael, MD, Jaap W. Groothoff, MD, PhD, Hadas Shasha-Lavsky, MD, John C. Lieske, MD, Yaacov Frishberg, MD, Eva Simkova, MD, Anne-Laure Sellier-Leclerc, MD, Arnaud Devresse, MD, PhD, Fitsum Guebre-Egziabher, MD, PhD, Sevcan A. Bakkaloglu, MD, Chebl Mourani, MD, Rola Saqan, MD, Richard Singer, MBChB, Richard Willey, MS, Bahru Habtemariam, PharmD, John M. Gansner, MD, PhD, Ishir Bhan, MD, MPH, Tracy McGregor, MD, Daniella Magen, MD

DOI: https://doi.org/10.1053/j.ajkd.2022.05.012