Lumasiran demonstrates sustained efficacy in treating rare genetic disorder PH1
The ILLUMINATE-C trial evaluating lumasiran, an RNA interference therapeutic, in patients with primary hyperoxaluria type 1 (PH1) and chronic kidney disease (CKD) stages 3b–5 has shown promising results, according to data presented at the Kidney Week 2023 Annual Meeting.
The trial, which included patients of all ages with eGFR (estimated glomerular filtration rate) ≤45 mL/min/1.73m², including those on hemodialysis (HD), showed significant reductions in plasma oxalate (POx) levels at month 24 compared to baseline.
Of the 21 patients enrolled in the study, 83% of Cohort A (non-HD) and 80% of Cohort B (HD) completed the month 24 visit. In Cohort A, there was a notable 60.5% mean reduction in POx levels from baseline, while Cohort B demonstrated a 30.6% reduction.
Two patients from Cohort A with low baseline eGFR initiated HD during the study. In Cohort B, 2 patients who underwent kidney-only transplantation remained in the study with functional grafts after 16 and 21 months, displaying comparable POx levels to those who underwent combined liver-kidney transplantation.
In addition, in patients with an ejection fraction (EF) below 55% at baseline, 2 out of 3 showed an improvement of ≥5% at month 24, with none experiencing EF deterioration. Mild injection site reactions were the most common lumasiran-related adverse events, occurring in 24% of patients overall. No deaths or serious/severe lumasiran-related adverse events, discontinuations, or withdrawals were reported.
The study’s findings suggest that lumasiran maintains its efficacy at month 24, with an acceptable safety profile in PH1 patients with CKD stages 3b–5, including those on HD, as well as in patients with an isolated kidney transplant.
Giorgia M, et al. Genetic Prevalence Estimates for All Types of Primary Hyperoxaluria. Presented at: Kidney Week 2023.