Lumasiran shows continued promise in infants and young children with primary hyperoxaluria type 1

Lumasiran, a liver-directed RNA interference therapeutic, showed reductions in urinary and plasma oxalates in infants and young children with primary hyperoxaluria type 1 and was well-tolerated over 60 months of therapy, in the ILLUMINATE-B trial.

Dr Wesley Hayes (Great Ormond Street Hospital for Children NHS, UK) reported long-term results from ILLUMINATE-B, a phase 3, open-label, single-arm study assessing lumasiran in children with hyperoxaluria type 1 [1]. Participants were eligible if they were aged <6 years, with urinary oxalate-to-creatinine ratio >upper limit of normal in ≥2/3 samples, and an eGFR >45 mL/min/1.73 m² (age ≥12 months) or normal creatinine (age <12 months) without extrarenal systemic oxalosis. Treatment consisted of weight-based dosing of lumasiran for 6 months, followed by an extension treatment period of 54 months [2].

All the initial 18 participants completed the 60 months of the trial. Following 5 years of treatment, the mean spot oxalate-to-creatinine ratio decreased by 74% from baseline, corresponding to a change from 0.63 to 0.11 mmol/mmol. Similarly, the mean plasma oxalate decreased by 25% after 5 years (13.2 to 8.2 µmol/L). Furthermore, among the 14 patients with medullary nephrocalcinosis, 86% had an improvement in grade from baseline to month 60. Kidney stone events were also improved from 0.24/person-year in the 12 months prior to the study to 0.11/person-year at month 60. Overall, lumasiran was well-tolerated and there were no treatment discontinuations.

“In this final 5-year safety and efficacy data of lumasiran in infants and young children with primary hyperoxaluria type 1, we saw sustained reductions in urinary and plasma oxalates,” summarised Dr Hayes. “Treatment was well-tolerated and there were no safety concerns. We saw a trend for sustained improvement in nephrocalcinosis, and kidney stone events were low.”

1. Frishberg Y, et al. Long-term efficacy and safety in the 60-month, phase 3 ILLUMINATE-B trial of lumasiran in infants and young children with primary hyperoxaluria type 1. 62nd ERA Congress, 4–7 June 2025, Vienna, Austria.
2. Hayes W, et al. Pediatr Nephrol. 2023;38(4):1075-1086.

Medical writing support was provided by Mihai Surducan, PhD.

Copyright ©2025 Medicom Medical Publishers



Posted on 11 June 202511 June 2025