Study shows benefit of lumasiran in treating advanced primary hyperoxaluria type 1
Phase 3 study results of lumasiran to treat advanced primary hyperoxaluria type 1 showed a significant reduction in plasma oxalate and “pre-dialysis” plasma oxalate from baseline to 6 months, a speaker at ASN Kidney Week said.
“Elevated plasma oxalate levels are responsible for much of the morbidity associated with advanced [primary hyperoxaluria type 1] PH1 and can result in oxalosis, leading to bone fractures, cardiomyopathy, vision loss, skin ulcers and other serious manifestations,” Mini Michael, MD, associate professor, renal section in the department of pediatrics at Baylor College of Medicine in Houston and a pediatric nephrologist at Texas Children’s Hospital, said in presenting the ILLUMINATE-C trial study results for Oxlumo (lumasiran, Alnylam Pharmaceuticals Inc.).
Lumasiran is an RNAi therapeutic targeting hydroxyacid oxidase 1 to treat patients with advanced PH1, a rare genetic disease characterized by progressive kidney failure and multiorgan dysfunction, according to a company press release.
“Excess oxalosis can result in the development of recurrent kidney stones, nephrocalcinosis, progressive kidney disease and ultimately kidney failure,” Michael said. “Patients who progress to or present with kidney failure require intensive hemodialysis and liver or combined liver-kidney transplants.”
The ILLUMINATE-C study included 21 patients: six in cohort A at stage 3b-5 CKD who did not require dialysis and 15 patients on hemodialysis in cohort B, Michael said.
“The primary efficacy endpoint of the study was the percent change in plasma oxalate (cohort A) and pre-dialysis plasma oxalate (cohort B) from baseline to 6 months, averaged across months 3 to 6,” she said.
Treatment with lumasiran in cohorts A and B, respectively, led to 33% and 42% mean reductions in plasma oxalate and “pre-dialysis” plasma oxalate from baseline to month 6, she said. “Reduction in [plasma oxalate] POx was evident by month 1 and persisted through the end of the 6-month primary analysis period,” Michael said.
There were no deaths and no serious or severe adverse events related to the drug, according to the press release, and no treatment discontinuations or study withdrawals.
“Changes in plasma oxalate of the magnitude seen in this study are encouraging and may positively impact long-term clinical outcomes, including those related to systemic oxalosis,” Michael said.
Lumasiran received FDA approvals in the United States and the European Union in November 2020 for the treatment of PH1 in adult and pediatric patients.
Reference:
Alnylam Presents Positive Results from ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1. https://investors.alnylam.com/press-release?id=26206. Published Nov. 5, 2021. Accessed Nov. 12, 2021.
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Michael M. ILLUMINATE-C, a single-arm, phase 3 study of lumasiran in patients with primary hyperoxaluria type 1 and CKD 3b-5, including those on dialysis. Presented at: ASN Kidney Week; Nov. 4-7, 2021 (virtual meeting).
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