Lumasiran: A Review in Primary Hyperoxaluria Type 1

• Published in: Drugs (New York, N.Y.) N.Y.), 2024-02, Vol.84 (2), p.219-226
• Main Author: Kang, Connie
• Format: Article
• Language: English
• Subjects: Adis Drug Evaluation; Calcinosis; Calculi; Clinical trials; Creatinine; Dialysis; Double-stranded RNA; Drug dosages; Enzymes; Glomerular filtration rate; Hemodialysis; Humans; Hyperoxaluria; Hyperoxaluria, Primary - drug therapy; Internal Medicine; Kidney diseases; Kidney stones; Kidneys; Liver transplants; Medicine; Medicine & Public Health; mRNA; Mutation; Nephrolithiasis; Oxalates; Oxalic acid; Patients; Pediatrics; Pharmacodynamics; Pharmacokinetics; Pharmacology/Toxicology; Pharmacotherapy; Placebos; Plasma; Primary hyperoxaluria; Randomized Controlled Trials as Topic; Ribonucleic acid; RNA; RNA, Small Interfering; RNA-mediated interference; Urine
• ISSN: 0012-6667; 1179-1950; 1179-1950
• DOI: 10.1007/s40265-023-01987-1

Lumasiran (Oxlumo ® ), a first-in-class synthetic, double-stranded, ribonucleic acid (RNA) interference molecule targeting glycolate oxidase through silencing HAO1 mRNA, is approved in several countries for patients of any age and stage of kidney function with primary hyperoxaluria type 1 (PH1). Approval was based on results from the phase III ILLUMINATE trials. In the double-blind, placebo-controlled, ILLUMINATE-A trial, subcutaneous lumasiran was significantly more effective than placebo in reducing 24-h urinary oxalate excretion in patients aged ≥ 6 years with PH1; this effect was sustained for ≥ 36 months in ongoing longer-term analyses. In the single-arm ILLUMINATE-B trial, lumasiran reduced urinary oxalate:creatinine ratios and plasma oxalate levels in patients aged