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Lumasiran: A Review in Primary Hyperoxaluria Type 1
1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. demail@springer.com.
Drugs
|January 22, 2024
Summary
Lumasiran (Oxlumo®) effectively reduces urinary oxalate in primary hyperoxaluria type 1 (PH1) patients. This RNA interference therapy shows sustained efficacy and an acceptable safety profile across various age groups and kidney function stages.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder causing excessive oxalate production and kidney damage.
- Current treatments for PH1 are limited, often involving supportive care and kidney transplantation.
- Lumasiran represents a novel therapeutic approach targeting the underlying cause of PH1.
Purpose of the Study:
- To evaluate the efficacy and safety of lumasiran in patients with PH1.
- To assess the impact of lumasiran on urinary oxalate excretion, plasma oxalate levels, and kidney function.
- To determine the long-term effectiveness and tolerability of lumasiran treatment.
Main Methods:
- Phase III ILLUMINATE trials (ILLUMINATE-A, -B, and -C) involving patients with PH1 of various ages and kidney function.
- Double-blind, placebo-controlled (ILLUMINATE-A) and single-arm (ILLUMINATE-B, -C) study designs.
- Administration of subcutaneous lumasiran and assessment of key clinical endpoints including urinary oxalate, plasma oxalate, and kidney function parameters.
Main Results:
- Lumasiran significantly reduced 24-h urinary oxalate excretion in patients aged ≥6 years (ILLUMINATE-A), with sustained effects over 36 months.
- In younger patients (<6 years, ILLUMINATE-B), lumasiran decreased urinary oxalate:creatinine ratios and plasma oxalate levels.
- Patients on dialysis and not on dialysis (ILLUMINATE-C) showed reduced plasma oxalate levels with lumasiran treatment.
- Secondary analyses indicated stable or improved nephrocalcinosis, kidney stone events, and GFR.
- Lumasiran demonstrated an acceptable tolerability profile, with most adverse events being mild injection-site reactions.
Conclusions:
- Lumasiran is a first-in-class RNA interference therapy demonstrating significant and sustained efficacy in reducing oxalate burden in PH1 patients.
- The treatment shows benefits across different age groups and stages of kidney disease, including those on dialysis.
- Lumasiran offers a well-tolerated and effective treatment option for managing primary hyperoxaluria type 1.
