Lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the treatment of adults and children with primary hyperoxaluria type 1 (PH1).
Primary hyperoxaluria type 1 (PH1)PH1 is an ultra-rare orphan disease characterized by excessive oxalate production, which can lead to end-stage renal disease (ESRD) and other systemic complications. PH1 affects approximately 3.5 to 4 individuals per million in Europe and the United States. Heterogeneity in disease manifestation often contributes to delays in diagnosis, with a median time to diagnosis of approximately six years.
PH1 leads to progressive kidney damage, and patients with advanced kidney disease require intensive dialysis to help filter waste products from their blood until they are able and eligible to receive a dual or sequential liver/kidney transplant, an invasive procedure associated with a high risk of morbidity and mortality, and life-long immunosuppression.
Topline Data Highlights (As of 22 OCT2020)
- Lumasiran Demonstrated a 72 Percent Mean Reduction in Urinary Oxalate and Improvements in Nephrocalcinosis in ILLUMINATE-B Phase 3 Study in Children Under the Age of Six and as Young as Three Months
- Alnylam Also Presents New Results from the 12-Month Extension Period of the ILLUMINATE-A Pivotal Study, Showing Sustained Reduction in Urinary Oxalate Levels and Evidence for a Decrease in the Rate of Renal Stone Events in Patients Treated with Lumasiran –
- Long-Term Results from the Ongoing Phase 2 Open-Label Extension Study Provide Additional Evidence for Sustained Reduction in Urinary Oxalate Levels and Acceptable Safety Profile
- FDA & EMA Approved
ILLUMINATE-A (NCT03681184)ILLUMINATE-B (NCT03905694)
- Brief Summary: The purpose of this study is to evaluate the efficacy and safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 (PH1).
- Phase 3
- Study Type: Randomized
- Primary Outcome Measures: Percent Change in 24-hour Urinary Oxalate Excretion from Baseline to Month 6 [ Time Frame: 6 months ]
- Brief Summary: The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Study Type: Open Label
- Primary Outcome Measures: Percentage Change in Urinary Oxalate Excretion from Baseline to Month 6 [ Time Frame: Up to 6 months ]