Lumasiran is approved as the first drug for the treatment of patients with primary hyperoxaluria type 1 (PH1).
Primary hyperoxaluria is a rare metabolic disorder that produces excess oxalate.
Oxalate, when combined with calcium, can cause kidney stones and loss of kidney function.
Lumasiran inhibits the production of oxalate.
ILLUMINATE-A trial included 39 patients aged ≥6 years with PH1.
In the treatment arm, patients received subcutaneous lumasiran monthly for 3 months followed by quarterly maintenance doses at 3 mg/kg.
There was a 65% average reduction of oxalate in the urine compared with an average reduction of 12% in the placebo arm.
Over half of patients receiving lumasiran achieved a normal 24-hour urinary oxalate level at 6 months, compared with no patients in the placebo group.
Adverse events are mild to moderate, with no severe or serious adverse events.
The most common AE are Injection site reactions,,occurring in 35% of users.
Phase 3 ILLUMINATE-B trial evaluated lumasiran in patients aged under 6 years with PH1: led to an average decrease of 71% in urinary oxalate at the 6-month point of the study.
Approximately 80% of all PH cases are PH1, and the rare disease affects an estimated 1 to 3 people per million in North America and Europe.