The Food and Drug Administration (FDA) has approved Oxlumo™ (lumasiran; Alnylam Pharmaceuticals) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

Lumasiran is an investigational ribonucleic acid interference therapeutic that inhibits hepatic production of oxalate by targeting hydroxyacid oxidase 1, thereby depleting glycolate oxidase. The approval was based on data from two phase 3 trials (ILLUMINATE-A and ILLUMINATE-B) that assessed the efficacy and safety of Oxlumo in patients with PH1.

The double-blind ILLUMINATE-A trial compared Oxlumo to placebo in 39 patients aged 6 years and older with eGFR greater than or equal to 30mL/min/1.73m2. Patients received 3 monthly doses of Oxlumo or placebo via subcutaneous injection followed by a quarterly dosing regimen. The primary end point of the study was the percent reduction from baseline in 24-hour urinary oxalate excretion corrected for body surface area averaged over months 3 through 6.

Findings from ILLUMINATE-A showed that treatment with Oxlumo resulted in a 65% mean reduction in urinary oxalate relative to baseline compared with an average 12% reduction in the placebo group. By month 6, 52% of patients treated with Oxlumo achieved a normal 24-hour urinary oxalate compared with 0% of placebo-treated patients (P =.001).

The single-arm ILLUMINATE-B trial included 18 patients less than 6 years of age with preserved renal function. Findings showed that patients treated with Oxlumo achieved a reduction in spot urinary oxalate:creatinine ratio from baseline (primary end point) of 71%.

With regard to safety, Oxlumo was found to be well tolerated and was not associated with any serious or severe adverse events. The most common adverse reactions reported included injection site reactions and abdominal pain.

Commenting on the approval, Jeffrey M. Saland, MD, Professor and Chief, Pediatric Nephrology and Hypertension, Jack and Lucy Clark Department of Pediatrics, Mount Sinai Kravis Children’s Hospital, New York City and investigator on the ILLUMINATE-A trial, said: “The consistent efficacy and safety profile of Oxlumo demonstrated in the ILLUMINATE-A and -B trials both in adults and children from as young as a few months old, combined with an infrequent dosing regimen that leads to rapid and sustained reduction of oxalate production, make Oxlumo an attractive therapeutic option to reduce the oxalate burden responsible for the severe clinical manifestations that individuals suffer due to PH1.”

The Company is also investigating Oxlumo in the ongoing ILLUMINATE-C trial in patients with advanced PH1, including patients on dialysis.

Oxlumo will be supplied as 94.5mg of lumasiran in 0.5mL single-dose vials and is expected to be available by the end of 2020.

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1.    Alnylam announces US Food and Drug Administration (FDA) approval of Oxlumo™ (lumasiran), the first and only treatment approved for primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients. [press release]. Cambridge, MA: Alnylam Pharmaceuticals, Inc; November 24, 2020. 

2.    Oxlumo™ [package insert]. Cambridge, MA: Alnylam Pharmaceuticals, Inc; 2020.