FDA OKs First Treatment for Primary Hyperoxaluria Type 1


Lumasiran (Oxlumo; Alnylam Pharmaceuticals) is the first drug approved for the treatment of primary hyperoxaluria type 1, a rare genetic disorder.


Officials with the FDA recently approved the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder, according to a press release.

Lumasiran (Oxlumo; Alnylam Pharmaceuticals) helps to decrease oxalate production in patients with PH1, which is caused by excess production of oxalate. This excess oxalate can combine with calcium to cause kidney stones and deposits in the kidneys; patients can experience progressive kidney damage, which can lead to kidney failure.

Lumasiran previously received orphan drug designation and breakthrough therapy designation from the FDA.

The approval is based on 2 studies in patients with PH1: a randomized, placebo-controlled trial in patients 6 years of age and older and an open-label study in patients younger than 6 years of age. At first dose, patients ranged in age from 4 months to 61 years.

For the first study, 26 patients received a monthly injection of lumasiran followed by a maintenance dose every 3 months, and 13 patients received placebo injections. Among the patients who received lumasiran, there was, on average, a 65% reduction of oxalate in the urine, compared with an average 12% reduction in the placebo group. At month 6 of the study, 52% of the patients treated with lumasiran reached a normal 24-hour urinary oxalate level, whereas no patients treated with the placebo did.

For the second study, 16 patients younger than 6 years of age received lumasiran. According to the findings, the patients showed, on average, a 71% decrease in urinary oxalate by month 6 of the study.

The most commonly reported adverse effects of lumasiran include injection site reaction and abdominal pain.

Lumasiran’s approval was a cumulation of the work of experts and community members coordinated by the Oxalosis & Hyperoxaluria Foundation and the Kidney Health Initiative.


1. FDA Approves First Drug to Treat Rare Metabolic Disorder. News release. FDA; November 23, 2020. Accessed November 25, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-rare-metabolic-disorder.

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