History
Lumasiran was evaluated by the U.S. Food and Drug Administration (FDA) in two studies of participants with PH1: a randomized, placebo-controlled trial in participants six years and older and an open-label study in participants younger than six years (NCT03681184 and NCT03905694). Participants ranged in age from four months to 61 years at the first dose. In the first study, 26 participants received a monthly injection of lumasiran followed by a maintenance dose every three months; 13 participants received placebo injections. Neither the patients nor the healthcare providers knew which treatment was being given until after the trial was completed. The primary endpoint was the amount of oxalate measured in the urine over 24 hours. In the lumasiran group, participants had, on average, a 65% reduction of oxalate in the urine, compared to an average 12% reduction in the placebo group. By the sixth month of the study, 52% of participants treated with lumasiran reached a normal 24-hour urinary oxalate level; no participants treated with the placebo did. In the second study, 16 participants younger than six years all received lumasiran. Using another measure of oxalate in the urine, the study showed, on average, a 71% decrease in urinary oxalate by the sixth month of the study. The trials were conducted at 25 centers in the United States, Europe, and the Middle East.The FDA granted the application for lumasiran orphan drug and breakthrough therapy designations. In addition, the manufacturer received a rare pediatric disease priority review voucher.
The FDA granted the approval of Oxlumo to Alnylam Pharmaceuticals, Inc.

Society and culture
Legal status
Lumasiran is available under the UK Early Access to Medicines Scheme (EAMS) as of July 2020.On 15 October 2020, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Oxlumo, intended for the treatment of primary hyperoxaluria type 1 (PH1). The applicant for this medicinal product is Alnylam Netherlands B.V.Lumasiran was approved for medical use in the European Union and in the United States in November 2020. References
External links
"Lumasiran". Drug Information Portal. U.S. National Library of Medicine. Clinical trial number NCT03681184 for "A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (ILLUMINATE-A)" at ClinicalTrials.gov
Clinical trial number NCT03681184 for "A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 (ILLUMINATE-B)" at ClinicalTrials.gov