For immediate release:

Today, the US Food and Drug Administration approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder. This approval is the cumulative work of experts and community members coordinated by the Oxalosis & Hyperoxaluria Foundation and the Kidney Health Initiative.

“Oxlumo’s approval represents a great triumph for community engagement in the fight against a rare disease. This is the result of contributions from patients, referring physicians, experts and sponsors at a patient-focused drug development meeting and through other collaborative efforts, ”said Norman Stockbridge, MD, Ph.D., director of the cardiology and nephrology division in the Center for Drug Evaluation and Research, FDA.

Primary hyperoxaluria (PH) is caused by excessive production of oxalate, a substance consumed in food and also produced by the body. PH1 is the most common and serious type. PH1 affects about one to three people per million in North America and Europe and accounts for about 80% of PH cases.

Patients with PH1 produce too much oxalate, which can combine with calcium to cause kidney stones and deposits in the kidneys. Patients may have progressive kidney damage, which can lead to kidney failure and the need for dialysis (a treatment that purifies the blood). As kidney function deteriorates, oxalate can build up and damage other organs, including the heart, bones, and eyes.

Oxlumo works to decrease the production of oxalate. It has been evaluated in two studies in patients with PH1: a randomized, placebo-controlled trial in patients six years of age and older and an open-label study in patients less than six years of age. Patients were between four months and 61 years old at the first dose. In the first study, 26 patients received a monthly injection of Oxlumo followed by a maintenance dose every three months; 13 patients received placebo injections. The primary endpoint was the amount of oxalate measured in urine over 24 hours. In the Oxlumo group, patients had, on average, a 65% reduction in urine oxalate, compared to an average reduction of 12% in the placebo group. By month 6 of the study, 52% of patients treated with Oxlumo achieved normal 24-hour urinary oxalate levels; no patient treated with placebo did so.

In the second study, 16 patients under the age of six all received Oxlumo. Using another measurement of oxalate in the urine, the study showed, on average, a 71% decrease in urine oxalate by month six of the study.

The most common side effects of Oxlumo include an injection site reaction and abdominal pain.

Oxlumo has received the Orphan Drug Designation, which provides incentives to help and encourage drug development for rare diseases. The application also got the designation of revolutionary therapy. In addition, the manufacturer has received a priority review voucher for rare pediatric diseases. The FDA’s Rare Pediatric Disease Priority Review Voucher program aims to encourage the development of new drugs and biologics to prevent and treat rare diseases in children.

The FDA granted Oxlumo approval to Alnylam Pharmaceuticals, Inc.

The FDA, an agency of the US Department of Health and Human Services, protects public health by ensuring the safety, efficacy, and safety of human and veterinary drugs, vaccines and other biologicals for human use, and medical devices . The agency is also responsible for the safety and security of the food supply, cosmetics, dietary supplements, products that emit electronic radiation, and the regulation of tobacco products.

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