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SILVER SPRING, Md., Nov.23, 2020 / PRNewswire / – Today, the U.S. Food and Drug Administration approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder. This approval is a cumulation of the work of experts and community members coordinated by the Oxalosis & Hyperoxaluria Foundation and the Kidney Health Initiative.
“The approval of Oxlumo represents a great triumph of community involvement in addressing a rare disease. It is the result of input from patients, treating physicians, experts and sponsors in a patient-focused drug development meeting and through other efforts. collaboration, “said Norman Stockbridge, MD, Ph.D., director of the Division of Cardiology and Nephrology at the FDA’s Center for Drug Evaluation and Research.
Primary hyperoxaluria (PHs) are caused by excessive production of oxalate, a substance consumed in food and also produced by the body. PH1 is the most common and severe type. PH1 affects approximately one to three individuals per million in North America and Europe and accounts for approximately 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 can experience progressive kidney damage, which can lead to kidney failure and the need for dialysis (a treatment that cleanses the blood). As kidney function worsens, oxalate can build up and damage other organs, including the heart, bones, and eyes.
Oxlumo works to decrease oxalate production. It has been evaluated in two studies in patients with PH1: a randomized, placebo-controlled study in patients aged six years and older and an open-label study in patients younger than six years. Patients ranged in age from four months to 61 years 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 oxalate in their urine, compared with an average 12% reduction in the placebo group. By the sixth month of the study, 52% of patients treated with Oxlumo achieved a normal 24-hour urinary oxalate level; no placebo-treated patients did this.
In the second study, 16 patients under the age of six received Oxlumo. 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 more common side effects of Oxlumo include injection site reactions and abdominal pain.
Oxlumo has received orphan drug designation, which provides incentives to assist and encourage drug development for rare diseases. The application was also granted the designation of innovative therapy. Additionally, the manufacturer received a Rare Pediatric Disease Priority Review Coupon. The FDA’s Pediatric Rare Disease Priority Review Voucher Program is intended to encourage the development of new drugs and biologics to prevent and treat rare diseases in children.
The FDA has granted Oxlumo approval to Alnylam Pharmaceuticals, Inc.
Additional Resources:
Contact with the media: Chanapa Tantibanchachai, 202-384-2219
Consumer requests: Email or 888-INFO-FDA
The FDA, an agency within the U.S. Department of Health and Human Services, protects public health by ensuring the safety, efficacy, and protection of human and veterinary drugs, vaccines, and other biological products for use human and medical devices. The agency is also responsible for the safety and security of food supply, cosmetics, food supplements, products that emit electronic radiation, and regulating our nation’s tobacco products.
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SOURCE US Food and Drug Administration
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