FDA approves first drug to treat rare metabolic disorders

The U.S. Food and Drug Administration today approved Oxlumo as the first treatment for type 1 primary oxalic aciduria (PH1), a rare genetic disorder. This approval is a cumulative accumulation of work by experts and community members coordinated by the Foundation for Herbic acid overrepulation and high herbic acid uremia and the Kidney Health Program.“ Oxlumo's approval represents a great victory for the community in addressing rare diseases. This is the result of input from patients, attending physicians, specialists and sponsors at patient-centred drug development conferences and other cooperative efforts. Dr
Norman Stockbridge, director of the Department of
And Nephrology at the FDA's Center for Drug Evaluation and Research.Primary high herbic aciduria (PHs) is caused by excessive production of herbic acid, which is a substance consumed in food and produced by the human body. PH1 is the most common and most serious type. In North America and Europe, PH1 is estimated to affect one to three parts per million individuals, accounting for about 80 per cent of PH cases.Patients with PH1 produce too much hydrochloric acid, which may bind to calcium and lead to kidney stones and kidney deposits. Patients suffer from aggressive kidney damage, which can lead to kidney failure and the need for dialysis (treatment to purify blood). As kidney function deteriorates, herbic acid accumulates and damages other organs, including
heart, bones, and
.Oxlumo is committed to reducing oxalate production. It was evaluated in two studies of PH1 patients: a randomized placebo-controlled trial for patients 6 years of age and older, and an open label study for patients under 6 years of age. The age of the first dose ranged from four months to 61 years. In the first study, 26 patients received Oxlumo injections each month and then a maintenance dose every three months. In 13 patients received a placebo injection, the main endpoint indicator was the amount of hertic acid in the urine within 24 hours. In the Oxlumo group, the average reduction in oxalic acid in the patient's urine was 65 percent, while in the placebo group, the average reduction was 12 percent. By the sixth month of the study, 52 percent of patients treated with Oxlumo had reached normal 24-hour uric acid levels;In the second study, 16 patients under the age of 6 were treated with Oxlumo. Using another measurement of herbic acid in urine, studies showed that by the sixth month of the study, the average level of herbal acid in urine had decreased by 71%.Oxlumo was awarded the title of Orphan Drug, an incentive designed to encourage and encourage the development of drugs for rare diseases. The app also won the title of Breakthrough Therapy. In addition, manufacturers received rare paediatric disease priority review vouchers. The FDA's Priority Review Voucher Program for Rare Pediatric Diseases is designed to encourage the development of new drugs and biologics to prevent and treat rare diseases in children.Oxlumo's most common side effects include injection site reactions and abdominal pain. (cyy123.com)source of the original text: