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Ultragenyx Announces Approval of Dojolvi™ (UX007/triheptanoin) in Canada for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children
First Approved Treatment for Adult and Pediatric Patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) in Canada NOVATO, Calif., Feb. 17, 2021
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"First Approved Treatment for Adult and Pediatric Patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) in Canada\nNOVATO, Calif., Feb. 17, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Health Canada has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of adult and pediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD). Long-chain fatty acid oxidation disorders (LC-FAOD) are a group of rare, genetic, life-threatening disorders caused by defects in the enzymes needed to produce energy from fatty acids. Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride specifically designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD. “Living with a serious disease like LC-FAOD comes with unique challenges such as management of caloric intake and around-the-clock monitoring of symptom deterioration and/or fatigue. We are pleased to bring Canadian patients with LC-FAOD an approved therapy to help them manage their disease,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “This would not have been possible without the LC-FAOD community, including all the patients, families and physicians who participated in the clinical trials. Similar to the approach we took in the United States to ensure access following FDA approval of Dojolvi, our goal is to ensure that patients in Canada with LC-FAOD who might benefit from Dojolvi will have access to it.” LC-FAOD are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to convert long-chain fatty acids into energy. The inability to produce energy from fat can lead to marked depletion of glucose in the body and serious complications, which can lead to hospitalizations or early death. LC-FAOD are included in newborn screening panels across Canada due to the risk of serious outcomes, including death early in life. Other current treatment options for LC-FAOD include avoidance of fasting, low-fat/high-carbohydrate diets, carnitine and even-carbon medium-chain triglyceride (...