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Ultragenyx Announces U.S. FDA Approval of Dojolvia?? (UX007/triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders

Fourth U.S. FDA Approval for Ultragenyx in the Past Three Years a?? All for Rare Diseases which Previously Had No Approved Therapies
Ultragenyx to Host Conference Call Today at 4:00 pm Eastern TimeNOVATO, Calif., June 30, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc.A (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that theA U.S. Food and Drug AdministrationA (FDA) has approved Dojolvia?? (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD are a group of rare, lifelong and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy. 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.a??With todaya??s FDA approval, patients living with this serious, unpredictable, and often catastrophic disease now have an approved therapy. Many patients with long-chain fatty acid oxidation disorders have difficult lives with frequent hospitalizations and major medical events despite the best current care. Now these patients have an approved treatment as an option to help manage their disease,a?? said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. a??We are grateful to the patients, caregivers, families, dietitians and doctors involved for helping to make this community milestone possible, and we are committed to ensuring that all patients in the U.S. with LC-FAOD who might benefit from Dojolvi will have access to it.a??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 severe 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 the U.S. and in certain European countries due to the risk for serious outcomes including death early in life. A Other current treatment options for LC-FAOD include avoidance of fasting, low-fat/high-carbohydrate diets, carnitine and even-carbon medium-chain triglyceride (MCT) oil, a medical food product. LC-FAOD affect an estimated 2,000 to 3,500 children and adults in the United States.
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