Ultragenyx Pharmaceutical Inc Announces U.S. FDA Approval of Dojolvi™ For The Treatment of Long-Chain Fatty Acid Oxidation Disorders

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Dojolvi™ (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.

“With today’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,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “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.”