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Ultragenyx Announces FDA Accepts New Drug Application for UX007 (triheptanoin) for Treatment of Long-chain Fatty Acid Oxidation Disorders
NOVATO, Calif., Oct. 14, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"NOVATO, Calif., Oct. 14, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy. The FDA has assigned a standard review designation with a Prescription Drug User Fee Act (PDUFA) target date of July 31, 2020.\n “We appreciate FDA’s collaboration on evaluating these data for this devastating and often catastrophic disease,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We look forward to working with the agency in the coming months with the goal of bringing this potential new treatment to patients as quickly as possible.\" The FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application. The NDA filing is supported by a comprehensive package of data including results from a company-sponsored Phase 2 study of UX007 in 29 patients, a long-term safety and efficacy extension study in 75 patients including 20 patients who were previously naïve to UX007, a retrospective medical record review of 20 original compassionate use patients, 67 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect on cardiac function. About LC-FAOD 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 liver, muscle and heart disease, 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. Current treatment options for LC-FAOD include avoidance of fasting, low-fat/high-carbohydrate diets, carnitine and medium-chain triglyceride (MCT) oil, a medical food product. Despite current management, many patients with LC-FAOD experience signific...