Ultragenyx Pharmaceutical Inc., submits NDA to FDA for UX 007 to treat long-chain fatty acid oxidation disorders .

Ultragenyx Pharmaceutical Inc.announced that it has submitted to the FDA a New Drug Application (NDA) for UX 007 (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 previously granted Rare Pediatric Disease Designation and Fast Track designation, which enables eligibility for Priority Review, if relevant criteria are met. Ultragenyx expects to hear back from FDA on submission acceptance and review designation within 60 days.

Fatty Acid Oxidation Disorders: 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. Patients with LC-FAOD are currently treated with the avoidance of fasting, low-fat/high carbohydrate diets, carnitine, and medium-chain triglyceride (MCT) oil, a medical food product. Despite current therapy, many patients have significant metabolic events including hospitalizations and mortality due to LC-FAOD.

The NDA submission is supported by a comprehensive package of data including results from a company-sponsored Phase II study of UX 007 in 29 patients, a long-term safety and efficacy extension study in 75 patients including 20 patients who were previously na�ve to UX 007, 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.