1 August 2019 - Ultragenyx today announced that it has submitted to the U.S. FDA a new drug application for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders, a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy. 

The FDA previously granted rare paediatric 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.

The submission 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 randomised controlled investigator-sponsored study of 32 patients showing an effect on cardiac function.

Read Ultragenyx press release