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US FDA accepts Ultragenyx's application for rare genetic disorder drug

Ultragenyx Pharmaceutical Inc. said the U.S. Food and Drug Administration accepted its new drug application for UX007 to treat a group of rare genetic disorders that prevent the body from breaking down certain fats into energy.

The Novato, Calif.-based biotechnology company plans to market UX007, or triheptanoin, as a treatment for patients with long-chain fatty acid oxidation disorders. Patients with these disorders are unable to use body fat for energy, which can lead to severe depletion of glucose in the body and serious complications for the liver, muscles and heart.

The FDA expects to take action on the application by July 31, 2020. The application is based on results from a phase 2 trial along with data from a long-term safety and efficacy extension study and a randomized controlled trial that studied the drug's effect on heart function.

UX007 was granted fast-track and rare-pediatric-disease designations from the FDA for treating long-chain fatty acid oxidation disorders in April.

Ultragenyx discontinued the development of UX007 for another genetic metabolic disorder, glucose transporter type-1 deficiency syndrome, in October 2018 after the drug failed to improve symptoms of the disease in a late-stage study.