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Ultragenyx seeks US FDA approval for rare genetic disorder treatment

Ultragenyx Pharmaceutical Inc. is seeking approval from the U.S. Food and Drug Administration for its drug candidate 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 is developing UX007, or triheptanoin, for patients with long-chain fatty acid oxidation disorders. Patients with these disorders are unable to use body fat for energy that can lead to severe depletion of glucose in the body and serious liver, muscle and heart disease.

Ultragenyx's new drug application for UX007 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.

The FDA granted fast-track and rare-pediatric-disease designations to UX007 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 after the drug failed to improve symptoms of the disease.