Ultragenyx Wins Fast Track for Rare Pediatric Disorder Treatment

The FDA granted fast-track status to Ultragenyx’ UX007, an experimental treatment for a rare and potentially deadly set of pediatric disorders.

UX007 is a synthetic fatty acid triglyceride that aims to help children and adolescents with long-chain fatty acid oxidation disorders, a cluster of about 20 different genetic problems that prevent patients from converting fat into energy. Left untreated, this can cause dangerously low blood-sugar levels and even death.

Ultragenyx hopes to submit a new drug application for UX007 by the middle of this year.