Development Program for Glut1 Deficiency Syndrome Drug to Be Discontinued

The study did not achieve its primary endpoint
The study did not achieve its primary endpoint

Ultragenyx Pharmaceutical has announced plans to discontinue the development of the investigational treatment UX007 for patients with glucose transporter type-1 deficiency syndrome (Glut1 DS) experiencing disabling paroxysmal movement disorders. 

The decision was made after results from a Phase 3 trial (N=44) comparing UX007 to placebo did not show a statistically significant reduction in paroxysmal movement events in favor of the treatment (primary endpoint). Key secondary endpoints (walking capacity and endurance, patient-reported health-related quality of life assessments of physical function, mobility, upper extremity function, fatigue, and pain, and duration of disabling paroxysmal movement disorder events) were also not met.

Related Articles

"We had previously observed significant improvements in individual cases of Glut1 DS with UX007 and so we are particularly disappointed by the results of the Glut1 DS study in a larger group of patients that did not demonstrate this same effect," said Emil Kakkis, MD, PhD, CEO and President of Ultragenyx.

UX007 is also being evaluated in the treatment of long-chain fatty oxidation disorders.

For more information visit Ultragenyx.com.