FDA Orphan Drug Designation Granted to Treatment for Pediatric Tourette Syndrome Patients

Mathew Shanley

On Monday, Neurocrine Biosciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to valbenazine for the treatment of pediatric patients with Tourette syndrome.

In the United States, valbenazine is marketed as Ingrezza capsules and is the only treatment approved for adults with tardive dyskensia, a movement disorder that can arise following the use some antipsychotic medications. The new designation means that the sponsor qualifies for certain benefits, but it does not indicate its safety or efficacy, or allow for its ability to be manufactured and marketed in the United States.

Tourette syndrome is a neuropsychiatric disorder characterized by motor and phonic movement issues, which are frequently accompanied by neurobehavioral disorders like attention-deficit hyperactivity disorder (ADHD) and obsessive-compulsive disorder (OCD). Diagnosis is usually made around the age of 7, and almost always before the child reaches his or her 18th birthday.

Per the Children’s National Health System, the most common symptoms associated with Tourette syndrome include: head jerking, squinting, blinking, shrugging, grimacing, and nose-twitching, among others.

"We are pleased that the FDA has granted valbenazine orphan drug designation to treat pediatric patients with Tourette syndrome as it represents another significant regulatory milestone for our company," said Malcolm Lloyd-Smith, Chief Regulatory Officer at Neurocrine Biosciences in a press release. "We will continue our clinical development program for Tourette's focused on pediatric patients as the first symptoms of Tourette syndrome typically present early in childhood and represent a significant challenge for these young patients."

Valbenazine is a novel selective vesicular monomine transporter 2 (VMA2) inhibitor and operates by reducing the amount of dopamine released in regions of the brain that control movement and motor function.

A phase 2b study is underway, testing the safety and efficacy of valbenazine in 120 pediatric patients with Tourette syndrome, and results are expected in 2018. An earlier phase 2 study in adults with Tourette syndrome failed to meet its primary endpoint despite meeting other secondary endpoints being met.

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