The FDA has granted an orphan drug designation to valbenazine (Ingrezza, Neurocrine Biosciences) for the treatment of pediatric patients with Tourette syndrome, a neurological disorder characterized by motor and vocal tics. Tourette syndrome becomes evident in early childhood or adolescence.
Valbenazine, a novel selective vesicular monoamine transporter 2, is marketed in the United States as Ingrezza capsules for the treatment of adults with tardive dyskinesia. The drug is in clinical development for the treatment of Tourette syndrome.
Tourette syndrome consists of rapid, nonrhythmic stereotyped motor and vocal tics. Motor tics are typically characterized by facial grimacing, head jerks, extremity movements, and other stereotyped movements. Vocal tics typically include grunting, throat clearing, and repeating words and phrases. The average age of onset for Tourette syndrome is 6 years, with symptoms reaching their peak severity at approximately age 10. Tourette syndrome is more commonly diagnosed in boys than girls and may be associated with attention-deficit/hyperactivity disorder and obsessive-compulsive disorder.
Orphan drug designation is granted by the FDA to drugs that are intended to treat rare diseases or conditions for patients in the U.S. The orphan drug designation makes the orphan drug indication for the drug eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as other development assistance and financial incentives.
Source: Neurocrine Biosciences; October 23, 2017.