A new drug designed specifically for children with Tourette syndrome has shown it can cut the risk of relapse by half, according to results from a large phase III clinical trial published in JAMA Neurology. The medication, called ecopipam, is the first treatment developed from the ground up to address the motor and vocal tics that define the condition. Its maker, Emalex Biosciences, now plans to seek approval from the U.S. Food and Drug Administration.
Tourette syndrome affects about 1 percent of school-aged children, typically appearing before age 10 and more often in boys. The disorder causes uncontrolled movements like blinking and shrugging, as well as vocal sounds such as grunting. For decades, doctors have had to rely on antipsychotic drugs originally approved for schizophrenia, including haloperidol, pimozide and aripiprazole. These medications target the dopamine D2 receptor in the brain and can cause harsh side effects including weight gain, stiffness, shakiness and, in rare cases, a movement disorder called tardive dyskinesia.
Ecopipam works differently by blocking the dopamine D1 receptor instead. The phase III trial enrolled 216 participants, including 167 children and teens, and began with 12 weeks of open label treatment. Then 104 participants, 90 of them pediatric, were randomized to receive either ecopipam or a placebo for another 12 weeks. Those who stayed on the drug showed a 50 percent reduction in relapse risk compared with the placebo group. Side effects were generally mild to moderate and consistent with earlier studies. Notably, patients did not gain weight or develop other movement disorders.
“Overall, the patients taking ecopipam got significant help with their tics, didn’t gain weight, and didn’t develop other movement disorders,” said Dr. Donald Gilbert, a movement disorders expert at Cincinnati Children’s and the principal investigator of the trial. “That’s an exciting combination that could make pediatricians more willing to prescribe treatment and families more willing to consider medication.”
The FDA has already granted ecopipam Orphan Drug and Fast Track designations for pediatric Tourette syndrome. While the drug is not yet approved, participants from the trial have been allowed to continue taking it, and an expanded access program is available for eligible patients who cannot use existing medications. Researchers and the drugmaker are now preparing to submit a formal approval application, with hopes that this could become the first medication designed specifically for Tourette syndrome to reach the U.S. market.