Teva Pharmaceuticals is one step closer to bringing a potential new treatment to children and adolescents with Tourette syndrome after the U.S. Food and Drug Administration accepted its New Drug Application for ecopipam.

 

The FDA has set a targeted action date under the Prescription Drug User Fee Act (PDUFA) for late in the first quarter of 2027, putting the investigational therapy on a path toward a potential U.S. approval.

 

Ecopipam is being developed as a first-in-class treatment for pediatric patients with Tourette syndrome, a debilitating neurodevelopmental condition affecting approximately 100,000 children and adolescents in the U.S.

 

Despite existing treatment options, many patients continue to struggle with inadequate tic control or side effects that make long-term treatment difficult. Only about half of affected patients receive prescription medication, and just 20% to 30% remain on therapy after one year.

 

“Ecopipam’s NDA acceptance is an important milestone that advances Teva’s Pivot to Growth strategy and brings us closer to addressing the unmet needs of children and their families affected by Tourette syndrome,” said Eric Hughes, Executive Vice President, Global R&D and Chief Medical Officer of Teva. 

 

“If approved, ecopipam would be the first new therapy for Tourette syndrome in more than 10 years and the first novel mechanism of action in more than 50 years, offering patients and families a long-awaited new treatment option.”

 

The FDA application is backed by positive results from Phase 2b and Phase 3 trials.

 

In the Phase 2b study, children treated with ecopipam showed a statistically significant and clinically meaningful reduction in tic severity compared with placebo after 12 weeks, as measured by the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS). The benefit was also sustained among participants who continued into an open-label extension study.

 

Results from a Phase 3 randomized withdrawal trial, published in JAMA Neurology, provided further evidence that ecopipam could maintain its effects over time. Pediatric patients who responded to ecopipam had a 53% lower risk of relapse over 12 weeks compared with those switched to placebo.

 

Safety findings across the Phase 2b, open-label extension and Phase 3 studies were also encouraging. Teva reported no clinically meaningful changes in body weight or BMI Z-score, vital signs, laboratory measures including metabolic parameters, ECG measurements, drug-induced movement disorders or measures of psychiatric comorbidities.

 

Ecopipam was generally well tolerated. The most common adverse events reported in pediatric patients included headache, insomnia, fatigue, somnolence, tics, anxiety, nausea and restlessness.

 

For Teva, the FDA acceptance marks a significant step in its broader Pivot to Growth strategy and its effort to expand its neuroscience portfolio into areas of high unmet need.

 

The company said it remains committed to advancing the ecopipam program and, if the therapy is approved, providing pediatric Tourette syndrome patients with a new treatment option in a field where available therapies have largely been adapted from treatments developed for other conditions.