FDA has accepted Teva Pharmaceuticals’ New Drug Application for ecopipam, an investigational treatment for pediatric patients with Tourette syndrome, and granted the submission Priority Review.
The agency set a target action date in late first-quarter 2027. If approved, ecopipam would be the first new treatment indicated for children with Tourette syndrome in more than 10 years and the first to use a novel mechanism in more than 50 years, according to Teva.
Ecopipam, also known as EBS-101, is a selective dopamine D1 receptor antagonist. It is designed to block dopamine signaling at the D1 receptor, which Teva said may contribute to repetitive and compulsive behaviors associated with Tourette syndrome. The drug has received FDA Orphan Drug designation.
Tourette syndrome is a chronic neurodevelopmental condition involving involuntary motor and vocal tics that generally begin in childhood, often between ages 5 and 10. Teva said approximately 100,000 U.S. children and adolescents are affected. It said only about half receive prescription treatment and an estimated 20% to 30% remain on therapy after one year.
The application is supported by Phase 2b and Phase 3 data. In the 12-week D1AMOND Phase 2b trial, involving 153 pediatric participants across 68 sites in North America and Europe, ecopipam produced a statistically significant and clinically meaningful improvement in Yale Global Tic Severity Scale–Total Tic Score compared with placebo, with a p-value of 0.01.
The subsequent Phase 3 randomized-withdrawal trial evaluated maintenance of efficacy in responders. Pediatric patients receiving ecopipam had a 53% lower risk of relapse over 12 weeks compared with placebo, with a p-value of 0.008. The trial included 216 pediatric and adult participants in an open-label stabilization period, with 104 randomized; however, Teva’s NDA seeks an indication only for pediatric patients.
Across the Phase 2b, open-label extension and Phase 3 studies, Teva said no clinically meaningful changes were observed in body weight, body mass index z-score, metabolic measures, ECG results, movement-disorder measures or psychiatric comorbidity assessments. The most common adverse events were headache, insomnia, fatigue, somnolence, tics, anxiety, nausea and restlessness.
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