FDA accepts ecopipam NDA with priority review for pediatric Tourette syndrome

The FDA has accepted Teva Pharmaceuticals' New Drug Application for ecopipam, a selective dopamine D1 receptor antagonist, for the treatment of pediatric patients with Tourette syndrome, with a target action date in late Q1 2027. The agency granted the application priority review, and ecopipam previously received orphan drug designation for the indication.

“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, MD, PhD, executive vice president of global research and development and chief medical officer at Teva. If cleared, ecopipam would represent the first new Tourette syndrome therapy in over a decade and the first agent in the drug class approved for the condition to work through a mechanism other than D2 receptor blockade.

Ecopipam trial design and efficacy data in pediatric Tourette syndrome

The application draws on a phase 2b trial and a subsequent phase 3 randomized withdrawal study. In the 12-week, double-blind, placebo-controlled Phase 2b trial (D1AMOND), 153 pediatric participants across 68 North American and European sites were randomized to ecopipam or placebo, with the primary endpoint measured on the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS). Ecopipam produced a statistically significant reduction in tic severity versus placebo at week 12. A 12-month open-label extension involving 121 participants supported durability of that effect.

The phase 3 trial enrolled 216 pediatric and adult participants into an open-label stabilization phase, then randomized 104 responders—90 of them pediatric—to continued ecopipam or placebo withdrawal. Pediatric responders who remained on ecopipam had a 53% lower risk of relapse over 12 weeks compared with those switched to placebo. Results were published in JAMA Neurology. Across the clinical program, investigators reported no clinically meaningful changes in body weight, BMI z-score, metabolic labs, ECG parameters, or drug-induced movement disorder scales; the most frequently reported adverse events were headache, insomnia, fatigue, somnolence, tics, anxiety, nausea, and restlessness.

Disease burden and current treatment gaps in Tourette syndrome

Tourette syndrome affects roughly 100,000 children and adolescents in the US, with tic onset typically occurring between ages 5 and 10. Only about half of affected patients receive pharmacologic treatment, and 20% to 30% remain on therapy beyond one year, reflecting persistent tolerability concerns with existing options.

Ecopipam mechanism and prior D2-antagonist approvals for tic disorders

All three FDA-approved agents for tic suppression—haloperidol, pimozide, and aripiprazole—are antipsychotics that act on D2 dopamine receptors and were originally developed for other psychiatric indications. Their use is often limited by weight gain, movement disorders, and metabolic effects. Ecopipam instead targets the D1 receptor, based on the hypothesis that D1 receptor hypersensitivity contributes to the repetitive, compulsive movement patterns seen in Tourette syndrome.

Interpreting the data and remaining questions

The magnitude of relapse-risk reduction reported in the phase 3 trial is clinically notable, and the absence of significant metabolic or movement-disorder signals across three studies is reassuring for a pediatric population that has had few tolerable options. Still, the pivotal phase 3 trial's randomized comparison involved just 104 responders, a modest sample for characterizing rarer adverse events, and the sponsored, single-company nature of the program means independent replication and postmarketing surveillance will matter. The accepted indication is limited to pediatric patients even though adults were included in the phase 3 trial, and durability data beyond 12 months remain limited to open-label extension findings rather than controlled follow-up.

References

1. Teva Pharmaceuticals. U.S. FDA accepts Teva's New Drug Application and grants priority review for ecopipam, a first-in-class investigational therapy for pediatric patients with Tourette syndrome. Press release. August 19, 2026.

2. Gilbert DL, Dubow JS, Cunniff TM, et al. Ecopipam for Tourette syndrome: a randomized trial. Pediatrics. 2023;151(2):e2022059574. doi:10.1542/peds.2022-059574

3. Gilbert DL, Atkinson SD, Kim DJB, et al. Efficacy and safety of ecopipam for Tourette syndrome: a phase 3 randomized clinical trial. JAMA Neurol. 2026;83(7):645-653. doi:10.1001/jamaneurol.2026.1431

4. Pringsheim T, Okun MS, Müller-Vahl K, et al. Practice guideline recommendations summary: treatment of tics in people with Tourette syndrome and chronic tic disorders. Neurology. 2019;92(19):896-906.