FDA Accepts NDA, Grants Priority Review to Ecopipam for Pediatric Tourette Syndrome

The FDA has accepted Teva Pharmaceuticals' new drug application (NDA) for ecopipam, a first-in-class selective dopamine D1 receptor antagonist under investigation for pediatric patients with Tourette syndrome (TS), and granted the submission priority review. The company noted that the agency set a target action date under the Prescription Drug User Fee Act (PDUFA) for late in the first quarter of 2027.¹

“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,” Eric Hughes, MD, PhD, executive vice president, global R&D and chief medical officer at Teva, said in a statement.¹ “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 NDA application is supported by data from the phase 2b and phase 3 D1AMOND clinical program. In the 12-week, randomized, double-blind, placebo-controlled phase 2b trial, 153 pediatric participants across 68 sites in North America and Europe were randomized to ecopipam or placebo. Findings showed that those receiving ecopipam had a statistically significant and clinically meaningful reduction in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) versus placebo at week 12 (P = .01).^1,2 A subsequent open-label extension followed 121 pediatric participants for up to 12 months and reported sustained tic control.

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The phase 3 trial (NCT05615220), a randomized-withdrawal study published in JAMA Neurology, enrolled 216 pediatric and adult participants across 77 sites and began with a 12-week open-label stabilization period.³ Responders (total, n = 104; pediatric, n = 90; adult, n = 14) were then randomized to continue ecopipam or switch to placebo for a 12-week double-blind withdrawal phase. In the pediatric cohort, 41.9% relapsed on ecopipam versus 68.1% on placebo, a 53% relative reduction in relapse risk (hazard ratio, 0.5; P = .008); a secondary analysis combining pediatric and adult responders showed a similar effect.^1,3

Across all 3 studies, investigators reported no clinically meaningful changes in body weight, BMI, vital signs, laboratory or ECG parameters, movement-disorder scales, or psychiatric comorbidity measures. The most common adverse events included headache, insomnia, fatigue, somnolence, tics, anxiety, nausea, and restlessness.

TS is a chronic neurodevelopmental disorder marked by involuntary motor and vocal tics that typically emerge between ages 5 and 10 years.⁴ National survey data estimate that roughly 1 in 333 children ages 3 to 17 years in the US, about 174,000 children, carry a TS diagnosis, with nearly half experiencing moderate to severe symptoms.⁴ According to Teva, only about half of affected children receive prescription treatment, and just 20% to 30% remain on therapy after one year.¹ Current pharmacotherapy is dominated by D2 dopamine receptor antagonists, such as haloperidol, pimozide, and aripiprazole, which carry risks of extrapyramidal symptoms, sedation, and metabolic effects.

Ecopipam works through a mechanistically distinct pathway, selectively blocking dopamine signaling at the D1 receptor rather than D2. D1 receptor hypersensitivity has been proposed as a contributor to the repetitive, compulsive motor patterns characteristic of TS. The drug previously received orphan drug designation, reserved for conditions affecting 200,000 or fewer patients in the US.¹

REFERENCES
1. U.S. FDA Accepts Teva's New Drug Application (NDA) and Grants Priority Review for Ecopipam, a First-in-Class Investigational Therapy for Pediatric Patients with Tourette Syndrome. News release. Teva Pharmaceuticals. August 19, 2026. Accessed August 19, 2026. https://www.tevapharm.com/news-and-media/latest-news/u.s.-fda-accepts-tevas-new-drug-application-nda-and-grants-priority-review-for-ecopipam-a-first-in-cl
2. Ecopipam for Tourette Syndrome: A Randomized Trial. Pediatrics. 2023;151(2):e2022059574. doi:10.1542/peds.2022-059574
3. Efficacy and Safety of Ecopipam for Tourette Syndrome: A Phase 3 Randomized Clinical Trial. JAMA Neurol. Published online 2026. doi:10.1001/jamaneurol.2026.1431
4. Centers for Disease Control and Prevention. Data and Statistics on Tourette Syndrome. Accessed August 19, 2026. https://www.cdc.gov/tourette-syndrome/data/index.html