FDA Grants Priority Review to First-in-Class Ecopipam for Pediatric Tourette Syndrome

The US Food and Drug Administration has accepted Teva Pharmaceuticals’ New Drug Application for ecopipam for the treatment of pediatric patients with Tourette syndrome and granted the application Priority Review, according to a company announcement.¹

The FDA set a target action date in late first quarter 2027. Ecopipam, also known as EBS-101, is an investigational, first-in-class selective dopamine D1 receptor antagonist with Orphan Drug designation for pediatric Tourette syndrome.¹

If approved, ecopipam would be the first new therapy indicated for pediatric patients with Tourette syndrome in more than 10 years and the first novel mechanism of action for the condition in more than 50 years, according to Teva.¹

Why is this relevant to primary care?

Tourette syndrome is a chronic neurodevelopmental disorder characterized by involuntary motor and vocal tics that typically begin in childhood. Symptoms can be visible, frequent, and disruptive, with potential effects on school functioning, social interaction, emotional health, and quality of life.¹

Tourette syndrome may first be recognized in primary care, often in children with coexisting attention-deficit/hyperactivity disorder (ADHD), obsessive-compulsive symptoms, anxiety, or other behavioral health concerns. In the published phase 3 trial, 59.7% of participants had at least 1 psychiatric comorbidity, including ADHD in 40.7%, obsessive-compulsive disorder in 20.4%, anxiety in 18.5%, and depression in 7.9%.²

Current pharmacologic treatment for Tourette syndrome may be limited by adverse effects and discontinuation. Behavioral therapies are recommended as first-line treatment, but when they are unavailable, ineffective, or not preferred, pharmacologic options include α-2 adrenergic receptor agonists or dopamine D2 receptor modulators, which can be limited by adverse events.²

“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 of Teva, said in the announcement.¹

What data supported the NDA?

The NDA is supported by positive phase 2b and phase 3 data. In the phase 2b D1AMOND study, pediatric patients treated with ecopipam had a statistically significant and clinically meaningful improvement in Yale Global Tic Severity Scale–Total Tic Score (YGTSS-TTS) vs placebo at week 12 (P = .01), according to Teva.¹

The phase 3 D1AMOND trial, published in JAMA Neurology, was a double-blind, placebo-controlled, randomized withdrawal trial conducted at 77 sites in 12 countries between January 31, 2023, and February 4, 2025. The trial enrolled individuals aged 6 years and older with Tourette syndrome.²

Participants entered a 12-week open-label period in which ecopipam was titrated over 3 to 4 weeks to a target dose of 1.8 mg/kg per day. Responders, defined as participants with at least a 25% improvement from baseline in YGTSS-TTS at both weeks 8 and 12, were randomly assigned to continue ecopipam or taper to placebo for a 12-week double-blind withdrawal period.²

What were the phase 3 efficacy findings?

The phase 3 trial enrolled 216 participants into the open-label ecopipam period, including 167 pediatric participants. Among responders entering the randomized withdrawal period, 43 pediatric participants were assigned to continue ecopipam and 47 were assigned to placebo.²

Ecopipam significantly reduced relapse risk compared with placebo among pediatric participants, with a hazard ratio of 0.47 (95% CI, 0.26-0.84; P = .008; n=90). Teva described this as a 53% decreased risk of relapse over 12 weeks.¹˒²

In the overall population, exploratory end points also favored ecopipam. From randomization to end of treatment, least-squares mean change in YGTSS-TTS was 3.3 with ecopipam vs 8.4 with placebo, for a between-group difference of –5.1 (95% CI, –8.8 to –1.4; P = .009).²

What safety findings were reported?

Across the phase 2b, phase 2b open-label extension, and phase 3 studies, Teva reported no clinically meaningful changes in body weight, BMI z score, vital signs, laboratory measures including metabolic parameters, electrocardiogram measurements, drug-induced movement disorder measures, or psychiatric comorbidity measures.¹

In the phase 3 trial, the most frequently reported adverse events with ecopipam across the open-label and double-blind periods were somnolence, anxiety, headache, insomnia, tic, and fatigue. Study authors reported no clinically meaningful impact on weight, metabolic parameters, or psychiatric scale measures, and no observed drug-induced movement disorders.²

Teva stated that across the clinical program, ecopipam was generally well tolerated. The most common adverse events in pediatric patients with Tourette syndrome were headache, insomnia, fatigue, somnolence, tics, anxiety, nausea, and restlessness.¹

For clinicians, ecopipam remains investigational. The NDA acceptance and Priority Review status mark a regulatory milestone but do not indicate FDA approval. Teva said the accepted NDA and proposed indication are exclusively for pediatric patients, although the phase 3 trial included both pediatric and adult participants.¹

References

1. Teva Pharmaceuticals. 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. Published 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. 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