The first-in-class D1 receptor antagonist earns Priority Review and Orphan Drug designation, with an FDA action date late in Q1 2027.
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Only half of the estimated 100,000 US children and adolescents with Tourette syndrome receive prescription treatment, and just 20% to 30% remain on therapy after 1 year, according to Teva.¹ Existing agents were developed primarily for other conditions, and no new treatment option or novel mechanism has reached this population in more than a decade, per the company.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,” said Eric Hughes, MD, PhD, executive vice president, Global R&D and chief medical officer, at Teva, in a statement.
The NDA acceptance for ecopipam rests on positive data from the phase 2b and phase 3 D1AMOND trials.²,³ The phase 2b D1AMOND study was a 12-week, randomized, double-blind, placebo-controlled trial enrolling 153 pediatric participants across 68 sites in North America and Europe, with the primary endpoint defined as change from baseline in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS).2 Patients receiving ecopipam showed a statistically significant and clinically meaningful reduction in YGTSS-TTS versus placebo at week 12 (P =.01).2
The subsequent phase 3 D1AMOND trial was a double-blind, placebo-controlled, randomized withdrawal study enrolling 216 pediatric and adult participants into an open-label stabilization period, then randomizing 104 responders, 90 pediatric and 14 adult, across 77 sites.3 For the primary endpoint, pediatric responders assigned to ecopipam had a 53% decreased risk of relapse over 12 weeks compared with placebo (P =.008).² The accepted NDA and sought indication cover pediatric patients exclusively.3
Durability of effect was demonstrated in participants who enrolled in the phase 2b open-label extension (OLE) study, which followed 121 pediatric subjects for up to 12 months to evaluate long-term safety and tolerability.⁴ Across the phase 2b, OLE, and pase 3 trials, Teva reported no clinically meaningful changes in body weight or BMI Z-score, vital signs or laboratory measures including metabolic parameters, electrocardiogram measurements, drug-induced movement disorders as assessed by the Abnormal Involuntary Movement Scale, Barnes Akathisia Rating Scale, or Extrapyramidal Symptom Rating Scale, and measures of psychiatric comorbidities.1
Ecopipam was generally well tolerated across the clinical program.1 The most common adverse events reported in pediatric patients with Tourette syndrome were headache, insomnia, fatigue, somnolence, tics, anxiety, nausea, and restlessness.1 No new safety signals emerged relative to prior studies, according to Teva.¹
Ecopipam is designed to block dopamine signaling at the D1 receptor, a mechanism Teva says may address D1 receptor hypersensitivity implicated in the repetitive and compulsive behaviors of Tourette syndrome.¹ The drug carries Orphan Drug designation, reserved for patient populations of 200,000 or fewer, and the FDA action date is set 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,” Hughes added.
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