Teva submits NDA for ecopipam for pediatric Tourette syndrome

Teva Pharmaceutical Industries Ltd announced on June 18 that it has submitted a New Drug Application to the FDA for ecopipam, an investigational dopamine D1 receptor antagonist, for pediatric Tourette syndrome.¹ The filing rests on phase 3 data published in JAMA Neurology showing that continued ecopipam treatment significantly delayed time to relapse versus placebo among pediatric patients who had already responded to the drug.²

"The NDA submission for ecopipam is a significant milestone for a potential first-in-class treatment option in pediatric Tourette syndrome," said Eric Hughes, MD, PhD, executive vice president of global R&D and chief medical officer of Teva. "This reflects the momentum in our innovative pipeline through our recent acquisition of this important asset, and advances our pivot to growth strategy and commitment to bringing differentiated medicines for patients."¹

Trial design and efficacy findings for ecopipam in pediatric Tourette syndrome

The phase 3 program used a randomized withdrawal design. Patients aged 6 years and older entered a 12-week open-label phase with ecopipam titrated to a target dose of 1.8 mg/kg/day.³ Those with at least a 25% improvement from baseline on the Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS) at weeks 8 and 12 were randomized to continue ecopipam or switch to placebo for a 12-week double-blind withdrawal phase.³ Of 104 patients randomized, 90 were pediatric.³

Time to relapse on the YGTSS-TTS scale served as the primary efficacy endpoint.¹ In the pediatric population, continued ecopipam use cut relapse risk by half relative to placebo (hazard ratio, 0.5; 95% CI, 0.3-0.8; P = .008), consistent with the combined pediatric-and-adult cohort (HR, 0.5; 95% CI, 0.3-0.8; P = .005).³ The most common adverse events were somnolence (11.1%), anxiety (9.7%), headache (9.7%), insomnia (8.8%), tic (7.9%), and fatigue (6.5%).¹

Disease burden and unmet need in pediatric Tourette syndrome

Tourette syndrome is a chronic neurodevelopmental disorder marked by involuntary motor and vocal tics that typically emerge between ages 5 and 10.¹ CDC survey data estimate that roughly 1 in 333 children aged 3 to 17 years in the US have a diagnosis, translating to about 174,000 children, with boys affected roughly three times as often as girls; nearly half of affected children may go undiagnosed.⁴

Despite available pharmacologic and behavioral options, many patients continue to experience inadequate symptom control or treatment-limiting adverse effects.¹ Aripiprazole has held FDA approval for pediatric Tourette tics since 2014, alongside older antipsychotics such as haloperidol and pimozide; comprehensive behavioral intervention for tics (CBIT) is also recommended but is not always accessible or sufficient.⁴

Mechanism and regulatory status of ecopipam

Unlike antipsychotic-based tic therapies that block D2 dopamine receptors, ecopipam selectively targets the D1 receptor, a pathway thought to contribute to Tourette syndrome's repetitive, compulsive motor patterns.¹ The drug holds Orphan Drug and Fast Track designations from the FDA for pediatric Tourette syndrome, the former reserved for conditions affecting 200,000 or fewer US patients.¹ It is also available through an expanded access program for patients who have exhausted other options.³

Interpreting the data and remaining questions

The relapse-based endpoint is a conservative durability test, asking whether benefit persists after response rather than measuring acute tic reduction. Reported P values met significance thresholds, and hazard ratios suggest a clinically meaningful effect, though the NDA announcement did not report absolute relapse rates or adverse events by age or psychiatric comorbidity.

Given the high rate of psychiatric comorbidity in Tourette syndrome, including ADHD, anxiety, and OCD, clinicians will want subgroup data before broad adoption.⁵ The double-blind phase covered just 12 additional weeks, leaving longer-term safety beyond roughly six months of exposure to be defined through follow-up data.

Limitations and next steps

The trial's modest randomized cohort (104 patients, 90 pediatric) and enrichment design, which selects early responders before randomization, mean findings may not generalize to nonresponders to initial open-label treatment. The FDA has not set a review timeline, and approval would mark the first new pharmacologic mechanism for pediatric Tourette syndrome in over a decade.

References

1. Teva Pharmaceutical Industries Ltd. Teva submits NDA for ecopipam, a first-in-class investigational therapy for pediatric Tourette syndrome. GlobeNewswire. Published June 18, 2026. Accessed June 24, 2026. https://www.globenewswire.com/news-release/2026/06/18/3314541/0/en/Teva-Submits-NDA-for-Ecopipam-a-First-in-Class-Investigational-Therapy-for-Pediatric-Tourette-Syndrome.html

2. Emalex Biosciences. JAMA Neurology publishes phase 3 data on D1 receptor antagonist ecopipam in Tourette syndrome. Business Wire. Published 2026. Accessed June 24, 2026. https://www.businesswire.com/news/home/20260526869991/en/JAMA-Neurology-Publishes-Phase-3-Data-on-D1-Receptor-Antagonist-Ecopipam-in-Tourette-Syndrome

3. Tomczak K, Gilbert DL, et al. Phase 3 randomized withdrawal trial of ecopipam in Tourette syndrome. JAMA Neurol. 2026. Summarized in: Ecopipam maintains efficacy and reduces relapse risk in phase 3 trial in Tourette syndrome. NeurologyLive. Accessed June 24, 2026. https://www.neurologylive.com/view/ecopipam-efficacy-reduces-relapse-phase-3-tourette-syndrome

4. Centers for Disease Control and Prevention. Data and statistics on Tourette syndrome. Updated March 27, 2026. Accessed June 24, 2026. https://www.cdc.gov/tourette-syndrome/data/index.html

5. Centers for Disease Control and Prevention. Other concerns and conditions of Tourette syndrome. Updated March 27, 2026. Accessed June 24, 2026. https://www.cdc.gov/tourette-syndrome/other-concerns/index.html