Teva Pharmaceutical Industries (NYSE: TEVA) has submitted a New Drug Application (NDA) to the US FDA for ecopipam, an investigational selective dopamine D1 receptor antagonist, seeking approval for the treatment of pediatric Tourette syndrome. If approved, ecopipam would represent the first new FDA-authorized therapy for pediatric tic disorder in more than a decade and the first agent in clinical use to target the D1 receptor subtype in this indication. The filing arrives weeks after Teva closed its acquisition of Emalex Biosciences, the Chicago-based company that developed ecopipam through Phase III, in a deal valued at up to USD 900 million.
The NDA is a standard submission seeking approval for ecopipam in pediatric patients with Tourette syndrome, the company said. Ecopipam has previously received both Orphan Drug Designation and Fast Track Designation from the FDA for this indication. The Orphan Drug Designation reflects the patient population of fewer than 200,000 in the US, and the Fast Track Designation is intended to facilitate a more expedited review process. No PDUFA target action date has yet been disclosed, as FDA acceptance of the filing has not been confirmed.
The NDA is supported by Phase III data published in JAMA Neurology in May 2026, which reported results from a randomized, placebo-controlled withdrawal study in pediatric patients. The trial enrolled 216 participants, including 167 children and adolescents, who received open-label ecopipam for 12 weeks. Those who achieved a clinical response were then randomized to continue ecopipam or switch to placebo for a further 12 weeks. The primary efficacy endpoint was time to relapse, measured using the Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS). The study demonstrated a statistically significant benefit for ecopipam over placebo in the pediatric population, with a reported p-value of 0.008. Continued ecopipam was associated with a 50% reduction in relapse risk compared with placebo, the company said. The safety profile was described as generally well tolerated; the most frequently reported adverse events in the ecopipam arm included somnolence (11.1%), anxiety (9.7%), headache (9.7%), insomnia (8.8%), tic (7.9%), and fatigue (6.5%).