Teva Pharmaceuticals has announced that the US Food and Drug Administration has accepted the new drug application for ecopipam, a first‑in‑class investigational therapy for paediatric patients with Tourette syndrome. The FDA has granted priority review, with a targeted action date in the first quarter of 2027.
Ecopipam is a selective D1 dopamine receptor antagonist with Orphan Drug designation. Teva said the milestone advances its Pivot to Growth strategy by using its neuroscience expertise to support patients in areas of high unmet need.
Tourette syndrome is a debilitating neuro‑developmental condition affecting around 100,000 children and adolescents in the US. Only half receive prescription medication for the condition and just 20–30% remain on therapy after one year.
Many continue to experience inadequate control or treatment‑limiting side effects.
Eric Hughes, Executive Vice President, Global R&D and Chief Medical Officer at Teva, said: “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.
“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 is supported by positive phase 2b and phase 3 data. In the phase 2b study, ecopipam produced statistically significant and clinically meaningful improvement in tic severity on the Yale Global Tic Severity Scale‑Total Tic Score at week 12. Durability of efficacy was shown in a subsequent open‑label extension.
A phase 3 randomized withdrawal study published in JAMA Neurology demonstrated maintenance of efficacy. Pediatric responders had a 53% decreased risk of relapse over 12 weeks compared with placebo.
Across phase 2b, phase 2b open‑label extension and phase 3 trials, no clinically meaningful changes were observed in body weight, BMI Z‑score, vitals, laboratory measures, ECG readings, drug‑induced movement disorders or psychiatric comorbidities.
Ecopipam was generally well‑tolerated, with the most common adverse events including headache, insomnia, fatigue, somnolence, tics, anxiety, nausea and restlessness.
Teva said it remains committed to advancing the clinical programme and, if approved, delivering a long‑awaited treatment option to pediatric patients with Tourette syndrome who have historically relied on therapies developed for other conditions.
