Teva Pharmaceuticals' ecopipam receives priority review by the FDA, supported by studies showing sustained tic control among pediatric patients with Tourette syndrome.
The FDA has accepted Teva Pharmaceuticals' new drug application (NDA) for ecopipam, a first-in-class selective dopamine D1 receptor antagonist under investigation for pediatric patients with Tourette syndrome and granted the submission priority review. The company noted that the agency set a target action date under the Prescription Drug User Fee Act for late in the first quarter of 2027.1
"We are encouraged by the FDA's acceptance of the ecopipam application and grant of priority review. Tourette syndrome is an area where innovation has been limited for far too long, with patients and clinicians often relying on treatments developed for other conditions,” Eric Hughes, MD, PhD, executive vice president, global R&D and chief medical officer at Teva, told NeurologyLive®.
“The phase 2b and phase 3 data supporting this application demonstrated meaningful and durable reductions in tic severity with a favorable tolerability profile, giving us confidence in ecopipam's potential as a differentiated treatment option,” Hughes added. “If approved, we believe ecopipam's novel D1 receptor mechanism could provide clinicians with an important new option to help children and adolescents better manage their symptoms and the impact those symptoms can have on daily life."
The NDA application is supported by data from the
READ MORE:
The
Across all 3 studies, investigators reported no clinically meaningful changes in body weight, body mass index, vital signs, laboratory or ECG parameters, movement-disorder scales, or psychiatric comorbidity measures. The most common adverse events included headache, insomnia, fatigue, somnolence, tics, anxiety, nausea, and restlessness.
Tourette syndrome is a chronic neurodevelopmental disorder marked by involuntary motor and vocal tics that typically emerge between ages 5 and 10 years.4 National survey data estimate that roughly 1 in 333 children ages 3 to 17 years in the US, about 174,000 children, carry a Tourette syndrome diagnosis, with nearly half experiencing moderate to severe symptoms.4 According to Teva, only about half of affected children receive prescription treatment, and just 20% to 30% remain on therapy after one year.1 Current pharmacotherapy is dominated by D2 dopamine receptor antagonists, such as haloperidol, pimozide, and aripiprazole, which carry risks of extrapyramidal symptoms, sedation, and metabolic effects.
Ecopipam works through a mechanistically distinct pathway, selectively blocking dopamine signaling at the D1 receptor instead of D2. D1 receptor hypersensitivity has been proposed as a contributor to the repetitive, compulsive motor patterns characteristic of Tourette syndrome. The drug previously received orphan drug designation, reserved for conditions affecting 200,000 or fewer patients in the US.1
