Key Takeaways:
The FDA granted priority review to Teva's ecopipam for pediatric Tourette syndrome, setting a PDUFA action date for late Q1 2027.
"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," Eric Hughes, MD, PhD, executive vice president of global R&D and chief medical officer at Teva, said.
The application draws on the phase 2b and phase 3 D1AMOND program. In the 12-week, randomized, double-blind phase 2b trial, 153 pediatric participants across 68 North American and European sites showed a statistically significant and clinically meaningful reduction in the Yale Global Tic Severity Scale-Total Tic Score versus placebo at week 12 (P = .01). A subsequent open-label extension followed 121 participants for up to 12 months and reported sustained tic control.
The phase 3 randomized-withdrawal study, published in JAMA Neurology, enrolled 216 pediatric and adult participants across 77 sites. Among 90 pediatric responders randomized to continued treatment or placebo withdrawal, 41.9 percent relapsed on ecopipam versus 68.1 percent on placebo, a 53 percent relative reduction in relapse risk (hazard ratio, 0.5; P = .008).
Across all three studies, investigators reported no clinically meaningful changes in body weight, BMI, vital signs, laboratory or ECG parameters, movement-disorder scales, or psychiatric comorbidity measures. The most common adverse events were headache, insomnia, fatigue, somnolence, tics, anxiety, nausea, and restlessness.
Ecopipam works by selectively blocking dopamine signaling at the D1 receptor, a mechanism distinct from the D2 receptor antagonists that dominate current therapy. Haloperidol, pimozide, and aripiprazole — the three FDA-approved agents for tic suppression — carry risks of extrapyramidal symptoms, sedation, and metabolic effects. The drug previously received orphan drug designation.
Tourette syndrome affects roughly 174,000 children in the US, according to CDC data, with tics typically emerging between ages 5 and 10. Only about half of affected children receive prescription treatment, and just 20 to 30 percent remain on therapy after one year, according to Teva.
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. The priority review shortens the review timeline, and investors will watch the PDUFA decision in late Q1 2027 as the next catalyst for Teva's neuroscience pipeline.
This article is for informational purposes only and does not constitute investment advice.
