The U.S. FDA has accepted Teva Pharmaceuticals' NDA for ecopipam, a first-in-class investigational therapy to treat Tourette syndrome in pediatric patients.
Ecopipam (EBS-101) is an oral selective dopamine (D1) receptor antagonist that reduces D1 receptor hypersensitivity to reduce repetitive and compulsive behaviors. Acceptance of the NDA is based on positive data from clinical trials. In a phase 2b study, the drug led to a statistically-significant reduction in tic severity at week 12, based on the Yale Global Tic Severity Scale-Total Tic Score, compared to a placebo. Efficacy is further supported by a phase 3 study, where the drug decreased the risk of relapse over 12 weeks by 53% compared to a placebo.
Ecopipam already had Orphan Drug designation; the FDA has now also granted it priority review, with a PFUFA date of the first quarter of 2027. If it receives approval, the drug will become the first new treatment for pediatric patients with Tourette syndrome in 10 years.