FDA grants priority review to Teva’s ecopipam, potentially the first new Tourette treatment in over a decade

Teva’s experimental drug ecopipam, a novel dopamine D1 receptor blocker, has received FDA priority review, bringing hope for a new treatment for Tourette syndrome after more than 50 years since the last innovation.

The US Food and Drug Administration has accepted Teva Pharmaceutical Industries’ application for ecopipam and given it priority review, putting the experimental Tourette syndrome treatment on a faster regulatory track. Teva said the agency has set a target decision date in the final quarter of the first quarter of 2027. If approved, the drug would become the first new treatment for Tourette syndrome in more than a decade and the first with a new mechanism in more than 50 years, according to the company.

Tourette syndrome is a chronic neurodevelopmental condition marked by involuntary motor and vocal tics, usually beginning in childhood. Teva has argued that the need for better options is significant, noting that many children diagnosed with the disorder do not receive prescription treatment and that persistence on therapy remains low. Current medicines are largely dopamine D2 receptor blockers, which can cause side effects including sleepiness, movement problems and weight gain.

Ecopipam takes a different approach. It is designed to selectively block dopamine D1 receptors rather than D2 receptors, a distinction researchers say could matter if D1 hypersensitivity contributes to the repetitive movements seen in Tourette syndrome. The drug has already received orphan drug and fast track designations from the FDA, which are intended to speed development for serious conditions affecting relatively small patient groups.

The filing is backed by phase 2b and phase 3 data. In the phase 3 study published in JAMA Neurology, 216 paediatric and adult participants at 77 sites first underwent a 12-week open-label stabilisation period before responders were randomised to stay on ecopipam or switch to placebo. Among children, 41.9% relapsed on ecopipam compared with 68.1% on placebo, a relative risk reduction of 53%, while the treatment was generally well tolerated with no clinically meaningful changes in weight, vital signs, laboratory results, electrocardiograms or psychiatric measures. Teva said the earlier phase 2b study also showed a statistically significant and clinically meaningful fall in tic severity, with benefits sustained in longer-term follow-up.