Investigational Therapy for Tourette Syndrome Receives FDA Fast Track Designation 

The Food and Drug Administration (FDA) has granted fast-track status to the investigational drug ecopipam (Emalex Biosciences, Inc., Northbrook, IL) being studied for treatment of Tourette’s syndrome (TS). Fast-track status is designated when a potential new therapy is intended to treat a serious condition and data demonstrate the potential to address an unmet medical need. Earlier this month, enrollment a phase 2b clinical study (NCT04007991) of ecopipam was initiated in children and adolescent s (age 6-18 years) with TS at multiple study centers in the US, Canada, and European Union.

Ecopipam selectively blocks dopamine at the D1 receptor. Previous phase 2 studies of the therapy in adults and children with TS suggested potential activity in reducing the frequency and severity of motor and vocal tics associated with the condition. Supersensitivity of the D1 dopamine receptor is thought to be a potential mechanism for the repetitive and compulsive behaviors associated with TS. Currently approved therapies for TS act at D2 dopamine receptors.

 “We are encouraged that the FDA has granted fast-track designation to ecopipam for the treatment of patients with TS, a serious disease with significant morbidity,” said Atul Mahableshwarkar, MD, senior vice president of drug development, Emalex. “The unmet medical need for patients with TS is significant and we look forward to working with the FDA throughout the development process for this novel compound.”