Teva Pharmaceutical Industries has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for ecopipam, an investigational treatment for paediatric Tourette syndrome. The company says the submission marks a significant milestone for what could become the first new FDA-approved therapy for children and adolescents with Tourette syndrome in more than a decade.
The application is supported by positive Phase 3 clinical trial results that were recently published in JAMA Neurology. In the study, ecopipam significantly delayed the time to relapse compared with placebo in paediatric patients who had previously responded to treatment during an open-label phase. The trial met its primary endpoint, demonstrating a statistically significant benefit for patients receiving ecopipam.
Ecopipam is a first-in-class selective dopamine D1 receptor antagonist. Unlike existing therapies that primarily target dopamine D2 receptors, the treatment has a novel mechanism of action designed to block dopamine signalling at the D1 receptor. Researchers believe D1 receptor hypersensitivity may contribute to the repetitive and compulsive behaviours associated with Tourette syndrome.
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According to Teva, the treatment was generally well tolerated in the Phase 3 study. The most commonly reported adverse events included somnolence, insomnia, anxiety, fatigue and headache. The therapy has already received both Orphan Drug and Fast Track designations from the FDA, highlighting its potential importance for a condition with limited treatment options.
Tourette syndrome is a chronic neurodevelopmental disorder characterised by involuntary motor and vocal tics that typically begin in childhood. Symptoms can be disruptive and may affect quality of life, while current treatments are often associated with limited efficacy or unwanted side effects. Teva argues that ecopipam could address an important unmet need if approved.
The NDA submission follows Teva’s acquisition of the ecopipam programme and forms part of the company’s broader strategy to expand its innovative medicines portfolio. The FDA will now review the application and determine whether the treatment can proceed towards approval in the United States.
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