Teva Pharmaceutical has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) seeking approval of ecopipam for paediatric Tourette syndrome.
If approved, ecopipam could become the first new FDA-approved treatment for paediatric Tourette syndrome in more than a decade.
Tourette syndrome is a neurodevelopmental disorder characterised by involuntary motor and vocal tics that typically begin in childhood. Although treatment options are available, many patients continue to experience inadequate symptom control or treatment-limiting side effects.
The submission is supported by phase 3 data recently published in JAMA Neurology. The study found that ecopipam significantly delayed time to relapse compared with placebo in children and adolescents who had responded to treatment during an earlier open-label phase.
The trial met its primary endpoint, demonstrating a statistically significant benefit for ecopipam over placebo.
Eric Hughes, Executive Vice President, Global R&D and Chief Medical Officer at Teva, said: “The NDA submission for ecopipam is a significant milestone for a potential first-in-class treatment option in paediatric Tourette syndrome.”
Ecopipam is a selective dopamine D1 receptor antagonist and has received both Orphan Drug and Fast Track designations from the FDA.
The therapy was generally well tolerated in the phase 3 study. The most commonly reported adverse events included somnolence, anxiety, headache, insomnia and fatigue.
Teva said the application represents an important step towards expanding treatment options for children and adolescents living with Tourette syndrome, where unmet clinical need remains significant despite currently available therapies.