The Food and Drug Administration (FDA) has granted Fast Track designation to ecopipam (Emalex Biosciences), an investigational agent for the treatment of Tourette syndrome (TS).
Unlike other TS treatments, which act on dopamine D2 receptors, ecopipam works by selectively blocking the actions of the D1 receptor. This mechanism may potentially reduce the likelihood of metabolic and movement disorders that have been previously linked to D2 antagonist therapy, while still treating the repetitive and compulsive behaviors associated with the disorder.
Ecopipam is currently being evaluated in the phase 2 D1AMOND study in children and adolescents with TS (N=150). The Yale Global Tic Severity Scale will be used to evaluate the effectiveness of the treatment compared with placebo over a 12 week treatment period.
To date, the investigational oral treatment has been shown to be well tolerated in clinical trials; the most frequently reported adverse effects included sedation, insomnia, psychiatric changes, nausea, and vomiting.
“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 at 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.”
For more information visit emalexbiosciences.com.