Drugmaker Emalex Biosciences has announced positive results from a Phase III clinical trial of ecopipam, the first drug designed to treat children with Tourette's.
The findings, published in JAMA Neurology, showed that continued use of ecopipam can cut the risk of relapse in half for children battling tics caused by Tourette syndrome.
The research was led by Cincinnati Children's Hospital and had Dr Donald Gilbert, a movement disorders expert with the Division of Neurology at Cincinnati Children's, as principal investigator for the 77-site clinical trial.
"I hope this will be the first drug approved specifically for Tourette syndrome in the US," Gilbert says.
There are three other drugs that are approved by the US FDA, but they are all antipsychotics that were initially approved for treating schizophrenia. This drug works in the brain in a totally different way.
Ecopipam is a first-in-class, investigational, oral medication designed to treat central nervous system disorders by selectively blocking dopamine at the D1 and D5 receptors.
Current FDA-approved therapies for Tourette syndrome work by blocking dopamine at the D2 receptor and often come with significant side effects.
Emalex said that ecopipam represents an alternative approach to these current therapies, aiming to reduce motor and vocal tics without so many of the severe side effects associated with standard D2 antipsychotics.
Emalex Bioscience said in its statement that it plans to seek approval from the US Food and Drug Administration (FDA) to take the new medicine to market.