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Emalex Biosciences is working towards getting approval for its new treatment for Tourette syndrome, called ecopipam, after successful results in a late-stage clinical trial. The drug, which targets a specific brain receptor, showed promising results in both children and adults with Tourette syndrome. This condition can cause involuntary tics and sounds. In the trial, only 41.9% of children and 41.2% of adults taking ecopipam experienced relapse, compared to 68.1% and 67.9% in the placebo groups, respectively.
Following these positive results, Emalex plans to discuss the drug’s approval with health authorities, including the FDA, in 2025. The study involved 167 children and 49 adults and showed a significant reduction in tics among those taking the drug during a 12-week period.
According to Emalex’s chief medical officer, these findings increase confidence in ecopipam as a potential new treatment for Tourette syndrome. The global market for Tourette therapies was valued at $248 million in 2024, but it’s expected to decrease significantly by 2030. Currently, the most commonly used drug for Tourette syndrome is Abilify (aripiprazole), which is anticipated to earn around $192 million by 2025.
The CDC reports that around 0.3% of children in the US have Tourette syndrome, with boys more likely to be affected than girls. In earlier trials, ecopipam reduced tics in children by 30% compared to a placebo.
Emalex’s CEO expressed hope that ecopipam will offer relief for those with Tourette syndrome, noting the limited treatment options currently available. Meanwhile, another company, SciSparc, has received FDA approval to start trials for its own Tourette treatment in adults.
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