A new mechanism for Tourette syndrome nears an FDA decision
Treatment for children with tics has long relied on behavioural therapy and antipsychotics. Teva's ecopipam, under priority review at the FDA, would add a different mechanism, on evidence that needs careful reading.
In brief
Teva's ecopipam, a D1 receptor antagonist under FDA priority review, could change Tourette care in children. What the evidence shows and where it stops.
- The FDA accepted Teva's ecopipam application with priority review, with a target date late in the first quarter of 2027.
- In the phase 3 withdrawal trial, paediatric responders staying on ecopipam had a 53% lower risk of relapse than those tapered to placebo.
- The longest-term data come from an open-label extension, with no control group.
Tourette syndrome usually declares itself in early school years, with tics typically emerging between the ages of five and ten. NeurologyLive, citing the US Centers for Disease Control and Prevention, puts the US figure at roughly one in 333 children aged three to 17, or about 174,000.
Few medicines are labelled for them. A 2020 review in the Journal of Pediatric Pharmacology and Therapeutics counted three FDA-approved drugs for tics: haloperidol, pimozide and aripiprazole, all antipsychotics. Teva's ecopipam, a dopamine D1 receptor antagonist, could become the fourth. The company announced on 19 August 2026 that the FDA had accepted its application for paediatric patients with priority review, and the target action date falls late in the first quarter of 2027.
Current treatment
The American Academy of Neurology's 2019 guideline gave its strongest support to Comprehensive Behavioral Intervention for Tics (CBIT).
The guideline found moderate confidence that haloperidol, risperidone, aripiprazole (in children), tiapride (in children), clonidine and onabotulinumtoxinA reduce tic severity against placebo. Antipsychotics carry known costs: movement disorders, weight gain and metabolic effects. The 2020 review also noted a risk of tardive dyskinesia, potentially irreversible, with haloperidol and pimozide, and QTc prolongation with pimozide.
What the ecopipam trials show
The phase 2b D1AMOND trial randomised 153 paediatric patients across 68 sites for 12 weeks, and ecopipam reduced tic severity significantly more than placebo on the Yale Global Tic Severity Scale. The phase 3 study, published in JAMA Neurology this year, used a withdrawal design. Of 216 paediatric and adult participants in an open-label stabilisation phase, 104 responders (90 of them paediatric) were randomised to continue ecopipam or taper to placebo.
Paediatric responders who stayed on the drug had a 53% lower risk of relapse (hazard ratio 0.47; 95% CI, 0.26 to 0.84; P = .008). Median time to relapse on placebo was four weeks. The commonest adverse events across the study were somnolence (11.1%), anxiety (9.7%) and headache (9.7%).
A withdrawal trial asks whether responders keep benefiting. It says less about how many patients respond in the first place.
Reading the newer data
At the International Congress of Parkinson's Disease and Movement Disorders in Seoul in October, Teva presented a pooled post hoc analysis of 292 patients, in which 69.8% reached at least a 25% improvement in tic score within eight weeks. An interim analysis of a 36-month open-label extension covered 118 children, adolescents and adults with a median exposure of 14.9 months. It reported a mean tic reduction of 45.6% through 18 months and no new safety signals.
Neither result has a control group. The trade publication Pharmacally noted that the company's release gave no 18-month denominator, confidence intervals or baseline scores. Those gaps should narrow when the FDA's review documents and full publications appear.
What approval would mean
Teva says ecopipam would be the first new Tourette therapy in more than a decade and the first with a novel mechanism in over 50 years. The drug also has orphan designation for paediatric Tourette syndrome. The main questions now are the age range and warnings on any label, and whether prescribers see a real alternative to antipsychotics once long-term safety data mature.
