The US FDA has accepted Teva Pharmaceutical Industries' (NYSE: TEVA) New Drug Application for ecopipam (EBS-101), a selective dopamine D1 receptor antagonist, for the treatment of pediatric patients with Tourette syndrome, and granted Priority Review. The Prescription Drug User Fee Act (PDUFA) target action date is set for late Q1 2027. If approved, ecopipam would be the first new therapy indicated for pediatric Tourette syndrome in more than a decade and the first agent targeting the D1 receptor subtype to reach the market for this condition.
The three drugs currently holding FDA approval for tic suppression in Tourette syndrome — haloperidol (Haldol, approved 1969), pimozide (Orap, 1984), and aripiprazole (Abilify, 2014) — all act primarily via D2 receptor blockade and carry adverse effect profiles including weight gain, sedation, extrapyramidal symptoms, and, in rare cases, tardive dyskinesia. Teva said only 20–30% of patients remain on prescription therapy after one year, citing high discontinuation rates driven by tolerability. Ecopipam selectively blocks the D1 receptor family, a mechanism pharmacologically distinct from all currently approved options and one that produced no clinically meaningful changes in body weight, metabolic parameters, or drug-induced movement disorders across the Phase II and Phase III trials.
The NDA is supported by the D1AMOND Phase III randomized withdrawal trial, published in JAMA Neurology in May 2026, which enrolled 216 participants across 77 sites in North America and Europe. Of these, 104 participants — 90 pediatric — were randomized following an open-label stabilization period. Pediatric responders continuing ecopipam had a 53% reduced risk of relapse compared with placebo over 12 weeks, with a reported p-value of 0.008 on the primary endpoint of time to relapse, measured using the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS). The filing also incorporates Phase IIb data from the D1AMOND Phase IIb trial, which demonstrated statistically significant improvement in YGTSS-TTS versus placebo at Week 12 (p = 0.01) in 153 pediatric participants, and a subsequent 12-month open-label extension. The most commonly reported adverse events across trials were headache, insomnia, fatigue, somnolence, anxiety, nausea, and restlessness. The accepted NDA seeks approval exclusively for pediatric patients, though the Phase III trial also enrolled adults.