Journal of Child Neurology Publishes Review Highlighting Dopamine’s Role in Tourette Syndrome and Ecopipam’s Selective D1 Receptor Mechanism for Tourette Syndrome in Pediatric Patients

The accepted drug application seeks an exclusively pediatric indication, although the Phase 3 trial also included adults.

Rhea-AI Summary

Teva (TEVA) announced a Journal of Child Neurology review examining ecopipam’s potential to treat Tourette syndrome in pediatric patients. Ecopipam is an investigational drug that selectively blocks dopamine D1 receptors. In Phase 3, continued treatment reduced pediatric participants’ risk of tic relapse by 50% versus placebo (HR: 0.5; P=0.008). Phase 2b and 12-month open-label data showed sustained reductions in tic severity.

Common adverse events included somnolence, headache, insomnia, fatigue and anxiety. Across trials, ecopipam was not associated with clinically relevant weight gain, adverse metabolic effects or drug-induced movement disorders. The FDA granted Priority Review and Orphan Drug designation for pediatric Tourette syndrome.

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0 major · 1 point

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Positive

• Major pointPhase 3 continued treatment reduced pediatric tic-relapse risk by 50% versus placebo (HR: 0.5; P=0.008).
• Moderate pointFDA Priority Review granted for ecopipam for pediatric Tourette syndrome.
• Minor pointPhase 2b and 12-month open-label data showed sustained reductions in tic severity.
• Minor pointTrial safety findings showed no association with clinically relevant weight gain, adverse metabolic effects or drug-induced movement disorders.
• Minor pointFDA Orphan Drug designation granted for ecopipam for pediatric Tourette syndrome.

Negative

• Minor pointCommon adverse events included somnolence, headache, insomnia, fatigue and anxiety across clinical trials.

News Explained

Teva reports FDA acceptance of ecopipam’s NDA, while the drug remains investigational and the requested indication is limited to pediatric Tourette syndrome.

Key Figures

Phase 3 trial participants: 216 participants, including 167 pediatric Tic relapse risk reduction: 50% Hazard ratio: 0.5 +3 more

Phase 3 trial participants

216 participants, including 167 pediatric

Ecopipam trial

Tic relapse risk reduction

50%

Continued ecopipam versus placebo in pediatric participants

Hazard ratio

0.5

Tic relapse in pediatric participants

P-value

P=0.008

Phase 3 tic relapse result

D2-antagonist treatment discontinuation

61.2%

By 3 months

D2-antagonist treatment discontinuation

82.1%

By 18 months

Key Terms

D1 receptor antagonist, D2 receptor antagonists, priority review, orphan drug designation, +1 more

5 terms

D1 receptor antagonist medical

"selective D1 (dopamine) receptor antagonist"

A D1 receptor antagonist is a drug or compound that binds to and blocks dopamine D1-family receptors on nerve cells, preventing dopamine from activating those receptors and thereby reducing D1-mediated cellular signaling. Such antagonists can be selective for D1 receptors or less selective and also affect other dopamine receptors; blocking D1 signaling alters processes that D1 receptors normally influence (for example certain aspects of movement, attention, and reward-related signaling) depending on where in the brain or body the receptors are blocked.

D2 receptor antagonists medical

"newly treated with D2 receptor antagonists"

Drugs that bind to and block dopamine D2 receptors, preventing dopamine from activating those receptors and thereby reducing dopamine signaling in the brain and nervous system; this mechanism underlies their use to reduce symptoms linked to excess dopamine activity (for example in psychosis or nausea) and also causes predictable effects such as increased prolactin levels and a risk of movement-related side effects when D2 blockade occurs in motor-control pathways.

priority review regulatory

"granted Priority Review by the FDA"

Priority review is a regulatory fast-track that shortens the time an agency spends evaluating a drug, vaccine or medical device application so a decision comes sooner than normal. For investors, it matters because a faster review is like an express lane to market: it can speed revenue potential and reduce regulatory uncertainty, but it does not guarantee approval and still requires the product to meet safety and effectiveness standards.

orphan drug designation regulatory

"with Orphan Drug designation"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

randomized withdrawal technical

"double-blind, placebo-controlled, randomized withdrawal trial"

A randomized withdrawal is a clinical trial design where people who initially get better on a treatment are later randomly assigned either to keep taking it or to stop, so researchers can measure whether benefits last and whether problems reappear. Think of testing a new routine by removing it for half the group to see if results continue. Investors care because these results reveal how durable a drug’s effects and safety profile are, which affect approval, labeling, prescribing and long-term sales.

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• The published review examines dopamine’s role in tic generation and how approved and investigational drugs may affect dopamine to reduce tics as well as synthesizes clinical trial evidence that ecopipam reduces tic severity, provides sustained benefit over time and helps prevent relapse without some of the complications associated with antipsychotic agents.¹
• Ecopipam is an investigational, selective D1 (dopamine) receptor antagonist targeting dopaminergic signaling in the brain’s direct motor pathway implicated in Tourette syndrome.
• Unlike D2 antipsychotics that target D2 receptors in the indirect pathway, ecopipam selectively targets D1 receptors in the direct motor pathway, offering a differentiated approach that may reduce tic severity while potentially avoiding common adverse events associated with D2-targeting therapies.¹
  
  

PARSIPPANY, N.J., Oct. 02, 2026 (GLOBE NEWSWIRE) -- Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), today announced that the Journal of Child Neurology published a comprehensive review examining dopamine pathways in Tourette syndrome and the therapeutic potential of ecopipam, a first-in-class investigational selective D1 (dopamine) receptor antagonist for the treatment of pediatric patients with Tourette syndrome.^1,2 The review evaluates current treatment limitations and details how selectively targeting D1 receptors in the brain’s direct motor pathway may offer a differentiated, non-antipsychotic approach for managing tics in pediatric patients.¹

“Tourette syndrome affects up to 1 in 130 children globally and can have a profound impact on a child's daily life, education and social development,” said Eric Hughes, M.D., Ph.D., Executive Vice President, Global R&D and Chief Medical Officer at Teva. “The review published in the Journal of Child Neurology reinforces the potential of ecopipam’s selective dopamine D1 receptor approach. This is the kind of innovative programs we are pursuing at Teva—advancing differentiated science to address meaningful unmet needs.”

The comprehensive review details the neurobiological rationale for D1 receptor antagonism compared with existing therapies. In Tourette syndrome, elevated dopaminergic activity in the basal ganglia is proposed to contribute to the expression of involuntary tics. Because D1 receptors are primarily expressed by neurons in the direct motor pathway, ecopipam is hypothesized to block excess dopaminergic activity in this pathway, thereby normalizing downstream basal ganglia signaling and movement patterns. In contrast, traditional antipsychotics target D2 receptors, which are primarily associated with the indirect pathway.

This mechanistic distinction is supported by clinical data synthesized across the ecopipam development program. In a Phase 3 trial (n=216, including 167 pediatric), continued ecopipam treatment cut the risk of tic relapse by 50% versus placebo in pediatric participants (HR: 0.5; P=0.008), building on Phase 2b and 12-month open-label data showing sustained reductions in tic severity. Across clinical trials, the most common adverse events included somnolence, headache, insomnia, fatigue and anxiety while ecopipam was not associated with clinically relevant weight gain, adverse metabolic effects or drug-induced movement disorders. The potential importance of this efficacy and tolerability profile is underscored by real-world treatment persistence data, which show that treatment discontinuation rates among patients newly treated with D2 receptor antagonists were 61.2% by 3 months and 82.1% by 18 months.¹

“Treatment of Tourette syndrome can be complex, as clinicians must balance tic improvement with the side effect profiles of current therapies,” said Donald L. Gilbert, M.D., M.S., senior author of the review and Pediatric Movement Disorders and Tourette Syndrome Specialist, Division of Neurology, Cincinnati Children’s Hospital Medical Center. “Ecopipam selectively targets the dopamine D1 receptor, and the data reviewed in this publication – demonstrating reduced tic severity and sustained benefit without significant weight gain, metabolic complications or movement disorders – supports continued research into D1 receptor antagonism for Tourette syndrome.”

For more information on the review published online in the Journal of Child Neurology, click here.

About Tourette Syndrome 
Tourette syndrome is a chronic neuro-developmental disorder characterized by involuntary motor and vocal tics beginning in childhood, often between 5 and 10 years of age.³ For people living with Tourette syndrome, symptoms can be frequent, visible and disruptive, affecting everyday life.³

About Ecopipam and Its Clinical Program 
Ecopipam is a first-in-class investigational therapy designed to block dopamine signaling at the D1 receptor. D1 receptor hypersensitivity may contribute to repetitive and compulsive behaviors associated with Tourette syndrome. 

Ecopipam was granted Priority Review by the FDA with Orphan Drug designation for the treatment of pediatric patients with Tourette syndrome. Orphan Drug designation is reserved for patient populations of 200,000 or fewer. 

The D1AMOND Phase 2b Trial was a 12-week randomized, double-blind, placebo-controlled trial that studied 153 pediatric participants across 68 sites in North America and Europe. The primary efficacy endpoint was the change in the YGTSS-TTS, i.e., sum of the motor and vocal tic scores, from baseline to end of therapy.⁴ The associated Phase 2b open-label extension enrolled 121 pediatric subjects from the Phase 2b trial and followed them for up to 12 months’ duration to evaluate the long-term safety and tolerability of ecopipam.⁵ The subsequent D1AMOND Phase 3 Trial was a double-blind, placebo-controlled, randomized withdrawal trial enrolling a total of 216 pediatric and adult participants into an open-label stabilization period and randomizing 104 participants (90 pediatric, 14 adult) across 77 sites in North America and Europe. The objective of this study was to evaluate the maintenance of efficacy of ecopipam in pediatric and adult responders utilizing the YGTSS-TTS change from randomization or increased Tourette-specific care to determine relapse.⁶ While this Phase 3 trial included adult participants, the accepted NDA and resulting indication sought by Teva are exclusively for pediatric patients. 

About Teva
Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) is transforming into a leading innovative biopharmaceutical company, enabled by a world-class generics business. For over 120 years, Teva’s commitment to bettering health has never wavered. From innovating in the fields of neuroscience and immunology to providing complex generic medicines, biosimilars and pharmacy brands worldwide, Teva is dedicated to addressing patients’ needs, now and in the future. At Teva, We Are All In For Better Health. To learn more about how, visit www.tevapharm.com. 

Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are based on management’s current beliefs and expectations and are subject to substantial risks and uncertainties, both known and unknown, that could cause Teva’s future results, performance or achievements to differ significantly from that expressed or implied by such forward-looking statements. All statements other than statements of historical fact are, or may be deemed to be, forward-looking statements. In some cases, you can identify these forward-looking statements by the use of words such as “should,” “expect,” “anticipate,” “developing,” “target,” “may,” “expand,” “intend,” “plan,” “believe” and other words and terms of similar meaning and expression in connection with any discussion of future performance. Important factors that could cause or contribute to such differences include risks and uncertainties relating to: our ability to successfully develop, obtain regulatory approval for and commercialize ecopipam; our ability to successfully compete in the marketplace including our ability to develop and commercialize ecopipam for the treatment of pediatric patients with Tourette syndrome; our ability to successfully execute on our Pivot to Growth strategy, including to expand our innovative and biosimilar medicines pipeline and to profitably commercialize our innovative medicines and biosimilar portfolio, whether organically or through business development, and to execute on our organizational transformation and to achieve expected cost savings; our significant indebtedness, which may limit our ability to incur additional indebtedness, engage in additional transactions or make new investments; and other factors discussed in this press release, in our Quarterly Report on Form 10-Q for the second quarter of 2026 and in our Annual Report on Form 10-K for the year ended December 31, 2025, including in the sections captioned “Risk Factors” and “Cautionary Note Regarding Forward Looking Statements.” Forward-looking statements speak only as of the date on which they are made, and we assume no obligation to update or revise any forward-looking statements or other information contained herein, whether as a result of new information, future events or otherwise. You are cautioned not to put undue reliance on these forward-looking statements.

References:

1. Karkanias, G. B., Flatt, J. A., Korb, R. T., Munschauer, F. E., Wanaski, S. P., Cunniff, T. M., & Gilbert, D. L. (2026). Targeting Dopamine Pathways for the Treatment of Tourette Syndrome. Journal of Child Neurology. https://doi.org/10.1177/08830738261491159
2. U.S. Food and Drug Administration (FDA) Approval Records: Haloperidol (1969), Pimozide (1984), Aripiprazole (2014); Pringsheim, T., et al. (2019). The pharmacological management of tic disorders: an updated practice guideline. Neurology. 
3. CDC | Tourette Syndrome | Data and Statistics on Tourette Syndrome, 2024; Mayo Clinic | Tourette Syndrome – Diagnosis and treatment, 2025. 
4. Gilbert DL, Dubow JS, Cunniff TM, et al. Ecopipam for Tourette Syndrome: A Randomized Trial. Pediatrics. 2023;151(2):e2022059574. doi:10.1542/peds.2022-059574 
5. Gilbert DL, Kim DJB, Miller MM, et al. Safety and Effect of 12-Month Ecopipam Treatment in Pediatric Patients with Tourette Syndrome. Mov Disord Clin Pract. 2025;12(8):1157-1166. doi:10.1002/mdc3.70091. 
6. Gilbert DL, Atkinson SD, Kim DJB, et al. Efficacy and Safety of Ecopipam for Tourette Syndrome: A Phase 3 Randomized Clinical Trial. JAMA Neurol. 2026;83(7):645–653. doi:10.1001/jamaneurol.2026.1431
   
   

Teva Media Inquiries:
tevacommunicationsnorthamerica@tevapharm.com

Teva Investor Relations Inquiries:
TevaIR@tevapharm.com

FAQ

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What did the ecopipam Phase 3 trial show for Teva’s pediatric Tourette syndrome treatment?

Continued ecopipam treatment reduced tic-relapse risk by 50% versus placebo in pediatric participants (HR: 0.5; P=0.008). The review also synthesized Phase 2b and 12-month open-label evidence showing sustained reductions in tic severity.

How was Teva’s ecopipam Phase 3 trial designed?

The Phase 3 trial used a double-blind, placebo-controlled, randomized withdrawal design to evaluate maintenance of efficacy in responders. It enrolled 216 pediatric and adult participants into open-label stabilization and randomized 104 participants, including 90 pediatric and 14 adult participants. Relapse was determined using changes in combined motor and vocal tic scores or increased Tourette-specific care.

Does Teva’s ecopipam application seek approval for adults with Tourette syndrome?

No. The accepted new drug application and the indication Teva seeks are exclusively for pediatric patients. Adult participants were included in the Phase 3 trial, but the requested indication does not include adults.