FDA Authorizes Emalex Biosciences’ Expanded Access Program for Ecopipam in Tourette Syndrome

Emalex Biosciences today announced that the U.S. Food and Drug Administration (FDA) has authorized the company’s Expanded Access Program (EAP) for ecopipam, an investigational therapy being developed for the treatment of Tourette syndrome (TS).
(ClinicalTrials.gov Identifier: NCT07093541)

The program allows physicians to request access to ecopipam for eligible patients who have previously been treated with FDA-approved therapies for Tourette syndrome — including aripiprazole, haloperidol, or pimozide — or any other D2 receptor antagonist, and who have experienced treatment failure, tolerability or safety issues, or lack access to these medications.

Emalex plans to submit a New Drug Application (NDA) for ecopipam to the FDA in late 2025.

“The FDA’s authorization of our Expanded Access Program marks a significant milestone as we advance toward NDA submission. This pathway offers a potential treatment option for patients and their physicians while development continues.”

• Frederick Munschauer, MD, Chief Medical Officer at Emalex Biosciences.

A New Mechanism of Action for Tourette Syndrome

Ecopipam is a first-in-class, investigational small molecule that selectively blocks dopamine-1 (D1) receptors, a novel mechanism distinct from current therapies that primarily target dopamine-2 (D2) receptors. Unlike traditional antipsychotics, ecopipam is not classified as an antipsychotic.

This D1 receptor-selective approach may address the repetitive and compulsive behaviors associated with Tourette syndrome, offering a new pathway for symptom management in a disorder that significantly affects physical, emotional, and social functioning.

About the Expanded Access Program

The FDA’s Expanded Access Program provides a regulatory pathway for patients with serious or life-threatening conditions to gain access to investigational therapies outside of clinical trials. Participation requires physician oversight, FDA authorization, and Institutional Review Board (IRB) approval. Enrollment is limited, with eligibility reviewed case by case.

In prior clinical studies involving pediatric patients with Tourette syndrome, ecopipam was generally well tolerated, with the most common adverse events (≥5%) including headache, insomnia, fatigue, somnolence, anxiety, nausea, and restlessness.

Ecopipam has received both Orphan Drug and Fast Track designations from the FDA for the treatment of pediatric Tourette syndrome.

About Emalex Biosciences

Emalex Biosciences, created by Paragon Biosciences, is a clinical-stage biopharmaceutical company focused on developing novel treatments for central nervous system (CNS) disorders with limited therapeutic options. The company is currently advancing ecopipam in late-stage development for Tourette syndrome.

About Paragon Biosciences

Paragon Biosciences, founded by Jeff Aronin, is a global life sciences leader that creates, builds, and funds innovative biology-based companies. Its portfolio companies accelerate scientific breakthroughs to address some of the world’s most challenging health conditions.

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