Emalex Biosciences has received authorization from the U.S. Food and Drug Administration (FDA) to launch an Expanded Access Program (EAP) for ecopipam, an investigational, first-in-class, non-antipsychotic therapy being developed for the treatment of Tourette syndrome.

This program, identified as ClinicalTrials.gov Identifier: NCT07093541, creates a vital regulatory pathway that allows patients with this chronic, childhood-onset neurodevelopmental disorder—which is characterized by debilitating motor and vocal tics—to potentially access ecopipam outside of formal clinical trials. T

he EAP is specifically for eligible patients who have failed, had tolerability issues with, or lack access to currently FDA-approved D2 receptor antagonist treatments like aripiprazole, haloperidol, or pimozide.

Ecopipam's mechanism of action is unique, as it selectively blocks dopamine-1 (D1) receptors, a novel approach distinct from all currently approved Tourette syndrome therapies that primarily target D2 receptors. This distinction is significant because D1 receptor supersensitivity is theorized to be a key mechanism driving the repetitive behaviors seen in Tourette syndrome.

While Emalex continues its late-stage development and plans to submit a New Drug Application (NDA) to the FDA in late 2025, the EAP offers a crucial potential option for patients with high unmet needs. Participation in the program is limited, requires approval from the patient's physician, the FDA, and an Institutional Review Board, and is determined on a case-by-case basis. In clinical studies to date, ecopipam has generally been well tolerated, with the most common adverse events including headache, insomnia, fatigue, and anxiety.