Emalex Biosciences has received FDA authorisation for an Expanded Access Program (EAP) to provide its investigational therapy, ecopipam, to eligible patients with Tourette syndrome. The Chicago-based biotechnology company said the regulatory clearance represents a key step ahead of its planned New Drug Application (NDA) submission later this year.

The program (ClinicalTrials.gov Identifier: NCT07093541) enables physicians to request access to ecopipam for patients who have previously been treated with an FDA-approved Tourette therapy—such as aripiprazole, haloperidol, or pimozide—or another dopamine-2 receptor antagonist (D2rA) and experienced treatment failure or tolerability issues, or who lack access to approved options.

Frederick Munschauer, chief medical officer of Emalex Biosciences, said: “The FDA’s authorization of our Expanded Access Program marks a significant milestone as we advance toward NDA submission. This pathway offers a potential option for patients and their physicians while development continues.”

Expanded Access Programs allow patients with serious or life-altering conditions to receive investigational drugs outside of clinical trials when no satisfactory alternatives are available. Participation requires both FDA and Institutional Review Board approval, and eligibility is determined on a case-by-case basis.

Tourette syndrome is a chronic, childhood-onset neurodevelopmental disorder characterised by recurrent motor and vocal tics. It can cause substantial impairment in daily function and quality of life, with few effective or well-tolerated treatments available.

Ecopipam is an investigational, first-in-class small-molecule drug that selectively blocks dopamine-1 (D1) receptors, offering a novel mechanism distinct from currently approved antipsychotic therapies that act primarily on dopamine-2 receptors. The drug is not classified as an antipsychotic.

In clinical studies in paediatric patients with Tourette syndrome, ecopipam was generally well tolerated. The most common adverse events (≥5%) included headache, insomnia, fatigue, somnolence, anxiety, nausea, and restlessness.

Emalex said it plans to submit its NDA for ecopipam to the FDA in December 2025.