Ecopipam

CHICAGO--(BUSINESS WIRE)--Emalex Biosciences today announced that the U.S. Food and Drug Administration has authorized the company’s Expanded Access Program (EAP) for ecopipam, an investigational therapy in development for the treatment of Tourette syndrome. “The FDA’s authorization of our Expanded Access Program marks a significant milestone as we advance toward NDA submission,” said Frederick Munschauer, MD, Emalex Biosciences chief medical officer. The program (ClinicalTrials.gov Identifier: NCT07093541) allows physicians to request access to ecopipam for eligible patients who have been treated with an FDA-approved treatment for Tourette syndrome (aripiprazole, haloperidol, or pimozide) or any other D2r antagonist and have experienced treatment failure, tolerability or safety issues or do not have access to any of these approved medications. Emalex plans to submit a New Drug Application (NDA) 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,” said Frederick Munschauer, MD, Emalex Biosciences chief medical officer. “This pathway offers a potential option for patients and their physicians while development continues.” Expanded Access Programs provide a regulatory pathway for patients with serious conditions to access investigational medicines outside of clinical trials. Tourette syndrome is a chronic, childhood-onset neurodevelopmental disorder characterized by motor and vocal tics. The condition is associated with increased mortality and significant morbidity. In the majority of individuals, Tourette syndrome substantially impacts day-to-day physical and social function. Ecopipam is an investigational, first-in-class small-molecule drug that selectively blocks dopamine-1 (D1) receptors. It is not an antipsychotic. Investigators are evaluating ecopipam as a potential therapy for Tourette syndrome. This mechanism represents a novel approach, distinct from currently approved therapies that primarily target dopamine-2 (D2) receptors. Participation in the Expanded Access Program requires physician oversight, FDA authorization, and Institutional Review Board approval. Enrollment is limited, and eligibility is determined on a case-by-case basis. Ecopipam is an investigational drug and has not been approved by the FDA for any indication. In clinical studies in pediatric patients with Tourette syndrome, ecopipam was generally well tolerated and the most common (≥ 5%) adverse events were headache, insomnia, fatigue, somnolence, anxiety, nausea, and restlessness. About Ecopipam Ecopipam is a first-in-class investigational compound that is being studied as a potential treatment for certain central nervous system (CNS) disorders. It blocks the actions of the neurotransmitter dopamine at the D1 receptor. Dopamine is a neurotransmitter in the central nervous system, and its receptors have been classified into two “families” based on their genetic structure: “D1” (including subtypes D1 and D5) and “D2” (including subtypes D2, D3, and D4). D1 receptor super-sensitivity may be a mechanism for the repetitive and compulsive behaviors associated with Tourette syndrome. Currently approved therapies for the treatment of Tourette syndrome act at D2 receptors. Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date and has received Orphan Drug and Fast Track designation from the FDA for the treatment of pediatric patients with Tourette syndrome. Adverse events affecting primarily the central nervous system have been reported in clinical trials conducted to date, including headache, fatigue, somnolence, insomnia, restlessness, anxiety, depression and rarely, suicidal ideation. About Emalex Biosciences Emalex Biosciences was created by Paragon Biosciences to develop new treatments for CNS disorders. Emalex is in late-stage development of a new class of drug for patients with Tourette syndrome and other conditions that have limited treatment options. Visit https://emalexbiosciences.com to learn more. About Paragon Biosciences Paragon Biosciences, founded by Jeff Aronin, is a global life science leader that creates, builds and funds innovative biology-based companies. Our portfolio companies accelerate scientific breakthroughs to solve some of society’s most challenging problems. Learn more at https://www.paragonbiosci.com.