CHICAGO--(BUSINESS WIRE)--Emalex Biosciences, Inc. (Emalex), a biopharmaceutical company developing innovative therapies for patients with neurological and psychiatric conditions, announced today that it has received Fast Track designation for its investigational product, ecopipam, from the U.S. Food and Drug Administration (FDA) for the treatment of patients with Tourette Syndrome (TS).
Fast Track designation is received when a potential new therapy is intended to treat a serious condition and data demonstrate the potential to address an unmet medical need. This designation signals FDA’s interest in the development program for ecopipam for the treatment of pediatric patients with TS, the ability to request a rolling new drug application (NDA) submission, as well as the potential for expedited NDA review.
“We are encouraged that the FDA has granted Fast Track designation to ecopipam for the treatment of patients with TS, a serious disease with significant morbidity,” said Atul Mahableshwarkar, MD, Senior Vice President of Drug Development at Emalex. “The unmet medical need for patients with TS is significant and we look forward to working with the FDA throughout the development process for this novel compound.”
Ecopipam selectively blocks the actions of dopamine at the D1 receptor. Previous Phase 2 studies in both adult and pediatric patients with TS have suggested potential activity in reducing the frequency and severity of motor and vocal tics associated with TS. Earlier this month, Emalex announced that the company had initiated enrollment into a Phase 2b double-blind clinical study of ecopipam in children and adolescent patients (aged >6 to <18 years) with TS at multiple study centers in the United States, Canada, and the European Union. Additional information about the clinical trial, including eligibility criteria and the location of clinical trial sites, can be found at https://clinicaltrials.gov/ct2/show/NCT04007991.
Ecopipam is an investigational first-in-class drug being evaluated in pediatric patients for the treatment of Tourette Syndrome (TS) and for childhood onset fluency disorder (stuttering) in adults. Ecopipam selectively 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 TS. Currently approved therapies for the treatment of TS act at D2 dopamine receptors.
Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date, including in adult and pediatric patients with TS. Adverse events affecting primarily the central nervous system [CNS] (e.g., sedation, insomnia, psychiatric changes) and the gastrointestinal system (e.g., nausea and vomiting) are the most frequently reported side effects.
About Tourette Syndrome
Tourette Syndrome (TS) is a neurological disorder characterized by motor or vocal tics that begins during childhood and is not caused by medications, other medical reasons, or a confirmed neurological abnormality. Motor symptoms may include eye-blinking, facial grimacing, mouth movements, head jerks, shoulder shrugs, and arm/leg jerks. Vocal symptoms may include fast, meaningless sounds or noises such as sniffing, throat clearing, grunting, barks, and squeals. TS is a complicated disease that is often difficult to diagnose due to commonly associated co-morbid complications such as attention deficit/hyperactivity disorder (ADHD), obsessive-compulsive disorder (OCD), anxiety, and depression.
About Emalex Biosciences, Inc
Emalex Biosciences is a biopharmaceutical company dedicated to the development of new and novel treatments for patients with central nervous system disorders who have limited or no treatment options. Emalex is headquartered in Chicago, Illinois. For more information, visit: EmalexBiosciences.com.