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Epygenix gets FDA orphan drug status for EPX-300 to treat Dravet Syndrome

Published 24 August 2017

Epygenix Therapeutics has secured orphan drug status from the US Food and Drug Administration (FDA) for its EPX-300 to treat patients with Dravet Syndrome.

Dravet Syndrome is a rare pediatric disease and lifelong form of epilepsy, which starts in the first year of life with frequent or prolonged seizures.

Currently, the company is engaged in the development of EPX-100, EPX-200 and EPX-300 drug candidates, which abolish convulsive behavior and electrographic seizure activity.

EPX-300 is an FDA-approved antidepressant, anti-anxiety and sleep-inducing insomnia drug, which acts through modulation of serotonin (5HT) signaling pathways.

The drug holds capacity to suppress pontaneous convulsive behavior and electrographic seizures in zebrafish disease models for Dravet Syndrome.

Through using an advanced phenotype-based zebrafish drug screening platform, the company has discovered the effectiveness of EPX-300.

The zebrafish is said to harbor 82% of human disease-associated genes and shares multiple physiological and metabolic pathways with humans.

Drug candidates have been identified from a screen of around 3,000 drugs by using the zebrafish model for Dravet Syndrome.

According to the company, patients with Dravet Syndrome also suffer with life-threatening seizures that cannot be adequately controlled by available medications.

Epygenix Therapeutics president and CEO Dr Hahn-Jun Lee said: "By receiving orphan drug designation for EPX-300, now all of our drug candidates including EPX-100 and EPX-200, have been granted orphan drug status for Dravet Syndrome from the FDA.

"As a result, we are now able to establish a diversified pipeline to support patients with Dravet Syndrome, and are now preparing a clinical testing of our drug candidates for patients as early as possible." 

Image: The FDA campus in New Hampshire Ave, Silver Spring, Maryland. Photo: courtesy of The U.S. Food and Drug Administration.