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Epygenix Therapeutics wins US FDA's orphan drug designation for EPX-300 for treating catastrophic form of epilepsy Dravet Syndrome.

M2 EQUITYBITES-August 24, 2017-Epygenix Therapeutics wins US FDA's orphan drug designation for EPX-300 for treating catastrophic form of epilepsy Dravet Syndrome


Biopharmaceutical company Epygenix Therapeutics Inc reported on Wednesday the receipt of the US Food and Drug Administration's orphan drug designation for EPX-300 for the treatment of patients with Dravet Syndrome, a rare pediatric disease under Section 529 of the Food, Drug, and Cosmetic Act.

Dravet Syndrome, a lifelong form of epilepsy, begins in the first year of life with frequent or prolonged seizures. Intellectual disability, behavioral abnormalities, gait and motor dysfunction and increased mortality are commonly observed as the disease progresses. This form affects one in 15,700 children and adults in the US.

According to the company, Dravet Syndrome is mostly caused by heterozygous de novo mutations or gene deletions of SCN1A, a gene encoding a brain voltage-gated sodium channel (Nav1.1).

The company said EPX-300 is an US FDA-approved drug for the treatment of major depression disorder which acts via modulation of 5HT signaling pathways. EPX-300 suppressed spontaneous convulsive behavior and electrographic seizures in zebrafish Dravet Syndrome models.

Utilising the proprietary phenotype-based zebrafish model for Dravet Syndrome, drug candidates were identified from a screen of more than 3,000 drugs that suppress seizures and symptoms associated with neurological diseases. The zebrafish harbors 82% of human disease-associated genes, and shares many physiological and metabolic pathways with humans, concluded the company.

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Publication:M2 EquityBites (EQB)
Date:Aug 24, 2017
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