Healthcare Industry News: U.S. Food and Drug Administration
News Release - August 23, 2017
Epygenix Therapeutics Receives US FDA Orphan Drug Designation for EPX-300 for the Treatment of Patients With Dravet SyndromeDravet Syndrome is a catastrophic form of epilepsy, affecting one in 15,700 children and adults in the United States
PARAMUS, N.J., Aug. 23, 2017 -- (Healthcare Sales & Marketing Network) -- Epygenix Therapeutics, Inc., a privately held biopharmaceutical company developing precision medicine for rare and catastrophic forms of genetic epilepsy, announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation for EPX-300 for the treatment of patients with Dravet Syndrome. Dravet Syndrome qualifies as 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. Patients with Dravet Syndrome also suffer with life-threatening seizures that cannot be adequately controlled by available medications. They also face a 15 to 20 percent mortality rate due to SUDEP (Sudden Unexplained Death in Epilepsy), which are commonly seizure-related accidents such as drowning or infections. In most cases, Dravet Syndrome is caused by heterozygous de novo mutations or gene deletions of SCN1A, a gene encoding a brain voltage-gated sodium channel (Nav1.1).
The effectiveness of EPX-300 was discovered using a proprietary phenotype-based zebrafish drug screening platform. The zebrafish harbors 82 percent of human disease-associated genes, and shares many physiological and metabolic pathways with humans. Using the 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.
"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," says Dr. Hahn-Jun Lee, M.Sc., Ph.D., president and CEO of Epygenix Therapeutics, Inc. "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."
Dr. Jules Mitchel, Ph.D., president at Target Health, Inc., in New York City, issued a statement: "Working together with Epygenix, the medical community, patients and their families, and the FDA, will allow for a rapid and efficient clinical development path to establish the efficacy and safety of EPX-300 for the treatment of Dravet Syndrome. Congratulations to Epygenix for taking this path where there is an unmet medical need."
Dr. Jack Parent, M.D., and William J Herdman, professor of neurology, co-director of the Comprehensive Epilepsy Center at the University of Michigan Medical School and one of the key scientific advisors of the company, said together that Epygenix has successfully made diversified pipelines to support Dravet Syndrome patients including non-responders to current treatment options. They further hope that EPX-300, together with EPX-100 and EPX-200, will be one of the important treatment options for Dravet Syndrome patients in a near future.
Under the U.S. Orphan Drug Act, the FDA's Office of Orphan Products Development provides sponsors with special status and incentives to facilitate the drug development for rare disease affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides seven years of market exclusivity if the drug candidate receives regulatory approval together with tax credits for qualified clinical trial cost, exemptions from certain FDA application fees, and assistance in clinical trial design.
To learn more about the company and their ongoing work, visit Epygenix.com.
About Epygenix Therapeutics, Inc.
Epygenix Therapeutics, Inc. is a precision medicine-based biopharmaceutical company focused on discovering and developing drugs to treat rare and intractable genetic epilepsy in childhood, such as Dravet Syndrome. Epygenix is currently focused on developing EPX-100, EPX-200 and EPX-300. These drug candidates abolish convulsive behavior and electrographic seizure activity and were discovered in a zebrafish Dravet Syndrome model which mimics the human pathology. For more information, please visit Epygenix.com.
EPX-300 is an 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.
Source: Epygenix Therapeutics
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