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Press Release

Epygenix Therapeutics Receives Orphan Medicinal Product Designation in Europe With EPX-200 for the Treatment of Patients With Dravet Syndrome

PARAMUS, N.J., Aug. 2, 2021 - Epygenix Therapeutics, Inc., a clinical stage biopharmaceutical company focused on rare and intractable genetic epilepsy announced today that the European Medicines Agency (EMA) granted Orphan Medicinal Product Designation to EPX-200 for the treatment of patients with Dravet syndrome (DS) based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA).

Dravet Syndrome (DS) is a rare, genetic epilepsy that results in severe seizures that fail to respond well to currently available medications. DS usually starts in the first year of life and progresses to include intellectual disability, behavioral abnormalities, gait and motor dysfunction, and increased mortality. Patients also suffer with life-threatening medically intractable seizures that may increase the risk of SUDEP (Sudden Unexplained Death in Epilepsy), seizure-related accidents such as drowning, or infections. Mutations in a sodium channel are the most common genetic cause of DS and reports suggest that 1 in 15,000 to 20,000 people suffer with this disease.

Dr. Hahn-Jun Lee, M.Sc., Ph.D., President and CEO of Epygenix Therapeutics, Inc. stated that the EMA's Orphan Medicinal Product Designation to EPX-200 is very important, as EMA recognizes the significant potential benefit of EPX-200 for DS treatment. We thank Premier Research for a regulatory agent of this progress. EPX-200 was first identified using a zebrafish DS model and high-throughput screening system in the lab of Prof. Baraban at the University of California, San Francisco (UCSF). The team and others subsequently confirmed it efficacy in a small group of DS patients. Epygenix' exciting journey from aquarium-to-bedside continues with diversified drug candidates poised to move forward into clinical trials.

Dr. Scott C. Baraban, Ph.D., Professor & William K. Bowes Jr. Endowed Chair in Neuroscience Research at UCSF and Chair of the Scientific Advisory Board at Epygenix Therapeutics, Inc. added that "it is an exciting time for the Dravet Syndrome community to see these zebrafish-based drug discoveries move one step closer to clinical trials."

Alex Yang, J.D., LLM, President and CEO of Mstone Partners Hong Kong and Chair of the Board at Epygenix Therapeutics also stated that "We feel quite strong about our multiple drug candidates which will be expanded to cover many forms of refractory epilepsies including Dravet. The Orphan Medicinal Product Designation from EMA for EPX-200 brings us one step closer to such position to bring our drugs to patients in Europe more efficiently."

About Epygenix Therapeutics, Inc.

Epygenix Therapeutics, Inc. is a clinical stage biopharmaceutical company focused on developing drugs to treat rare and intractable genetic epilepsy. Epygenix is currently focused on developing EPX-100, -200, and -300. These candidates abolish convulsive behavior and electrographic seizure activity and were discovered in a zebrafish Dravet Syndrome model which mimics the human pathology and confirmed its validity by the human efficacy with EPX-200 and -300. For more information, please visit

About EPX-200

EPX-200 is a weight gain management drug that acts via modulation of serotonin (5HT) signaling pathways. EPX-200 was firstly identified in a zebrafish disease model for Dravet Syndrome. EPX-200 has already been successfully tested in five Dravet Syndrome patients at the University of Colorado Hospital, where it showed an ability to reduce seizure frequency in a small compassionate use trial.

Media Contact

Hahn-Jun Lee, M.Sc., Ph.D.


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