Orphan Drug Designates EPX-200 For Treatment Of Lennox-Gastaut Syndrome

Release Date: 26-Sep-2021

US FDA has granted orphan drug designation to EPX-200 drug for the treatment in Lennox-Gastaut Syndrome. It is a rare epileptic encephalopathy presenting in childhood with intractable seizures. Around 48,000 children and adults in the U.S are currently suffered with LGS. EPX-200 is a weight gain management drug which acts via modulation of serotonin (5HT) signaling pathways. EPX-200 was firstly identified using a zebrafish disease model for Dravet Syndrome. It also has been reported to have anti-seizure effects based on the result from small number of patients.


The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.


A company named Epygenix Therapeutics, Inc. is a clinical stage biopharmaceutical company which is currently focusing upon developing EPX-100, -200, and -300.

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