Marinus Pharmaceuticals Receives FDA Orphan Drug Designation for Ganaxolone

Marinus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its CNS-selective GABAA modulator, ganaxolone, for the treatment of Fragile X Syndrome (FXS).

“We are pleased to receive Orphan Drug Designation from the FDA for ganaxolone in Fragile X Syndrome,” stated Christopher M. Cashman, chief executive officer of Marinus Pharmaceuticals. “This designation underscores the significant unmet medical need for children suffering from a genetic mutation that causes autism-like symptoms including anxiety, mood swings and attention deficit.”

Orphan Drug Designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.