Marinus Pharma Receives FDA Orphan Drug Designation for Ganaxolone to Treat Fragile X Syndrome

Drug Research

Marinus Pharmaceuticals Inc , a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders announced that the U.S. 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.

About Fragile X Syndrome
Fragile X syndrome (FXS) is the most common genetic cause of autism and caused by a mutation in the FMR1 gene.  FXS is characterized by a range of developmental problems and symptoms, including cognitive impairment, learning disabilities and behavioral challenges.  Approximately one million individuals in the United States have, or are at risk for developing a Fragile X associated disorder, with approximately 100,000 people having FXS.  According to the Centers for Disease Control and Prevention, FXS affects 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females of all races and ethnic groups. Patients with FXS exhibit autism-like symptoms including anxiety and mood swings, attention deficit and heightened stimuli. Approximately 7% of women and 18% of men with FXS have seizures. Currently, there are no known cures or approved therapies for FXS.

About Ganaxolone
Ganaxolone is a CNS-selective GABAA modulator being developed in three different dose forms (IV, capsule, and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Ganaxolone acts on a well-characterized synaptic and extrasynaptic GABAA target known for anti-seizure and anti-anxiety activity.  Ganaxolone has been studied in more than 1,400 subjects, both pediatric and adult, at therapeutically relevant dose levels and treatment regimens for up to two years. In these studies, ganaxolone was generally safe and well tolerated, with the most commonly reported adverse events of somnolence, dizziness and fatigue.

About Marinus Pharmaceuticals
Marinus Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety and convenient dosing, to improve the lives of patients suffering from epilepsy and neuropsychiatric disorders. Ganaxolone is a CNS-selective GABAA modulator that acts on a well-characterized target in the brain known to have both anti-seizure and anti-anxiety effects. Ganaxolone is being developed in three different dose forms (IV, capsule and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Marinus is currently evaluating ganaxolone in orphan pediatric indications for the treatment of genetic seizure and behavior disorders, and preparing to initiate Phase 2 studies in status epilepticus, an orphan indication, and postpartum depression. For more information visit


Lisa M. Caperelli
Senior Director, Investor Relations & Corporate Communications
Marinus Pharmaceuticals, Inc.
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