Ganaxolone is being developed in three different dose forms that include IV, capsule, and liquid, aimed at maximizing therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings.
It has been evaluated in over 1,400 pediatric and adult subjects, at therapeutically appropriate dose levels and treatment regimens lasting nearly two years.
In the studies, ganaxolone was generally safe and well tolerated while showing most common adverse reactions such as somnolence, fatigue and dizziness.
Marinus Pharmaceuticals CEO Christopher Cashman said: “We are pleased to receive Orphan Drug Designation from the FDA for ganaxolone in Fragile X Syndrome.
“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.”
The Orphan Drug Designation for ganaxolone enables Marinus a 7-year-period of exclusive marketing in the US alongside tax credits for clinical research expenditure, the scope to seek annual grant funding, assistance for clinical research trial design and removal of Prescription Drug User Fee Act (PDUFA) filing fees.
Caused by a mutation of the FMR1 gene, FXS is a rare disease that has been considered to be the most common genetic cause of autism.
The syndrome results in a number of developmental disorders and symptoms such as cognitive impairment, behavioral challenges and learning disabilities.
Based in Radnor, Marinus develops innovative therapeutics intended to treat epilepsy and neuropsychiatric related problems.