Marinus announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation to ganaxolone for the treatment of Fragile X Syndrome.

FXS, caused by a mutation in the FMR1 gene, is the most common genetic cause of autism. There are no known cures or approved treatment for FXS.

Ganaxolone, a CNS-selective GABA-A modulator, is being evaluated in three dosage forms: intravenous (IV), capsule, and liquid. It acts on a well-characterized synaptic and extrasynaptic GABA-A target known for anti-seizure and anti-anxiety activity. Clinical studies involving over >1,400 pediatric and adult patients have studied ganaxolone at therapeutically relevant dose levels and treatment regimens for up to 2 years. Ganaxolone was shown to be generally safe and well tolerated.

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