Pharma Focus Asia

Marinus Pharmaceuticals Receives FDA Orphan Drug Designation for Ganaxolone

Thursday, March 23, 2023

Marinus Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to ganaxolone for the treatment of Lennox-Gastaut syndrome (LGS).

This is the seventh orphan drug designation for ganaxolone, strengthening its potential as an innovative treatment option for rare epilepsies and seizure disorders, including LGS.

LGS is a severe form of epilepsy that begins in childhood with neurodevelopmental impairment and intractable atonic, tonic and generalised seizures. Given the overlap in seizure types and etiologies with other disorders where ganaxolone has therapeutic potential, such as CDKL5 deficiency disorder and tuberous sclerosis complex, it is believed that ganaxolone represents a promising opportunity to address the continuing unmet need in the LGS patient population.

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