Ganaxolone Granted Orphan Drug Status for Lennox-Gastaut Syndrome

The Food and Drug Administration (FDA) has granted Orphan Drug designation to ganaxolone for the treatment of Lennox-Gastaut syndrome.

Lennox-Gastaut syndrome is a severe form of epilepsy. The disorder begins in childhood and is characterized by neurodevelopmental impairment and intractable atonic, tonic, and generalized seizures.

Ganaxolone is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator designed to regulate brain activity by inhibiting the abnormal electrical discharges that cause seizures. A multiple ascending dose study of a second generation formulation of ganaxolone is expected to begin in the second quarter of 2023.

“This is the seventh Orphan Drug designation for ganaxolone, which we believe reinforces its potential as an innovative treatment option for rare epilepsies and seizure disorders, including LGS,” said Kimberly McCormick, PharmD, Chief Regulatory and Quality Assurance Officer of Marinus Pharmaceuticals. “We look forward to working closely with the FDA and the LGS community as we finalize our clinical development plans over the next several months and are committed to continuing to pursue development paths for ganaxolone that could benefit more patients.”

Ganaxolone is currently marketed under the brand name Ztalmy for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder in patients 2 years of age and older.