Marinus Pharmaceuticals receives FDA ODD for ganaxolone

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 designation applies to the active moiety of ganaxolone and is not dependent on the formulation. Marinus expects to initiate a multiple ascending dose study of a second generation ganaxolone formulation in the second quarter of 2023 and is planning to finalize the clinical program design for LGS in the second half of this year. "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."