RADNOR, PA — Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) announced that the U.S. Food and Drug Administration (FDA) recently 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.”
LGS is a severe form of epilepsy that begins in childhood with neurodevelopmental impairment and intractable atonic, tonic and generalized 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, Marinus believes that ganaxolone represents a promising opportunity to address the continuing unmet need in the LGS patient population.
The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval, if received, exemption of FDA application fees and tax credits for qualified clinical trials.
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