MRNS - Marinus ganaxolone for severe epilepsy gets FDA orphan drug status

2023-03-22 09:39:04 ET

• The U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to Marinus Pharmaceuticals' ( NASDAQ: MRNS ) ganaxolone to treat Lennox-Gastaut syndrome (LGS).
• LGS is a severe form of epilepsy which usually shows up during infancy or early childhood.
• The company said the designation applies to the active moiety of ganaxolone and is not dependent on the formulation.
• Marinus expects to start a multiple ascending dose trial of a second generation ganaxolone formulation in Q2 2023 and plans to finalize the clinical program design for LGS in H2 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, chief regulatory and quality assurance officer, Marinus.
• The FDA grants orphan drug status to therapies which treat or prevent rare diseases that affect fewer than 200K people in the U.S. The designation provides certain incentives, including seven years of market exclusivity, if approved.

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