In September 2024, Marinus Pharmaceuticals announced its plans to launch ZTALMY (ganaxolone) for treating tuberous sclerosis complex (TSC). The company revealed that the Phase 3 TrustTSC trial had completed enrollment with 129 participants, and topline data was expected in the first half of Q4 2024. The trial maintained a low discontinuation rate of 6.2%, with 93% of patients transitioning to the open-label extension phase. Marinus is targeting a supplemental New Drug Application (sNDA) submission to the U.S. Food and Drug Administration (FDA) in April 2025, aiming for priority review. The company also highlighted a new patent allowance for ganaxolone's oral titration regimen, expected to extend through September 2042. With a U.S. market opportunity of approximately USD 2.5 billion for both CDKL5 deficiency disorder and TSC, Marinus is preparing for a rapid and efficient U.S. launch, pending FDA approval.
