Marinus' Rare Genetic Epilepsy Drug Meets Primary Goal In Late-Stage Study

(RTTNews) - Marinus Pharmaceuticals Inc. (MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, Monday announced positive top-line results from its Phase 3 clinical trial evaluating the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder, a rare, genetic epilepsy with refractory seizures.

In the trial, patients given ganaxolone showed a significant 32.2 percent median reduction in 28-day major motor seizure frequency, compared to a 4.0 percent reduction for those receiving the placebo, achieving the primary endpoint.

Ganaxolone was generally well tolerated with a safety profile consistent with previous clinical studies.

Based on these results, Marinus plans to submit an NDA for ganaxolone in the treatment of CDD to the U.S. Food and Drug Administration in mid-2021 and a Marketing Authorization Application for ganaxolone for the treatment of CDD to the European Medicines Agency by the end of third quarter of 2021.