Posted by Michael Wonder on 28 Aug 2021
European Medicines Agency grants Marinus Pharmaceuticals accelerated assessment of ganaxolone for treatment of CDKL5 deficiency disorder
26 August 2021 - Marinus Pharmaceuticals today announced that the CHMP of the EMA has granted its request for accelerated assessment of its lead product candidate, ganaxolone, for the treatment of seizures associated with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy.
The marketing authorisation application will be supported by data from the Phase 3 Marigold trial, a double-blind placebo-controlled trial in 101 patients. In the Marigold trial, patients treated with ganaxolone showed a 30.7% median reduction in 28 day major motor seizure frequency, compared to a 6.9% reduction for those receiving placebo, achieving the trial’s primary endpoint (p=0.0036).
