The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has approved Marinus Pharmaceuticals’ 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 (MAA) will be supported by data from the Phase III Marigold trial, which previously achieved its primary endpoint of a reduction in motor seizure frequency.
Ganaxolone, a positive allosteric modulator of GABAA receptors, was generally well-tolerated with a safety profile consistent with previous clinical trials.
"We believe this accelerated assessment by the EMA underscores ganaxolone's potential to address areas of unmet medical need for patients and families afflicted by CDD," said Kimberly McCormick, senior VP of regulatory affairs at Marinus.
"We plan to submit a marketing authorisation application by the end of the third quarter and look forward to working with the EMA during its review of the application. If approved, our collaboration with Orion Corporation supports our efforts to bring ganaxolone to European markets as quickly as possible for CDD patients who may benefit," she added.
Marinus Pharmaceuticals specialises in the development of innovative therapeutics to treat seizure disorders, and the CHMP endorsement closely follows its new drug application (NDA) submission to the US Food and Drug Administration (FDA) for the use of ganaxolone to treat seizures associated with CDD.
An NDA filing notification letter from the FDA is expected before the end of Q3 2021.