> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# FDA OKs Marinus Ganaxolone NDA Filing
- URL: https://www.fdaweb.com/fda-oks-marinus-ganaxolone-nda-filing/
- Published: 2021-01-13T12:00:00.000Z
- Updated: 2026-09-14T16:42:36.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5148440

FDA has told Marinus Pharmaceuticals that the efficacy and safety data resulting from the company’s Phase 3 Marigold Study using oral ganaxolone in children and adults with CDKL5 deficiency disorder appear sufficient to support the filing of an NDA. The company [says](https://www.marketwatch.com/press-release/marinus-pharmaceuticals-receives-positive-response-from-fda-on-sufficiency-of-one-phase-3-clinical-trial-for-filing-of-new-drug-application-nda-for-the-use-of-ganaxolone-in-cdkl5-deficiency-disorder-cdd-2021-01-13?tesla=y&ref=fdaweb.com) that the adequacy of the data to support the drug’s approval for the proposed indication will be a matter of future agency review. It says that based on the feedback from a recent meeting with FDA, it is targeting a pre-NDA meeting by the end of the first quarter of 2021 to support submission.

The company describes ganaxolone as a positive allosteric modulator of GABAA receptors that is being developed in intravenous and oral formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. It says that in the Marigold trial, patients treated with ganaxolone showed a significant 32.2% median reduction in 28-day major motor seizure frequency, compared to a 4% reduction in those receiving a placebo.