Marinus Pharmaceuticals Announces FDA Acceptance for Filing and Priority Review of New Drug Application for Ganaxolone in CDKL5 Deficiency Disorder

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that the U.S. Food and Drug Administration (FDA) accepted for filing the company’s New Drug Application (NDA) for the use of ganaxolone in the treatment of seizures associated with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy. The NDA was granted Priority Review designation and the FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of March 20, 2022. Priority Review designation is given to an investigational medicine that, if approved, would be a significant improvement in the safety or effectiveness of the treatment of a serious condition and accelerates the timing of the FDA review of the application compared to a standard review.

“The FDA’s acceptance of our NDA submission is an important step toward potentially bringing the first approved therapy specifically for treatment of seizures associated with CDD—a devastating disorder with high unmet medical need—to families and healthcare providers,” said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. “We believe that ganaxolone has the potential to provide meaningful clinical benefit for patients and we look forward to working closely with the FDA during the review process.”

Ganaxolone received orphan drug designation and Rare Pediatric Disease (RPD) designation for CDD in June 2017 and July 2020, respectively. If the NDA is approved, Marinus is eligible to receive a RPD Priority Review Voucher that may be sold or transferred.

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