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Marinus Pharmaceuticals Receives European Commission Approval for ZTALMY® (Ganaxolone) as Adjunctive Therapy for Epileptic Seizures Associated With CDKL5 Deficiency Disorder

Monday, July 31, 2023

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) has received approval from the European Commission (EC) for the oral suspension medication ZTALMY® (ganaxolone). This approval allows ZTALMY to be used as an adjunctive treatment for epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged two to 17 years. Furthermore, the medication can also be used for patients who are 18 years and older.

Scott Braunstein, M.D., the Chairman, and Chief Executive Officer of Marinus, expressed the significance of this approval for children, families, and physicians who have faced the challenges of CDD without adequate treatment options. The approval reflects Marinus Pharmaceuticals' commitment to providing an effective solution for individuals living with CDD, and they are grateful to all stakeholders involved in making this approval possible.

The EC approval of ZTALMY for CDD is supported by data from the Phase 3 Marigold trial, which was a double-blind placebo-controlled study involving 101 patients. The study demonstrated that patients treated with ZTALMY experienced a median 30.7% reduction in 28-day major motor seizure frequency, compared to a median 6.9% reduction in the placebo group, successfully meeting the primary endpoint of the trial.

This approval comes after a positive opinion was issued by the Committee for Medicinal Products for Human Use of the European Medicines Agency in May 2023. The approval covers all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Marinus Pharmaceuticals and Orion Corporation entered into a collaboration agreement in July 2021, granting Orion the right to commercialize ZTALMY in Europe.

CDKL5 deficiency disorder is a rare genetic condition characterized by severe neurodevelopmental impairment and difficult-to-control seizures. The disorder is caused by a mutation in the cyclin-dependent kinase-like 5 (CDKL5) gene, which plays a crucial role in normal brain development and function.

ZTALMY, the approved medication, is a neuroactive steroid GABAA receptor modulator that has been proven to have anti-seizure effects. It is now approved for adjunctive treatment of epileptic seizures associated with CDKL5 deficiency disorder in patients aged two to 17, as well as in patients who are 18 years and older.

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