RADNOR, PA — Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recently adopted a positive opinion recommending the approval of ZTALMY® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two to 17 years of age. ZTALMY may be continued in patients 18 years of age and older.
“The CHMP’s positive opinion reinforces the safety and efficacy profile of ZTALMY and is a testament to the tireless efforts and dedication of our team and the CDD community,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “This recommendation brings us one step closer to addressing a significant unmet need for CDD patients with treatment-resistant seizures in Europe. We look forward to the European Commission’s decision and working with our commercial partner, Orion Corporation, to bring this important treatment option to more patients who may benefit.”
The CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). A final decision by the EC is expected within 67 days of receipt of the CHMP opinion and will be applicable to all 27 EU member states plus Iceland, Norway and Liechtenstein. If approved, ZTALMY will be the first treatment in the EU indicated for the adjunctive treatment of seizures associated with CDD and will be commercialized by Orion Corporation.
Marinus’ application for the approval of ZTALMY in CDD is supported by data from the Phase 3 Marigold double-blind placebo-controlled trial, in which 101 patients were randomized and individuals treated with ZTALMY showed a median 30.7% reduction in 28-day major motor seizure frequency, compared to a median 6.9% reduction for those receiving placebo, achieving the trial’s primary endpoint (p=0.0036). In the Marigold open label extension study, patients treated with ZTALMY for at least 12 months (n=48) experienced a median 49.6% reduction in major motor seizure frequency. In the clinical development program, ZTALMY demonstrated efficacy, safety and tolerability with the most common adverse reactions (incidence ≥5% and at least twice the rate of placebo) in the ZTALMY group being somnolence, pyrexia, salivary hypersecretion and seasonal allergy. In May 2022, the results from the Marigold study were published in The Lancet Neurology1.
1 The Lancet Neurology, Volume 21, Issue 5, P417-427, May 01, 2022
2 Olson H et al. 2019 Pediatric Neurology
3 Jakimiec M et al. 2020 Brain Sci.
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