European Commission Approval of ZTALMY® (ganaxolone) for the Adjunctive Treatment of Epileptic Seizures Associated with CDKL5 Deficiency Disorder
31 July 2023: Marinus Pharmaceuticals announced that the European Commission (EC) has granted 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. ZTALMY is the first treatment approved by the European Commission for seizures associated with CDKL5 deficiency disorder in children and adolescents.
The EC approval of ZTALMY in CDD is supported by data from the Phase 3 Marigold double-blind placebo-controlled trial, in which 101 patients were randomised 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 programme, 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 Neurology.1
The approval follows a positive opinion issued in May 2023 by the Committee for Medicinal Products for Human Use of the European Medicines Agency and is applicable to all 27 European Union member states plus Iceland, Norway and Liechtenstein. In July 2021, Marinus and Orion Corporation entered into a collaboration agreement which grants Orion the right to commercialise ZTALMY in Europe.
The approval by the European Commission represents a significant milestone for children, families and physicians who, until now, have long been challenged by the impact of seizures and lack of treatments available for CDD. This achievement reflects our organisation’s unwavering commitment to bring an effective treatment option to individuals living with CDD and we are grateful to all the stakeholders who made the approval possible.
Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus
References
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.
About CDKL5 Deficiency Disorder
CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder characterised by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment.2 It is caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. The CDKL5 gene produces a protein that is important for normal brain development and function.3
About ZTALMY®
ZTALMY (ganaxolone) is a neuroactive steroid GABAA receptor modulator that acts on a well-characterised target in the brain known to have anti-seizure effects. ZTALMY is an oral prescription medicine approved by the European Commission for the adjunctive treatment of epileptic seizures associated with CDKL5 deficiency disorder in patients two to 17 years of age. ZTALMY may be continued in patients 18 years of age and older.
