The approval was backed by the results from the Phase 3 trial in which Ztalmy showed a median 30.7% reduction in 28-day major motor seizure frequency against a median 6.9% reduction in the placebo group
European Commission approves Marinus’ ZTALMY. (Credit: Robina Weermeijer on Unsplash)
Marinus Pharmaceuticals has received approval from the European Commission (EC) for Ztalmy (ganaxolone) oral suspension to treat epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD).
EC has approved Ztalmy for the adjunctive treatment of patients two to 17 years of age and it can be continued in patients aged 18 years and older.
Ztalmy is a neuroactive steroid GABAA receptor modulator. It targets the region in the brain that has anti-seizure effects.
The approval was backed by the results from the Phase 3 Marigold double-blind placebo-controlled trial. It randomised 101 patients to receive Ztalmy.
In the trial, epileptic seizure therapy demonstrated a median 30.7% reduction in 28-day major motor seizure frequency against a median 6.9% reduction in individuals receiving a placebo, achieving the study’s primary endpoint.
Patients who received Ztalmy for at least 12 months in the Marigold open-label extension study saw a median 49.6% decrease in the frequency of major motor seizures.
The most frequent side events in the Ztalmy group were somnolence, pyrexia, salivary hypersecretion, and seasonal allergies during the clinical development phase.
Marinus chairman and CEO Scott Braunstein said: “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.”
The clearance comes after a favourable opinion issued in May 2023 by the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA).
A collaboration deal between Marinus and Finland-based Orion was signed in July 2021, giving Orion the authority to market Ztalmy in Europe.
In March last year, the company secured the US Food and Drug Administration (FDA) approval for Ztalmy for the treatment of a rare type of genetic epilepsy related to seizures.