The European Commission (EC) has approved Marinus Pharmaceuticals’ ZTALMY (ganaxolone) as an adjunctive treatment for epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged between two and 17 years.
Patients aged 18 years and older may continue with ZTALMY.
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ZTALMY oral suspension functions as a neuroactive steroid GABA [gamma-aminobutyric acid] receptor modulator, exhibiting its effects on a well-established brain target known for its anti-seizure properties.
The authorisation follows a positive opinion in May 2023 from the European Medicines Agency’s Committee for Medicinal Products for Human Use.
The approval applies to all 27 member states of the European Union and to Norway, Liechtenstein and Iceland.
The approval from the EC was based on data from the Phase III Marigold double-blind placebo-controlled study, which randomised and treated 101 patients with ZTALMY.
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By GlobalDataThe study attained its primary endpoint by demonstrating a median 30.7% reduction in 28-day major motor seizure frequency compared to a 6.9% median reduction for those receiving a placebo.
Marinus chairman and CEO Scott Braunstein stated: “The approval by the EC represents a significant milestone for children, families and physicians who, until now, have been challenged by the impact of seizures and lack of treatments available for CDD.
“This achievement reflects our organisation’s unwavering commitment to bringing an effective treatment option to individuals living with CDD and we are grateful to all the stakeholders who made the approval possible.”
