Ztalmy

Neurology· 780 cited findings· 1 source document

Company: Marinus Pharmaceuticals

What the regulatory reviews say

Ztalmy (ganaxolone) is indicated for the adjunctive treatment of epileptic seizures associated with CDD in patients aged 2 to 17 years. It received orphan medicinal product designation on 13 November 2019 and was approved on 18 March 2022.

In terms of efficacy, the median percent change from baseline in 28-day seizure frequency for major motor seizure types was -29.39% for Ztalmy (GNX) compared to -6.49% for placebo (PBO). The percentage of subjects with at least a 50% reduction in major motor seizure frequency was 30.6% for Ztalmy (GNX) versus 12.0% for placebo (PBO), with a p-value of 0.0283 for the 50% responder rate.

Regarding safety, 88.2% of patients experienced adverse events (AEs), with 9.8% experiencing serious AEs. Treatment-related AEs were observed in 43.1% of patients, and 20.6% experienced severe AEs. Discontinuation due to AEs occurred in 1 patient in the ganaxolone group compared to 4 in the placebo group.

Key figures — cited to the source page

Median percent change from baseline in 28-day seizure frequency for major motor seizure types (GNX)	-29.39	Marinus Emerald — Ztalmy EMA EPAR public assessment report, p.77
Median percent change from baseline in 28-day seizure frequency for major motor seizure types (PBO)	-6.49	Marinus Emerald — Ztalmy EMA EPAR public assessment report, p.77
Percentage of subjects with >=50% reduction in major motor seizure frequency (GNX)	30.6	Marinus Emerald — Ztalmy EMA EPAR public assessment report, p.77
Percentage of subjects with >=50% reduction in major motor seizure frequency (PBO)	12.0	Marinus Emerald — Ztalmy EMA EPAR public assessment report, p.77
P-value for 50% responder rate	0.0283	Marinus Emerald — Ztalmy EMA EPAR public assessment report, p.77
Patients experiencing AEs	88.2	Marinus Emerald — Ztalmy EMA EPAR public assessment report, p.101
Patients experiencing Serious AEs	9.8	Marinus Emerald — Ztalmy EMA EPAR public assessment report, p.101
Approval date	18 March 2022	Marinus Emerald — Ztalmy EMA EPAR public assessment report, p.110

Source documents

Marinus Emerald — Ztalmy EMA EPAR public assessment report (EMEA/H/C/005825, 2023) (2023)

Explore the full evidence

All 780 cited findings for Ztalmy — searchable, comparable and answerable in plain language — are in the Neurology research library →

Figures are as stated in the cited regulatory review documents (different drugs are studied in different trials — values are not head-to-head comparisons). Educational reference only — not medical or investment advice.