Marinus Emerald — Ztalmy EMA EPAR public assessment report (EMEA/H/C/005825, 2023)
Marinus Emerald · 2023 · EMA EPAR public assessment report
Report summary
The EMA assessed Ztalmy (ganaxolone) for CDKL5 deficiency disorder (CDD) seizures in patients aged 2 and older. While initially fast-tracked, the application reverted to standard review due to concerns. Ztalmy demonstrated a statistically significant reduction in major motor seizure frequency (29.39% vs. 6.49% for placebo) and a higher 50% responder rate in a pivotal trial. However, somnolence and sedation were common adverse events. Non-clinical studies confirmed anticonvulsant activity but also highlighted potential for sedation and dependence. Pharmacokinetic data showed rapid absorption and extensive metabolism, influenced by food and age. Long-term safety data, especially concerning sexual maturation and growth, are limited, restricting the initial indication to children and adolescents.
Key findings — cited to the page
| EC10 Daphnia sp. Reproduction Test | 43 µg/L | · p.36 |
| GNX oral suspension concentration | 50 mg/mL | · p.46 |
| Female to male ratio in CDKL5 deficiency disorder | 4:1 | · p.13 |
| Recommended daily dose for patients > 28 kg | 1800 mg/day | Ztalmy · p.14 |
| Number of subjects in population PK analysis | 146 | 1042-CDD-POPPK-001 · p.43 |
| Number of PK observation records in population PK analysis | 694 | 1042-CDD-POPPK-001 · p.43 |
| Recommended daily dose for patients ≤ 28 kg | 63 mg/kg/day | Ztalmy · p.14 |
| Orphan medicinal product designation date | 13 November 2019 | Ztalmy · EU · p.9 |
| Marketing authorisation application submission date | 9 October 2021 | Ztalmy · p.9 |
| CHMP positive opinion date | 25 May 2023 | Ztalmy · p.12 |
| Incidence of CDKL5 deficiency disorder | 1:40,000 to 1:60,000 live births | · p.13 |
| Cmax increase with high-fat meal | 2 fold | 1042-0400 · p.46 |
Source: Marinus Emerald ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →