Marinus Pharmaceuticals has secured China’s National Medical Products Administration (NMPA) approval for Ztalmy (ganaxolone) oral suspension to treat a rare type of epileptic seizures.
Ztalmy is a neuroactive steroid indicated to treat seizures related to cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in people aged two years and older.
The drug is already approved in the US and European Union (EU) as a prescription medication for the treatment of CDD-associated epileptic seizures.
In late 2022, Marinus granted China-based Tenacia Biotechnology to develop and commercialise the drug in Mainland China, Hong Kong, Macau, and Taiwan.
The licensing agreement includes royalties and milestone-based payments.
Tenacia Biotechnology CEO Xiaoxiang Chen said: “CDD is a severe and rapidly progressive neurodevelopmental disorder and before ganaxolone, there had been no treatments approved for this condition in China.
“There is an urgent need to bring innovative new therapies to patients and families living with CDD, particularly as diagnosis rates continue to rise. We look forward to delivering the first and only treatment option for these patients in China.”
The NMPA approval of Ztalmy is based on results from Marigold, a double-blind, placebo-controlled Phase 3 clinical trial conducted in 101 patients.
In the Phase 3 study, treatment with Ztalmy significantly reduced major motor seizure frequency compared to placebo, meeting the primary trial endpoint of the study.
The long-term treatment with the drug in an open-label extension showed continued seizure reduction, maintaining a safety profile consistent with earlier studies.
Marinus Pharmaceuticals chief regulatory and quality assurance officer Kimberly McCormick said: “CDD brings unpredictability and increased healthcare needs and costs to patients and their families, and can significantly impact their quality of life.
“The approval in China represents an important step forward for patients living with CDD and underscores our commitment to bringing innovative medicines to people living with rare genetic epilepsies across the globe.”
In March 2022, Marinus received the US Food and Drug Administration (FDA) approval for Ztalmy oral suspension to treat a rare type of genetic epilepsy related to seizures.
