Tenacia, a central nervous system (CNS) specialist company incubated by Bain Capital, has announced that it has received marketing approval from the National Medical Products Administration (NMPA) for its in-licensed drug, ganaxolone oral suspension. This medication is indicated for the treatment of epileptic seizures in patients aged 2 years and above with cyclin-dependent kinase-like 5 (CDKL5) deficiency. Ganaxolone oral suspension is the world’s first and currently the only drug in China approved to treat epileptic seizures in patients with CDKL5 deficiency (CDD).
CDD is a rare genetic neurodevelopmental disorder characterized by uncontrollable epileptic seizures during infancy, severely impacting the quality of life for affected individuals. Patients often present with symptoms such as severe motor developmental delay, intellectual disability, sleep disorders, cerebral visual impairment, and gastrointestinal dysfunction. Epilepsy related to CDD can be a lifelong condition.
Ganaxolone, a neuroactive steroid and GABA-A receptor modulator, has demonstrated significant efficacy and a favorable safety profile in a pivotal global Phase III study. Compared to the placebo group, it significantly reduced the frequency of epileptic seizures in patients. The findings from a 2-year long-term open-label study indicated that 30% to 50% of patients experienced a reduction in seizure frequency of ≥ 50%, and nearly 10% of patients achieved seizure freedom within 3 months.- Flcube.com
