In advance of topline data from that study, Marinus has begun preparations for an Expanded Access Program (EAP) in CDD that will allow the company, on positive data, to offer ganaxolone to patients who were unable to participate in the Phase 3 study. The global, double blind, placebo-controlled Phase 3 Marigold Study evaluating the use of oral ganaxolone in children and young adults has enrolled 101 patients between the ages of 2 and 21 with a confirmed disease related CDKL5 gene variant.
Ganaxolone, a positive allosteric modulator of GABAA receptors, is being developed in intravenous and oral formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Unlike benzodiazepines, ganaxolone exhibits anti-seizure and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors. Ganaxolone has been studied in more than 1,600 subjects, both pediatric and adult, at therapeutically relevant dose levels and treatment regimens for up to four years.
CDD is a serious and rare genetic disorder that is caused by a mutation of the cyclin‑dependent kinase‑like 5 (CDKL5) gene, located on the X chromosome. It predominantly affects females and is characterized by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment. The CDKL5 gene encodes proteins essential for normal brain function. Most children affected by CDD cannot walk normally, talk, or care for themselves. Many also suffer from scoliosis, visual impairment, gastrointestinal difficulties and sleep disorders. There are no treatments approved specifically for CDD. Genetic testing is available to determine if a patient has a mutation in the CDKL5 gene. No previous late-stage clinical trials have been conducted in this patient population.