FDA indicates phase III Marigold Study of oral ganaxolone supports filing in CDKL5 deficiency disorder.- Marinus Pharma

Marinus Pharmaceuticals announced it has received a positive response from the FDA that the efficacy and safety data resulting from the company’s pivotal Phase III Marigold Study on the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD) appear sufficient to support the filing of a New Drug Application (NDA). Adequacy of these data to support an approval of ganaxolone for the proposed indication will be a matter of future FDA review. In the Phase III Marigold trial, patients treated with ganaxolone showed a significant 32.2% median reduction in 28-day major motor seizure frequency, compared to a 4.0% reduction for those receiving the placebo, achieving the trial’s primary endpoint (p=0.002). In this trial, ganaxolone was generally well tolerated with a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence. Comment: CDKL5 deficiency disorder (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. CDD is characterized by early-onset, difficult-to-control seizures and severe neuro-developmental impairment. Most children affected by CDD cannot walk, talk, or feed themselves. Currently, there are no therapies approved specifically for CDD.