Ganaxolone’s Significant Effect on CDKL5 Deficiency Disorder: Elia Pestana-Knight, MD


The pediatric epileptologist at Cleveland Clinic provided insight on her presentation at the 2022 AAN Annual Meeting that evaluated ganaxolone in phase 3 study of patients with CDKL5 deficiency disorder. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

"The treatment-emergent adverse event percentage was 12% for the ganaxolone group and 9.8% for the placebo group. The discontinuation rate was 4% in the ganaxolone group and 8% in the placebo group. I think this speaks to what this medication brings to the table for the treatment of seizures in children who have CDKL5 deficiency disorder."

CDKL5 deficiency disorder (CDD) is a rare, genetically determined developmental and epileptic encephalopathy characterized by early-onset refractory seizures and severe neurodevelopmental impairment. After years of demonstrating its clinical benefit in trials, the FDA made a landmark decision, greenlighting ganaxolone (Ztalmy; Marinus Pharmaceuticals) as the first approved therapy for CDD. Approved under priority review and orphan drug and rare pediatric disease designations, the therapy is expected to be available via specialty pharmacy in July 2022.

Ganaxolone, a neuroactive steroid that acts as a positive allosteric modulator of the GABAA receptor, was accepted based on data from the phase 3 Marigold trial (NCT03572933), a double-blind, placebo-controlled trial that randomized 101 patients. At the 2022 American Academy of Neurology Annual Meeting (AAN), lead investigator Elia Pestana-Knight, MD, presented some of the topline notable data from the study, including the fact that ganaxolone resulted in a median 30.7% reduction in major motor seizure frequency compared with a 6.9% reduction in the placebo group.1

Pestana-Knight, pediatric epileptologist at Cleveland Clinic’s Epilepsy Center, sat down with NeurologyLive® ahead of the meeting to discuss the results in full, along with the significance of ganaxolone’s effect and how it may alter the care for patients with CDD.

For more coverage of AAN 2022, click here.

1. Olson H, Moosa AN, Rybak E, et al. Extended Duration Safety and Efficacy of Adjunctive Ganaxolone Treatment in Patients with CDKL5 Deficiency Disorder: 8-Month Minimum Open-Label Extension Follow-up. Presented at: AAN Annual Meeting; April 2-7, 2022; Seattle, WA, and virtual. Abstract 003540.
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