Expert Interview
A look at the recent approval of Marinus’ Ztalmy (ganaxalone) and potential use in CDKL5 deficiency disorder
Ticker(s): MRNSInstitution: Johns Hopkins
- Professor of Neurology, Director, Child Neurology Residency Program and Medical Director of the Pediatric Ketogenic Diet Center at Johns Hopkins.
- Treats two patients with CDKL5 deficiency disorder and is familiar with ganaxalone.
- Research focuses on the diagnosis and treatment of childhood seizures and epilepsy, particularly treatments other than medications such as diet, neurostimulation and surgery; one of the world experts on dietary treatment for epilepsy.
Please describe your clinical practice. How many patients with CDKL5 Deficiency Disorder do you see on a yearly basis?
How often is seizure control challenging, and require multiple anticonvulsants or vagal nerve stimulation? How common is it for seizures in CDKL5 deficiency disorder to be resistant to treatment?
From the ongoing research aiming to better understand how CDKL5 mutations affect brain function so that potential treatments can be developed, which one are you most excited about and why?
Can you tell us more about the standard of care for CDKL5 Deficiency Disorder, and how ganaxolone could fit in the space? What are some of the unmet needs it could meet?
Could you please discuss the results of the Phase 3 Marigold study, where, in 101 patients treated with ganaxolone, 30.7% showed a median reduction in 28-day major motor seizure frequency, vs 6.9% in placebo, (p=0.0036). Patients in the open-label extension study treated with ganaxolone for at least 12 months (n=48) experienced a median 49.6% reduction in major motor seizure frequency. How does it compare to what you’ve been previously prescribing to your patients?
How likely are you to switch patients to Ganaxolone, if approved?
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