Ztalmy® (ganaxolone) is now available for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder in patients 2 years of age and older.
Cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) is a rare genetic disorder that results in difficult-to-control seizures and severe neurodevelopmental delay. The precise mechanism by which ganaxolone exerts its therapeutic effects in the treatment of seizures associated with CDD is unknown, but its anticonvulsant effects are thought to result from positive allosteric modulation of the gamma-aminobutyric acid type A receptor in the CNS. Ganaxolone has been classified as a Schedule V controlled substance.
The approval was based on data from the randomized, placebo-controlled, double-blind phase 3 Marigold study (ClinicalTrial.gov Identifier: NCT03572933), which evaluated the efficacy and safety of Ztalmy in 101 patients aged 2 to 19 years with genetically confirmed CDKL5 gene mutation. Findings from the study showed that treatment with Ztalmy met the primary endpoint demonstrating a statistically significantly reduced the frequency of monthly major motor seizures by a median of 30.7% vs 6.9% for placebo (P =.0036).
The most common adverse reactions associated with Ztalmy were somnolence, pyrexia, salivary hypersecretion, and seasonal allergy.
Ztalmy is supplied as a cherry-flavored oral suspension containing 50mg/mL of ganaxolone; each bottle contains 110mL of Ztalmy oral suspension. The medication is administered 3 times daily and must be taken with food.
Marinus Pharmaceuticals announces US commercial launch of Ztalmy® (ganaxolone) for seizures associated with CDKL5 deficiency disorder. News release. Marinus Pharmaceuticals, Inc. Accessed July 28, 2022. https://www.businesswire.com/news/home/20220722005348/en/Marinus-Pharmaceuticals-Announces-U.S.-Commercial-Launch-of-ZTALMY%C2%AE-ganaxolone-for-Seizures-Associated-With-CDKL5-Deficiency-Disorder