General Information

Ztalmy (ganaxolone) oral suspension is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator.

Ztalmy is specifically indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older.

Ztalmy is supplied as an oral suspension to be administered by mouth three times daily. It must be taken with food. 

Titrate Ztalmy gradually according to the recommended schedules. See full prescribing information.

Dosage for patients weighing 28 kg or less:

• the starting dosage is 6 mg/kg three times daily (18 mg/kg/day)
• the maximum dosage is 21 mg/kg three times daily (63 mg/kg/daily)

Dosage for patients weighing over 28 kg:

• the starting dosage is 150 mg three times daily (450 mg daily)
• the maximum dosage is 600 mg three times daily (1800 mg daily)

Mechanism of Action

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 (GABAA) receptor in the CNS.

Side Effects

Adverse effects associated with the use of Ztalmy may include, but are not limited to, the following:

• somnolence
• pyrexia
• salivary hypersecretion
• seasonal allergy

Clinical Trial Results

The FDA approval was based on data from the Phase 3 Marigold trial. The double-blind placebo-controlled trial enrolled 101 patients between the ages of 2 and 21 with a confirmed disease-related CDKL5 gene variant. The patients treated with Ztalmy showed a median 30.7% reduction in 28-day major motor seizure frequency, compared to a median 6.9% reduction for those receiving placebo, achieving the trial’s primary endpoint. In the Marigold open label extension study, patients treated with Ztalmy for at least 12 months (n=48) experienced a median 49.6% reduction in major motor seizure frequency.