Ztalmy® (ganaxolone) oral suspension, made by Marinus Pharmaceuticals, has become the first FDA-approved treatment for cyclin-dependent kinase-like 5 deficiency disorder (CDD). It is indicated to treat seizures associated with CDD in patients who are at least two years old.
- CDD is a rare, difficult-to-treat form of epilepsy caused by a genetic mutation. Although other anti-seizure drugs are used off-label for CDD, there has been little clinical evidence to guide treatment decisions.
- Recommended dosing for Ztalmy is based on the patient’s weight:
- 28kg (62 pounds) or less: Starting dosage is 6mg/kg of the patient’s weight three times daily (18mg/kg/day), which can be titrated up to a maximum dosage of 21mg/kg three times daily (63mg/kg/daily).
- Greater than 28kg: Starting dosage is 150mg three times daily (450mg daily), which can be titrated up to a maximum dosage of 600mg three times daily (1,800mg daily).
- Ztalmy is expected to launch in July 2022 with a wholesale acquisition cost of $2,425 per 110mL bottle containing 50mg of ganaxolone per milliliter. It is currently awaiting controlled substance scheduling by the U.S. Drug Enforcement Administration (DEA).
