European Commission approves Ztamly for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder

Ztamly may be continued in patients 18 years of age and older.

The EC approval of Ztamly in CDD is supported by data from the Phase III Marigold double-blind placebo-controlled trial, in which 101 patients were randomized and individuals treated with Ztamly 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 (p=0.0036). In the Marigold open label extension study, patients treated with Ztamly for at least 12 months (n=48) experienced a median 49.6% reduction in major motor seizure frequency. In the clinical development program, Ztamly demonstrated efficacy, safety and tolerability with the most common adverse reactions (incidence greater then 5% and at least twice the rate of placebo) in the Ztamly group being somnolence, pyrexia, salivary hypersecretion and seasonal allergy. In May 2022, the results from the Marigold study were published in The Lancet Neurology.