Marinus Pharmaceuticals Announces FDA Approval of ZTALMY for CDKL5 Deficiency Disorder

FDA has approved ZTALMY as a treatment for seizures associated with CDKL5 deficiency disorder (CDD).

Marinus Pharmaceuticals, a biopharmaceutical company focused on the development of treatments for seizure disorders, announced on March 18, 2022 that FDA has approved ZTALMY (ganaxolone) for the treatment of seizures associated with CDKL5 (cyclin-dependent kinase-like) deficiency disorder (CDD) in patients two years of age and older.

CDD is a rare form of genetic epilepsy characterized by early onset, difficult to control seizures, and severe neurodevelopmental impairment. It is caused by a mutation in the (CDKL5) gene, which produces a protein that is important for normal brain development and function.

“There has been a great unmet medical need for treatments that address seizures associated with CDKL5 deficiency disorder given their prominent role and profound impact on patients,” said Scott Demarest, MD, principal investigator for the Marigold trial and neurologist and clinical director of precision medicine at Children’s Hospital Colorado. “To date, antiseizure treatment decisions have been based on very limited clinical evidence in this patient population and the resulting outcomes underscore the need for therapies that further improve seizure control. Thanks to our research and this trial, we now have the first treatment specifically approved for seizures associated with CDKL5 deficiency disorder that was shown to have a positive benefit-risk profile.”

Source: Marinus