FDA approval for first drug for rare form of epilepsy

Those living in the US with CDKL5 deficiency disorder, or CDD, may now have access to the first FDA-approved treatment for their condition. ZTALMY® (ganaxolone) is an oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD).

“Today is a historic milestone not only for Marinus but for CDD patients, families and caregivers who have long been navigating the unpredictable, often devastating reality of living with uncontrolled seizures,” said Scott Braunstein, MD, Chief Executive Officer of Marinus. “The approval of ZTALMY would not have been possible without the patients, caregivers and investigators who participated in the clinical trials to develop this important new therapy.”

CKDL5 deficiency disorder is a rare genetic disorder, causing seizures, developmental delay, and disability. Most children living with the disorder have 1 to 5 seizures every day. These seizures usually begin within a few months after birth, and are difficult to control with medications.

“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 (PI) 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.”

“As the mother of a daughter living with CDD, I’ve experienced first-hand the devastating impact seizures can have on these patients,” said Karen Utley, President and Co-founder of the International Foundation for CDKL5 Research. “This approval is monumental for the CDD community – bringing not only the first approved treatment option specifically for CDD patients, but renewed hope to those who have struggled to find medications that are effective in significantly reducing the number of seizures these patients experience on a daily basis.”

Ana Ovey