CDKL5 deficiency

Aug 21, 2023

Posted: 31 July 2023 | Catherine Eckford (European Pharmaceutical Review) | No comments yet

The European Commission (EC) has approved the first treatment for paediatric patients in the EU to treat seizures associated with CDKL5 deficiency disorder.

The first treatment for children and adolescents who have epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) has been approved in the EU by the European Commission (EC).

EC has granted approval of ZTALMY® (ganaxolone) oral suspension as adjunctive treatment in CDD patients from two to 17 years old.

The European regulatory body’s decision “represents a significant milestone” according to Dr Scott Braunstein, Chairman and Chief Executive Officer of Marinus Pharmaceuticals, the company that developed the treatment.

In the Phase III Marigold trial, 101 patients were enrolled. Data demonstrated a median 30.7 percent reduction in 28-day major motor seizure frequency. This is compared to a median 6.9 percent reduction to patients given placebo.

According to authors of the data published in The Lancet Neurology, the Phase III study is the first controlled trial in CDD to suggest a potential treatment benefit for ganaxolone. In the Phase III, the treatment was found to be well tolerated.

In the open label extension study for the trial, patients treated with ZTALMY for at least one year experienced a median 49.6 percent reduction in major motor seizure frequency.

The EC’s approval of ZTALMY follows a positive opinion issued in May 2023 by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

ZTALMY (ganaxolone)

ZTALMY (ganaxolone) is an oral small molecule neuroactive steroid γ-Aminobutyric acid type A (GABA-A) receptor modulator. According to the FDA, ZTALMY is the 3β-methylated synthetic analog of allopregnanolone, which is a derivative of progesterone.

Biopharmaceuticals, Clinical Development, Clinical Trials, Drug Development, Industry Insight, Regulation & Legislation, Research & Development (R&D), Therapeutics

Committee for Medicinal Products for Human Use (CHMP), European Commission (EC), European Medicines Agency (EMA), Marinus Pharmaceuticals

Ztalmy (ganaxolone)

Dr Scott Braunstein

CDKL5 deficiency disorder