RARE Daily

MHRA Approves Marinus Pharma’s Ztalmy to Treat CDKL5 Deficiency Disorder

March 8, 2024

Rare Daily Staff

The Medicines and Healthcare products Regulatory Agency has approved Marinus Pharmaceuticals’ Ztalmy as the first anti-seizure medication in the UK to treat cyclin-dependent kinase-like 5 deficiency disorder (CDD).

Cyclin-dependent kinase-like 5 deficiency disorder (CDD) is a rare genetic disorder caused by mutations in the CDLK5 gene, which is necessary for normal brain development, and affects around 1 in 42,000 people in the UK. Most people affected experience seizures from as early as the first week of life and have severe delays in development, impacting brain function, movement, speech and sight.

Ztalmy (ganaxolone) is the first anti-seizure medication to treat patients with the rare epileptic seizure disorder in the United Kingdom.

“Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us,” said Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access.

Ganaxolone is a steroid that attaches to specific receptors in the brain and aims to stop epileptic seizures.

The approval is supported by evidence from a phase 3 randomized, double-blind, placebo-controlled clinical trial involving 101 patients with CDD aged 2 to 19 years with 51 patients being given the placebo and the other 50 receiving ganaxolone for 17 weeks. After 13 weeks, there was a statistically significant difference in 28-day seizure frequency between the two groups. Patients receiving ganaxolone saw an average reduction in seizure frequency of almost 30 percent, while those receiving the placebo had a reduction of almost 6.5 percent. The most common side effects of the medicine (which may affect more than 1 in 10 people) include feeling drowsy or sleepy and fever.

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