BRIEF—Ztalmy approved by MHRA for seizures associated with CDKL5 deficiency

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has today (March 7) approved US drugmaker Marinus Pharmaceuticals’ Ztalmy (ganaxolone) as the first anti-seizure medication in the UK to treat cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD).

The drug has previously been approved for the same indication in the USA and Europe.

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.

This approval is supported by evidence from a Phase III randomized, double-blind, placebo-controlled clinical trial involving 101 patients with CDD aged 2 to 19 years.

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%, while those receiving the placebo had a reduction of almost 6.5%.