The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Marinus Pharmaceuticals Emerald’s ganaxolone, marketed as Ztalmy, an anti-seizure medication for cyclin-dependent kinase-like 5 (CDLK5) deficiency disorder (CDD).

CDD is a rare genetic ailment characterised by early-onset seizures and severe developmental delays due to mutations in the CDLK5 gene, essential for normal brain development.

Ganaxolone, administered orally via a dosing syringe, is a steroid that targets specific brain receptors to prevent epileptic seizures.

The approval of ganaxolone is based on results from a Phase III clinical trial.

The double-blind placebo-controlled study involved 101 subjects aged two to 19 years.

The subjects were randomised with 50 receiving ganaxolone and 51 a placebo over 17 weeks.

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Those treated with ganaxolone showed a significant reduction in 28-day seizure frequency, averaging almost 30%, versus a 6.5% reduction for those on the placebo at 13 weeks.

Side effects linked to ganaxolone are drowsiness or sleepiness and fever.

The MHRA will monitor the safety and efficacy of the drug.

Individuals experiencing adverse effects are encouraged to consult their healthcare provider and report the incident through the MHRA Yellow Card scheme, accessible online or via mobile app stores.

MHRA Healthcare Quality and Access interim executive director Julian Beach stated: “Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us.

“We’re assured that the appropriate regulatory standards for the approval of this medicine have been met.

“As with all medicines, we will keep its safety under close review.”

The latest development comes after the MHRA granted marketing authorisation for a new formulation of Amgen’s XGEVA (denosumab) to prevent serious bone-related complications.