The Medicines and Healthcare products Regulatory Agency (MHRA) has approved Marinus Pharmaceutical’s Ztalmy (ganaxolone) to treat cyclin-dependant kinase-like 5 (CDKL5) deficiency disorder (CDD).
The approval marks Ztalmy as the first anti-seizure medication to be used in the UK to treat the rare epileptic seizure disorder.
Affecting around one in 42,000 people in the UK, CDD is a rare genetic disorder that occurs from mutations in the CDLK5 gene, which is crucial for normal brain development, causing seizures as early as the first week of life.
Ztalmy, taken orally through a dosing syringe, is a steroid that attaches to specific receptors in the brain and aims to stop epileptic seizures from occurring.
The approval was supported by evidence from the phase 3 Marigold randomised, double-blind, placebo-controlled clinical trial, which involved 101 patients – 50 who received Ztalmy for 17 weeks and 51 in a placebo group – with CDD aged two to 19 years.
After 13 weeks, a reduction in seizure frequency of almost 30% was observed in patients receiving the steroid, while those receiving the placebo had a reduction of almost 6.5%.
Patients treated with Ztalmy for at least 12 months experienced a reduction of nearly 50% in major motor seizure frequency.
Julian Beach, interim executive director, healthcare quality and access, MHRA, said: “We’re assured that the appropriate regulatory standards for the approval of this medicine have been met.
“Keeping patients safe and enabling their access to high-quality, safe and effective medical products are key priorities for us.”
The MHRA will continue to keep the safety and effectiveness of Ztalmy under close review.
Marinus’ treatment has already received approvals from the US Food and Drug Administration and the European Commission for patients living with CDD.
The company also provides a patient support programme in the US, Ztalmy One, to facilitate access to treatment for patients and provide ongoing prescription drug support and education throughout the treatment journey.
