Medicines Healthcare Products Regulatory Agency
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.
Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said:
Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us.
Were 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 treatment is taken orally through a dosing syringe. The prescribing doctor or pharmacist will advise as to the number of doses each person should take.
Ganaxolone is a steroid that attaches to specific receptors in the brain and aims to stop epileptic seizures.
This approval is supported by evidence from a phase 3 randomised, double-blind, placebo-controlled clinical trial involving 101 patients with CDD aged 2 to 19 years.
Chosen at random, 51 patients were given the placebo and the other 50 received 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%, while those receiving the placebo had a reduction of almost 6.5%.
The most common side effects of the medicine(which may affect more than 1 in 10 people) include feeling drowsy or sleepy and fever.
As with any medicine, the MHRA will keep the safety and effectiveness of ganaxolone under close review. Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the MHRA Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.
Notes to editors?
- The new marketing authorisation was granted on 7 March 2024 to Marinus Pharmaceuticals Emerald Limited.
- More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval.
- This was a randomised, double-blind clinical trial, where neither the participants nor the staff on the trial were aware of whether the patients were given ganaxolone or the placebo.For more information about the study, see the Summary of Product Characteristics.
- For more information about CDKL5 deficiency disorder, visit: https://curecdkl5.org.uk/.
- The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe.? All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
- The MHRA is an executive agency of the Department of Health and Social Care.
- For media enquiries, please contact the newscentre@mhra.gov.uk, or cal