Ganaxolone is not recommended, within its marketing authorisation, as an add-on treatment option for seizures caused by cyclin-dependent kinase‑like 5 (CDKL5) deficiency disorder (CDD) in children and young people aged 2 to 17 years and adults who turn 18 while on treatment.
This recommendation is not intended to affect treatment with ganaxolone that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop. For children and young people, this decision should be made jointly by the healthcare professional and the child or young person, or their parents or carers.
Why the committee made these recommendations
Usual care for seizures caused by CDD includes antiseizure medications. There is no specific treatment for controlling seizures caused by CDD, so people often try several antiseizure medications and add-on treatments.
Clinical trial evidence suggests that ganaxolone plus usual care reduces seizure frequency compared with placebo plus usual care. But it is uncertain how much ganaxolone reduces seizure frequency because there was a large increase in seizure frequency in the placebo group in the trial. There are also uncertainties in how well it works in the long term.
There are structural uncertainties in the economic model which mean that the cost-effectiveness estimates for ganaxolone are not reliable. There is not enough evidence to establish that ganaxolone is cost effective. So, ganaxolone is not recommended.