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.
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.
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 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/.
