Basic Information
EMA regulatory identification and product classification information
EMA Identifiers
Overview Summary
Comprehensive product overview and regulatory summary
Ztalmy is a medicine used to treat epileptic seizures in children from 2 to 17 years of age who have a condition known as cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder. These patients can continue taking Ztalmy when they become adults if a clear benefit has been observed.
The medicine is used in combination with other anti-epileptic medicines.
CDKL5 deficiency disorder is rare, and Ztalmy was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 13 November 2019. Further information on the orphan designation can be found on the EMA website.
Ztalmy contains the active substance ganaxolone.
Active Substances (1)
Ganaxolone
Documents (12)
Ztalmy : EPAR - Orphan Maintenance Assessment Report
July 31, 2023
CHANGES_SINCE_INITIAL_AUTHORISATION
Ztalmy : EPAR - Public Assessment Report
July 31, 2023
CHANGES_SINCE_INITIAL_AUTHORISATION
Ztalmy : EPAR - Medicine Overview
July 31, 2023
OVERVIEW_DOCUMENT
Ztalmy : EPAR - Risk management plan
July 31, 2023
RISK_MANAGEMENT_PLAN_SUMMARY
Ztalmy : EPAR - Procedural steps taken and scientific information after authorisation (archive)
July 11, 2024
CHANGES_SINCE_INITIAL_AUTHORISATION
Ztalmy : EPAR - Procedural steps taken and scientific information after authorisation
June 2, 2025
CHANGES_SINCE_INITIAL_AUTHORISATION
CHMP summary of positive opinion for Ztalmy
May 26, 2023
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Ztalmy : EPAR - Public Assessment Report
July 31, 2023
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Ztalmy : EPAR - All authorised presentations
July 31, 2023
AUTHORISED_PRESENTATIONS
Ztalmy : EPAR - Orphan Maintenance Assessment Report
July 31, 2023
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Ztalmy : EPAR - Product Information
July 31, 2023
DRUG_PRODUCT_INFORMATION
CHMP summary of positive opinion for Ztalmy
May 26, 2023
CHANGES_SINCE_INITIAL_AUTHORISATION
Overview Q&A (7)
Question
How is Ztalmy used?
Answer
The medicine can only be obtained with a prescription, and treatment should be started and supervised by a doctor who has experience in treating patients with epilepsy.
Ztalmy is available as a liquid to be taken by mouth and is usually given three times a day. The dose is determined by the patient’s weight.
For more information about using Ztalmy, see the package leaflet or contact your doctor or pharmacist.
Question
How does Ztalmy work?
Answer
The active substance in Ztalmy, ganaxolone, mimics the action of a substance in the body called allopregnanolone. It switches on so-called GABA receptors, which reduces excessive electrical activity in the brain and thus lowers the number of seizures.
Question
What benefits of Ztalmy have been shown in studies?
Answer
A main study showed that Ztalmy reduces the frequency of seizures in children and adolescents with CDKL5 deficiency disorder who are taking at least one other epilepsy medicine.
The study involved a total of 101 patients with CDKL5 deficiency disorder and compared Ztalmy with placebo (a dummy treatment), both given in addition to existing epilepsy medicines.
On average, the monthly number of major seizures was reduced by 29% in the group of patients treated with Ztalmy, and by 6% in the group treated with placebo.
Question
What are the risks associated with Ztalmy?
Answer
For the full list of side effects and restrictions with Ztalmy, see the package leaflet.
The most common side effects with Ztalmy (which may affect more than 1 in 10 people) include sleepiness and fever.
Question
Why is Ztalmy authorised in the EU?
Answer
The main study showed that Ztalmy is effective at reducing the number of seizures in children with CDKL5 deficiency disorder. The side effects are considered manageable. The European Medicines Agency therefore decided that Ztalmy’s benefits are greater than its risks and it can be authorised for use in the EU.
Question
What measures are being taken to ensure the safe and effective use of Ztalmy?
Answer
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Ztalmy have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Ztalmy are continuously monitored. Suspected side effects reported with Ztalmy are carefully evaluated and any necessary action taken to protect patients.
Question
Other information about Ztalmy
Answer
Ztalmy received a marketing authorisation valid throughout the EU on 26 July 2023.