Ztalmy

ganaxolone

Table of contents

Opinion

On 25 May 2023, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Ztalmy, intended for the treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in children and adolescents. The applicant for this medicinal product is Marinus Pharmaceuticals Emerald Limited.

Ztalmy will be available as a 50 mg/ml oral suspension. The active substance of Ztalmy is ganaxolone, an antiepileptic (ATC code: N03AX27). The precise mechanism of action is not known but its anticonvulsant effects are thought to result from a positive allosteric modulation of the GABAA receptors.

The benefits of Ztalmy lie in its ability to reduce the frequency of the seizures associated with CDD as demonstrated in a double-blind, randomised, placebo-controlled study in children and adolescents. The most common side effects are somnolence and pyrexia.

The full indication is:

Ztalmy is indicated for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older.

The treatment should be initiated and supervised by physicians with experience in the treatment of epilepsy.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.

Key facts

Name
Ztalmy
Agency product number
EMEA/H/C/005825
International non-proprietary name (INN) or common name
  • ganaxolone
Active substance
  • Ganaxolone
Therapeutic area
Epileptic Syndromes
Spasms, Infantile
Orphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Date opinion adopted
26/05/2023
Company name
Marinus Pharmaceuticals Emerald Limited
Status
Positive
Application type
Initial authorisation

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