FDA approves GABA receptor allosteric modulator ganaxolone

FDA Approved:Yes (First approved March 18, 2022)
Brand Name:Ztalmy
Compound Name:ganaxolone
Dosage Form: Oral Suspension
Company: Marinus Pharmaceuticals, Inc.

FDA Approved: FDAYes (First approved March 18, 2022)

The U.
S.
Food and Drug Administration has approved Ztalmy (ganaxolone) oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency (CDD) in patients over two years of age

CDKL5 deficiency is a serious and rare genetic disorder caused by mutations in the CDKL5
gene located on the X chromosome .

The disease is characterized by uncontrollable seizures and severe neurodevelopmental impairment for which there is no previously approved therapy

CDKL5

Ztalmy is a positive allosteric modulator targeting GABAA receptors with both intravenous and oral administration options
.

GABA is one of the inhibitory neurotransmitters in the central nervous system, which is related to emotional changes such as anxiety, tension and depression

Ztalmy acts on neuronal synapses and extrasynaptic GABAA receptors to achieve antiepileptic and anxiolytic activity

The approval is based on data from a randomized, double-blind, placebo-controlled Phase 3 clinical trial that enrolled 101 patients
.

On treatment day 28, the trial met its primary endpoint, with a median reduction of 30.
7% (p=0.
0036) in the frequency of major motor seizures in the Ztalmy group and a 6.
9% reduction in the placebo group

The median reduction in major motor seizure frequency was 30.

The main information is summarized as follows:

• CDKL5 deficiency (CDD) is a serious and rare genetic disorder caused by mutations in the cyclin-dependent kinase-like 5 (CDKL5) gene located on the X chromosome
  .
  
  CDD is characterized by early onset, uncontrolled seizures, and severe neurodevelopmental impairment
  
  

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