Express GABA receptor allosteric modulator is eligible for priority review, significantly reducing the frequency of seizures

▎Editor of WuXi AppTec's content team recently, Marinus Pharmaceuticals announced that the U.
S.
FDA has granted priority review status for its new drug application (NDA) for its GABAA receptor positivity modulator, ganaxolone, to treat epileptic seizures related to CDKL5 deficiency (CDD)
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The PDUFA target date for this application is set at March 20, 2022
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This new drug application is based on data support from a phase 3 clinical trial, and a total of 101 patients were enrolled
.

On the 28th day of treatment, the trial reached the primary endpoint.
The median reduction in the frequency of major motor seizures in the ganaxolone group was 30.
7% (p=0.
0036), and that in the placebo group was 6.
9%
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In the open-label extension study, patients who received ganaxolone for at least 12 months (n=48) had a median reduction in the frequency of major motor seizures by 49.
6%
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In terms of safety, ganaxolone is generally well tolerated in this phase 3 trial and showed safety characteristics consistent with previous clinical trials.
The most common adverse event was drowsiness
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CDKL5 deficiency is a serious and rare genetic disease caused by mutations in the cyclin-dependent kinase-like 5 (CDKL5) gene located on the X chromosome
.

The disease is characterized by uncontrollable seizures and severe neurodevelopmental disorders.
There is currently no approved treatment
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Ganaxolone is an under-study sex modulator targeting GABAA receptors.
It has two modes of administration: intravenous injection and oral administration.

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GABA is one of the inhibitory neurotransmitters of the central nervous system, which is related to mood changes such as anxiety, tension, and depression
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Ganaxolone acts on neuronal synapses and extra-synaptic GABAA receptors to achieve anti-epileptic and anti-anxiety activities
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Ganaxolone obtained Orphan Drug Designation and Rare Pediatric Disease (RPD) designation for the treatment of CDKL5 deficiency diseases in June 2017 and July 2020, respectively
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Image source: Dr.
Scott Braunstein, CEO of 123RFMarinus, said: “We believe that ganaxolone has the potential to provide patients with meaningful clinical benefits.
We look forward to working closely with the FDA during the review process
.

” References: [1] Marinus Pharmaceuticals Announces FDA Acceptance for Filing and Priority Review of New Drug Application for Ganaxolone in CDKL5 Deficiency Disorder.
Retrieved September 24, 2021, from https:// The content team focuses on introducing global biomedical health research progress
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This article is not a treatment recommendation either
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If you need guidance on the treatment plan, please go to a regular hospital for treatment
.