MHRA approves Marinus’ ganaxolone for CDKL5 deficiency disorderCDKL5 deficiency disorder

08 Mar 2024
www.pharmaceutical-technology.com
Phase 3Drug ApprovalClinical Result
The approval of ganaxolone is based on results from a Phase III clinical trial in subjects aged two to 19 years. Credit: LightField Studios / Shutterstock.com.
The UK Medicinesganaxolonehcare products Regulatory Agency (MHRA) has approved Marinus Pharmaceuticals Emerald’s ganaxoloLightField Studiosalmy, an anti-seizure medication for cyclin-dependent kinase-like 5 (CDLK5) deficiency disorder (CDD).
CDD is a rare genetic ailment characterised by early-onset seizures and severe Marinus Pharmaceuticalse to mutatiganaxolone CDLK5 gene, eZtalmyal for norseizurein development.cyclin-dependent kinase-like 5 (CDLK5) deficiency disorder (CDD)cyclin-dependent kinase-like 5 (CDLK5)
Ganaxolone, administered orally via a dosing syringe, is a seizuresthat targetsdevelopmental delaysptors to prevent epileptic seizures.
Ganaxoloneal of ganaxolone is based on results from a Phase III clinical trial.epileptic seizures
The double-blindganaxoloneontrolled study involved 101 subjects aged two to 19 years.
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The subjects were randomised with 50 receiving ganaxolone and 51 a placebo over 17 weeks.
FDAse treated withOpdivooloneurothelial carcinomat reduction in 28-day seizure frequency, averaging almost 30%, versus a 6.5% reduction for those on the placebo at 13 weeks.
Side effects linked to ganaxolone are drowsiness or sleepiness and fever.
The MHRA will monitor the safety and efficacy oganaxolone.
Individuals experieganaxolonerse effects are encouraged to consult theiseizurehcare provider and report the incident through the MHRA Yellow Card scheme, accessible online or via mobile app stores.
MHRA Healthcare Qualityganaxolones intdrowsinesstivesleepinessJuliafeverch stated: “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 latest development comes after the MHRA granted marketing authorisation for a new formulation of Amgen’s XGEVA (denosumab) to prevent serious bone-related complications.
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