Ganaxolone is under clinical development by Marinus Pharmaceuticals and currently in Phase III for Status Epilepticus. According to GlobalData, Phase III drugs for Status Epilepticus have a 67% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Ganaxolone’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Ganaxolone (Ztalmy) is a synthetic analog of the neuroactive steroid allopregnanolone acts as anticonvulsant agent. It is formulated as suspension for oral route of administration, Ganaxolone is indicated treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older.
Ganaxolone nanoparticle formulation is under development for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) (X-chromosome disorder), status epilepticus (SE), tuberous sclerosis complex. The drug candidate is administered orally (capsule and suspension) and intravenously. Ganaxolone (synthetic analog of allopregnanolone) acts by targeting gamma-aminobutyric acid type A (GABAA) receptors.
It was also under development for the treatment of infantile spasm (West syndrome), adult partial-onset epileptic seizures, Angelman syndrome, smoking cessation, fragile-X syndrome, postpartum depression (PPD), Lennox Gastaut syndrome and female children with epilepsy caused by a mutation of the protocadherin 19 gene (PCDH19), and Dravert syndrome, treatment-resistant depression (TRD) and post-traumatic stress disorder.
Marinus Pharmaceuticals overview
Marinus Pharmaceuticals is a biopharmaceutical company. It discovers, develops and commercializes medicines for the treatment of rare genetic epilepsies and other seizure disorders. The company’s product is Ganaxolone, a novel gamma-aminobutyric acid (GABA) receptor modulator, which is developed in intravenous (IV) and oral formulations. Marinus Pharmaceuticals carries out research and development, educational grants, and other services. It markets products under the brand name Ztalmy. The company product help in the treatment of acute and chronic care settings, seizure disorders, rare epilepsies, pediatric epilepsies, brain disorders, and other disorders. It serves healthcare providers, caregivers, healthcare professionals, and others. Marinus Pharmaceuticals is headquartered in Radnor, Pennsylvania, the US.
For a complete picture of Ganaxolone’s drug-specific PTSR and LoA scores, buy the report here.