Ganaxolone is under clinical development by Marinus Pharmaceuticals and currently in Phase III for X Chromosome Disorders. According to GlobalData, Phase III drugs for X Chromosome Disorders have a 29% 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 overview

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 and epileptic encephalopathies. 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 and cyclin-dependent kinase-like 5 (CDKL5).

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 therapies for the treatment of rare genetic epilepsies and other seizure disorders. The company’s product includes 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’s 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.

This content was updated on 10 June 2024

Data Insights


The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.