Ganaxolone is under clinical development by Marinus Pharmaceuticals and currently in the Phase II and Phase III in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect Ganaxolone’s likelihood of approval (LoA) and phase transition for Dravet Syndrome (Severe Myoclonic Epilepsy of Infancy) took place on 12 Sep 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.

In addition, the same event on 12 Sep 2022 increased Ganaxolone’s LoA and PTSR for Lennox-Gastaut Syndrome.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Ganaxolone Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

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), Lennox Gastaut syndrome and female children with epilepsy caused by a mutation of the protocadherin 19 gene (PCDH19), and Dravert syndrome, 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),and 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.

Quick View Ganaxolone LOA Data

Report Segments
  • Innovator (NME)
Drug Name
  • Ganaxolone
Administration Pathway
  • Intravenous
  • Oral
Therapeutic Areas
  • Central Nervous System
  • Genetic Disorders
  • Women’s Health
Key Developers
Highest Development Stage
  • Marketed

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 its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.