Ganaxolone

Drug Profile

Ganaxolone

Alternative Names: CCD-1042; Ganaxalone-IV; gnx

Latest Information Update: 13 Sep 2017

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At a glance

  • Originator Purdue Pharma
  • Developer Marinus Pharmaceuticals; Purdue Pharma
  • Class Analgesics; Antidepressants; Antiepileptic drugs; Antimigraines; Anxiolytics; Neuroprotectants; Pregnanes; Small molecules
  • Mechanism of Action GABA A receptor agonists
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Status epilepticus; Infantile spasms; Fragile X syndrome; Epilepsy
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Epilepsy; Fragile X syndrome; Lennox-Gastaut syndrome; Postnatal depression
  • Phase I Status epilepticus
  • Preclinical Angelman syndrome
  • No development reported Post-traumatic stress disorders
  • Discontinued Infantile spasms; Migraine; Neuropathic pain; Partial epilepsies

Most Recent Events

  • 22 Nov 2017 Marinus Pharmaceuticals plans a phase II trial for Epilepsy in November 2017 (NCT03350035),
  • 31 Oct 2017 Marinus Pharmaceuticals plans a phase II trial for Epilepsy (In children) in 2018
  • 11 Sep 2017 Updated top-line efficacy data from a phase II trial in Epilepsy released by Marinus Pharmaceuticals
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