Drug Profile


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

Latest Information Update: 21 Feb 2017

Price : $50

At a glance

  • Originator Purdue Pharma
  • Developer Marinus Pharmaceuticals
  • Class Antiepileptic drugs; Antimigraines; 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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • New Molecular Entity Yes

Highest Development Phases

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

Most Recent Events

  • 14 Feb 2017 Preclinical trials in Angelman syndrome in USA (unspecified route)
  • 06 Jan 2017 Marinus Pharmaceuticals terminates a phase III extension trial for Partial epilepsies (Adjunctive treatment) in USA (NCT02519439)
  • 03 Jan 2017 Ganaxolone receives Orphan Drug status for Fragile X syndrome in USA
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