CDEK
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ganaxolone

Trade name: Ztalmy

Small moleculeapprovedOrphan Drug FDA

Approved

Mar 18, 2022

Ganaxolone, sold under the brand name Ztalmy, is a medication used to treat seizures in people with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder. Ganaxolone is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator. — Wikipedia

Clinical trial activity

23 trials · 10 clinical orgs · 1 marketing orgs

Phase 1
0
Phase 2
21
Phase 3
7
Phase 4
0

Earliest trial started Jan 1, 2007 (NCT00442104)

Timeline

2000s

  1. Jan 1, 2007

    Marinus Pharmaceuticals — Earliest Phase 2 Sponsor(trial)

2010s

  1. Jan 1, 2013

    Marinus Pharmaceuticals — Earliest Phase 3 Sponsor(trial)

  2. Jun 28, 2017

    Orphan Drug Designation

2020s

  1. Mar 18, 2022

    Marinus Pharmaceuticals — Earliest FDA Approval

  2. Mar 18, 2022

    Marinus Pharmaceuticals — Marketing Organization

  3. Mar 18, 2022

    Marinus Pharmaceuticals — NDA Secondary Org

  4. Mar 18, 2022

    Marinus Pharmaceuticals — NDA Organization

Indications

Approved for

Studied for

Mechanism of action

Approval history

  • approvedPriority reviewMar 18, 2022

Chemistry & pharmacology

Loading structure…

SMILES

CC(=O)[C@H]1CC[C@H]2[C@@H]3CC[C@H]4C[C@](C)(O)CC[C@]4(C)[C@H]3CC[C@]12C
Mol. weight
332.53 g/mol
Lipinski Ro5
Pass
Rule of 3
No
Chirality
Single Stereoisomer
Inorganic
No
Polymer
No
Delivery
Oral
Availability
Prescription Only

Oral

Yes

Parenteral

No

Topical

No

First in class

Sources

Also known as

  • 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one
  • ccd 1042
  • ccd-1042
  • ccd-1042
  • ccd-1042
  • ganaxolone
  • ganaxolone
  • ganaxolone
  • ganaxolone
  • ganaxolone

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT05814523Phase 3Sep 30, 2023Marinus Pharmaceuticals
NCT05249556Phase 3May 31, 2023Marinus Pharmaceuticals
NCT05757544Phase 2Apr 1, 2023Marinus Pharmaceuticals
NCT04391569Phase 3Aug 31, 2020Marinus Pharmaceuticals
NCT04285346Phase 2Apr 30, 2020Marinus Pharmaceuticals
NCT03865732Phase 3May 31, 2019Marinus Pharmaceuticals
NCT03572933Phase 3Jun 30, 2018Marinus Pharmaceuticals
NCT03350035Phase 2Feb 19, 2018Marinus Pharmaceuticals
NCT03460756Phase 2Dec 28, 2017Marinus Pharmaceuticals
NCT03228394Phase 2Jun 27, 2017Marinus Pharmaceuticals
NCT02900092N/ANov 1, 2016Harvard University
NCT02358538Phase 2Mar 1, 2015Marinus Pharmaceuticals, Sponsor GmbH
NCT02519439Phase 3Feb 1, 2015Marinus Pharmaceuticals
NCT01963208Phase 3Oct 1, 2013Marinus Pharmaceuticals
NCT01857531Phase 2Jun 1, 2013Duke University, National Institute on Drug Abuse (NIDA)
NCT01725152Phase 2Nov 1, 2012Marinus Pharmaceuticals, United States Army, University of Antwerp, University of California, Davis
NCT01339689Phase 2Apr 1, 2011Boston University, Duke University, INTRuST, Marinus Pharmaceuticals, United States Army
NCT01002820Phase 2Oct 1, 2009Marinus Pharmaceuticals
NCT00512317Phase 2Jun 1, 2007Marinus Pharmaceuticals
NCT00465517Phase 2Feb 1, 2007Marinus Pharmaceuticals
Showing 20 of 23 trials
Page 1 / 2

Organizations

Research & Development (10)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
Marinus PharmaceuticalsFor profit212132007
Duke UniversityAcademic/Hospital2112011
United States ArmyGovernment2012011
Boston UniversityAcademic/Hospital1012011
Harvard UniversityAcademic/Hospital1112016
INTRuSTAcademic/Hospital1012011
National Institute on Drug Abuse (NIDA)Government1012013
Sponsor GmbHAcademic/Hospital1012015
University of AntwerpAcademic/Hospital1012012
University of California, DavisAcademic/Hospital1012012
10 organizations
Page 1 / 1

Marketing (3)

OrganizationOrg typeRelationshipDate
Marinus PharmaceuticalsFor profitMKTGMar 18, 2022
Marinus PharmaceuticalsFor profitNDA2Mar 18, 2022
Marinus PharmaceuticalsFor profitNDAMar 18, 2022