CDEK
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defactinib

Small moleculeapprovedOrphan Drug FDA

Approved

May 8, 2025

Clinical trial activity

36 trials · 40 clinical orgs · 1 marketing orgs

Phase 1
16
Phase 2
25
Phase 3
1
Phase 4
0

Earliest trial started Dec 1, 2008 (NCT00787033)

Timeline

2000s

  1. Jan 1, 2008

    Verastem, Inc. — Earliest Phase 1 Sponsor(trial)

2010s

  1. Jan 1, 2013

    Verastem, Inc. — Earliest Phase 2 Sponsor(trial)

2020s

  1. Jan 1, 2023

    Verastem, Inc. — Earliest Phase 3 Sponsor(trial)

  2. Mar 5, 2024

    Orphan Drug Designation

  3. May 8, 2025

    Earliest FDA Approval

  4. May 8, 2025

    Verastem, Inc. — NDA Secondary Org

Indications

Approved for

Studied for

Mechanism of action

Combination products

  • avmapki fakzynja co-pack (copackaged)

    with avutometinib

    Also known as avutometinib and defactinib

Approval history

  • approvedPriority reviewMay 8, 2025

Chemistry & pharmacology

Loading structure…

SMILES

CNC(=O)c1ccc(Nc2ncc(C(F)(F)F)c(NCc3nccnc3N(C)S(C)(=O)=O)n2)cc1
Mol. weight
510.5 g/mol
Lipinski Ro5
1 violation(s)
Rule of 3
No
Chirality
Achiral Molecule
Inorganic
No
Polymer
No

Oral

No

Parenteral

No

Topical

No

Sources

Also known as

  • defactinib
  • defactinib
  • defactinib
  • defactinib
  • defactinib
  • defactinib hydrochloride
  • defactinib hydrochloride
  • n-methyl-4-((4-(((3-methyl(methylsulfonyl)aminopyrazin-2-yl)methyl)amino)-5-(trifluoromethyl)pyrimidin-2-yl)amino)benzamide
  • pf-04554878
  • pf-04554878
  • pf-04554878
  • pf-04554878
  • vs-6063
  • vs-6063
  • vs-6063
  • vs-6063

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT07126158Phase 2Nov 30, 2025Verastem, Inc., Washington University in St. Louis
NCT06630260Phase 1/Phase 2 (Phase 2)Nov 4, 2024Cancer Research UK, Minderoo Foundation, National Health Service, United Kingdom, Royal Marsden Hospital NHS Trust, University of Cambridge, University of London, Verastem, Inc.
NCT06369259Phase 2Oct 31, 2024University of Texas at Houston
NCT06682572Phase 2Oct 30, 2024Japanese Gynecologic Oncology Group, Verastem, Inc.
NCT06487221Phase 2Oct 28, 2024Columbia University, Verastem, Inc.
NCT06495125Phase 2Jul 31, 2024Emory University, National Cancer Institute (NCI)
NCT06394804Phase 2Apr 29, 2024Memorial Sloan-Kettering Cancer Center, Verastem, Inc.
NCT06194929Phase 1/Phase 2 (Phase 2)Feb 8, 2024University of Utah, Verastem, Inc.
NCT06072781Phase 3Dec 31, 2023Australia New Zealand Gynaecological Oncology Group, European Network for Gynaecological Oncological Trial, GOG Foundation, Inc., Korean Gynecologic Oncology Group, Verastem, Inc.
NCT06007924Phase 2Aug 16, 2023Memorial Sloan-Kettering Cancer Center, Verastem, Inc.
NCT05798507Early Phase 1 (Phase 1)Jul 28, 2023Emory University, National Cancer Institute (NCI), Verastem, Inc.
NCT05787561Phase 2Mar 15, 2023Memorial Sloan-Kettering Cancer Center, Verastem, Inc.
NCT05669482Phase 1/Phase 2 (Phase 2)Jan 15, 2023Verastem, Inc.
NCT05512208Phase 2Dec 31, 2022University of Oklahoma, Verastem, Inc.
NCT05636514Phase 1Dec 5, 2022Australian National University, Clinical Hub for Interventional Research (CHOIR), University of New South Wales
NCT04720417Phase 2Jan 26, 2021Thomas Jefferson University, Verastem, Inc.
NCT04625270Phase 2Dec 21, 2020European Network for Gynaecological Oncological Trial, GOG Foundation, Inc., Gynecologic Oncology Group, Verastem, Inc.
NCT04620330Phase 2Nov 30, 2020University of Colorado, Denver, Verastem, Inc.
NCT04331041Phase 2Apr 30, 2020Washington University in St. Louis
NCT04201145Phase 1Jan 31, 2020Harvard University, Merck
Showing 20 of 36 trials
Page 1 / 2

Organizations

Research & Development (40)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
Verastem, Inc.For profit281432008
National Cancer Institute (NCI)Government4222015
Memorial Sloan-Kettering Cancer CenterAcademic/Hospital3312023
MerckFor profit3022017
Washington University in St. LouisAcademic/Hospital3322016
Cancer Research UKAcademic/Hospital2012017
ECOG-ACRIN Cancer Research GroupAcademic/Hospital2012015
Emory UniversityAcademic/Hospital2222023
European Network for Gynaecological Oncological TrialAcademic/Hospital2022020
GOG Foundation, Inc.Academic/Hospital2022020
Harvard UniversityAcademic/Hospital2122013
University of LondonAcademic/Hospital2222017
Australia New Zealand Gynaecological Oncology GroupAcademic/Hospital1012023
Australian National UniversityAcademic/Hospital1012022
ChugaiFor profit1012017
Clinical Hub for Interventional Research (CHOIR)Academic/Hospital1112022
Columbia UniversityAcademic/Hospital1112024
Gynecologic Oncology GroupAcademic/Hospital1012020
Japanese Gynecologic Oncology GroupAcademic/Hospital1012024
Johns Hopkins UniversityAcademic/Hospital1112019
40 organizations
Page 1 / 2

Marketing (1)

OrganizationOrg typeRelationshipDate
Verastem, Inc.For profitNDA2May 8, 2025