Drug Profile

Defactinib - Verastem Oncology

Alternative Names: AVMAPKI FAKZYNJA CO-PACK; Defactinib hydrochloride; FAKZYNJA™; PF-04554878; PF-4554878; VS-6063

Latest Information Update: 28 Aug 2026

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At a glance

Originator Pfizer
Developer Cancer Research UK; National Cancer Institute (USA); University of Utah; Verastem; Verastem Oncology
Class Amines; Antineoplastics; Benzamides; Fluorinated hydrocarbons; Pyrazines; Pyrimidines; Small molecules; Sulfones
Mechanism of Action Focal adhesion protein tyrosine kinase inhibitors

Orphan Drug Status

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

Yes - Ovarian cancer; Pancreatic cancer; Mesothelioma
New Molecular Entity Yes

Marketed Ovarian cancer
Phase II Adenocarcinoma; Gynaecological cancer; Lymphoma; Multiple myeloma; Non-small cell lung cancer; Solid tumours; Thyroid cancer; Uveal melanoma
Phase I/II Glioblastoma; Malignant melanoma
Discontinued Breast cancer; Cancer; Malignant-mesothelioma

28 Aug 2026 No recent reports of development identified for phase-0 development in Glioblastoma(Monotherapy, Recurrent) in USA (PO)
31 Jul 2026 University of California in collaboration with Verastem terminates phase-I/II clinical trials in Ovarian cancer (Combination therapy, Second-line therapy or greater) in USA (PO) due to loss of funding (NCT03287271)
17 Jun 2026 Efficacy and adverse events data from the phase-Ib/II RAMP205 trial in Adenocarcinoma released by Verastem Oncology

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