Drug Profile

Defactinib - Verastem Oncology

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

Latest Information Update: 26 Aug 2025

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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; Mesothelioma; Pancreatic cancer
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

30 May 2025 Updated adverse events and efficacy data from the phase-Ib/II RAMP205 trial in Adenocarcinoma presented at the 61st Annual Meeting of the American Society of Clinical Oncology (ASCO-2025)
22 May 2025 Efficacy and adverse events data from a phase III RAMP 205 trial in Pancreatic cancer released by Verastem Oncology
22 May 2025 Verastem Oncology plans a phase III trial for Pancreatic cancer (Late stage disease, Metastatic disease, First line therapy) in USA in 2026

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