Defactinib Designated Orphan Drug for Mesothelioma

Verastem announced that the FDA has granted its lead stem cell inhibitor, defactinib (VS-6063) Orphan Drug Designation for use in the treatment of mesothelioma, a rare form of lung cancer. VS-6063 is an oral small molecule inhibitor of focal adhesion kinase (FAK), which is a critical pathway for cancer stem cells. 

Verastem recently outlined details of the registration-directed clinical study of defactinib in patients with malignant pleural mesothelioma. This study is designed as a double-blind, placebo-controlled trial with an expected enrollment of approximately 350–400 patients at clinical sites in 11 countries.

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