Pexidartinib to treat TGCT gets priority reviewApril 9, 2019
Daiichi Sankyo said pexidartinib for the treatment of tenosynovial giant cell tumour (TGCT) has been granted priority review by US FDA.
TGCT, also referred to as pigmented villonodular synovitis (PVNS) or giant cell tumour of the tendon sheath (GCT-TS), is a non-malignant tumour of the joint or tendon sheath, which can be locally aggressive and debilitating in some patients. There are no currently approved systemic therapies for TGCT.
The NDA is based on results of the pivotal phase 3 ENLIVEN study of oral pexidartinib, the first placebo-controlled study of a systemic investigational therapy in patients with TGCT.
Pexidartinib is an investigational oral small molecule that potently inhibits CSF1R (colony stimulating factor-1 receptor), which is a primary growth driver of abnormal cells in the synovium that cause TGCT. Pexidartinib also inhibits c-kit and FLT3-ITD.