Posted by Michael Wonder on 06 Feb 2019
FDA grants priority review for Daiichi Sankyo’s new drug application for CSF1R inhibitor pexidartinib for treatment of patients with TGCT, a rare, debilitating tumour
6 February 2019 - If approved by the FDA, pexidartinib would be the first and only approved therapy for tenosynovial giant cell tumour when associated with severe morbidity or functional limitations, and not amenable to improvement with surgery.
Daiichi Sankyo announced that the U.S. FDA has accepted a new drug application and granted priority review for pexidartinib for the treatment of adult patients with symptomatic tenosynovial giant cell tumour (TGCT), which is associated with severe morbidity or functional limitations, and which is not amenable to improvement with surgery. TGCT, also referred to as pigmented villonodular synovitis 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 FDA has designated 3 August 2019 as the PDUFA Action date for this application.
The application 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.
