CSF 1R filed with EMA for symptomatic tenosynovial giant cell tumor

Daiichi Sankyo announced that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for CSF 1R (pexidartinib) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT), which is associated with severe morbidity or functional limitations, and which is not amenable to improvement with surgery. TGCT is also referred to as pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS). Validation confirms that the application is complete and commences the scientific review process by the EMA's Committee for Medicinal Products for Human Use (CHMP).

The EU MAA is based on results of the pivotal phase III ENLIVEN study of oral pexidartinib, the first placebo-controlled study of a systemic investigational therapy in patients with TGCT, which met its primary endpoint of overall response rate. Results of the phase III ENLIVEN study were presented during an oral presentation at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting.