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Daiichi Sankyo Europe GmbH

Breakthrough Science

Pexidartinib CSF1R Inhibitor

Pexidartinib is an investigational, oral, selective inhibitor of CSF1R in late stage development for tenosynovial giant cell tumor (TGCT), a tumor of the joint or tendon sheath, and previously referred to as pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS). TGCT is a rare, usually non-cancerous tumor that affects the synovium-lined joints, bursae, and tendon sheaths and there is currently no approved therapy for this disease.

Pexidartinib has been granted Breakthrough Therapy Designation for the treatment of patients with PVNS or GCT-TS, where surgical resection may result in potentially worsening functional limitation or severe morbidity and Orphan Drug Designation for PVNS/GCT-TS by the U.S. FDA. Pexidartinib also has received Orphan Drug Designation from EMA for the treatment of TGCT.

Daiichi Sankyo will submit an NDA based upon results of the phase 3 ENLIVEN study to the U.S. FDA for pexidartinib as a treatment for TGCT associated with severe morbidity or functional limitations, and for which surgery is not recommended, and will also engage European regulators regarding review of pexidartinib.

These are investigational agents and have not been approved by the EMA, FDA or any other worldwide regulatory agency as a treatment for any indication. Safety and efficacy have not been established.