Passion for Innovation. Compassion for Patients.™

Daiichi Sankyo Nordics ApS c/o

Investigational Breakthrough Science Franchise

The investigational Breakthrough Science pipeline of the Daiichi Sankyo Cancer Enterprise aims to leverage our world-class science to research and develop innovative therapies with new technologies through internal discovery and active partnership.

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 Priority Review and 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.

Pexidartinib is currently under regulatory review in the U.S. and EU 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.


For legal reasons detailed information about prescription medicine may only be given to healthcare professionals. Pexidartinib is an investigational agent that has only been approved for indication in the US so far. Safety and efficacy have not been established outside the US. All other agents discussed on the website are investigational and have not been approved by the EMA or any other regulatory agency worldwide as a treatment for any indication. Safety and efficacy have not been established. There is no guarantee that these compounds will become commercially available.