FDA Breakthrough designation for Daiichi Sankyo's pexidartinib

The Food and Drug Administration has granted Breakthrough Therapy designation to Japanese drug major Daiichi Sankyo (TYO: 4568) and its US subsidiary Plexxikon’s investigational oral CSF-1R inhibitor pexidartinib (formerly PLX3397) for the treatment of tenosynovial giant cell tumor (TGCT) where surgical removal of the tumor would be associated with potentially worsening functional limitation or severe morbidity.

Daiichi Sankyo’s gained 2.2% to 2,384 yen on Friday following the announcement.

"Surgery is the primary treatment for TGCT, but for patients with a diffuse form of the condition, the tumor is more difficult to remove and has a high rate of recurrence, resulting in multiple complicated surgeries and even amputation in some patients," said Mahmoud Ghazzi, executive vice president and global head of development for Daiichi Sankyo. "We are pleased that the FDA recognizes the unmet need for the treatment of TGCT and we look forward to working closely with the Agency on the expedited development of this potential non-surgical treatment for patients with TGCT," he added.