Daiichi Sankyo, Inc. and Plexxikon Inc., a member of the Daiichi Sankyo Group, announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to its investigational oral CSF-1R inhibitor pexidartinib (formerly PLX3397) for the treatment of tenosynovial giant cell tumour (TGCT) where surgical removal of the tumour would be associated with potentially worsening functional limitation or severe morbidity.
Breakthrough Therapy designation is designed to expedite the development and review of medicines that may demonstrate substantial benefit over currently available treatments in order to ensure that patients with serious diseases have access to new treatments as soon as possible. Currently, there is no FDA-approved systemic therapy for the treatment of TGCT.
The Breakthrough Therapy designation was granted based on results from an extension cohort of a single-arm, multi-center phase 1 study that assessed the safety and efficacy of pexidartinib. Results of this study were published in the July 30, 2015 issue of The New England Journal of Medicine.