Daiichi Sankyo's cancer drug wins the FDA's coveted 'breakthrough' tag

Daiichi Sankyo CEO Joji Nakayama

Daiichi Sankyo picked up the FDA's breakthrough therapy designation for a Phase III cancer drug, putting it in line for preferential access to agency experts as it moves toward approval.

The treatment, pexidartinib, is in development to treat a rare cancer of the tendons called tenosynovial giant cell tumor, or TGCT. Such tumors are commonly removed through surgery, but diffuse forms of TGCT ensnarl themselves in bone and joint tissue, creating the need for therapeutic intervention. In March, Daiichi began a Phase III trial on pexidartinib, enrolling 126 TGCT patients to test how well the drug can shrink tumors and improve range of motion.

The FDA's breakthrough award is based on Phase I data in which pexidartinib demonstrated proof of concept in TGCT with enough evidence to skip straight to Phase III, Daiichi said. The designation grants Daiichi a direct route to key FDA decisionmakers and frequent feedback throughout Phase III, making the drug eligible for priority review when the company gets its submission together.

Pexidartinib, formerly PLX3397, is a product of Plexxikon, which Daiichi acquired in a $935 million deal in 2011. Plexxikon made its name with the melanoma drug vemurafenib, approved that same year under the trade name Zelboraf and partnered with Roche ($RHHBY) under a 2006 alliance.

Meanwhile, Daiichi is upending its U.S. operation in an effort to cut costs, planning to lay off about 1,200 stateside workers as it shifts its focus from primary care drugs to specialty pharma. The company's latest restructuring, announced this month, is designed to absorb the blow of patent losses for the blood pressure drug Benicar and diabetes treatment Welchol.

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