Daiichi gets ODAC split, with FDA panel backing pexidartinib for rare cancer

Missing data and lingering safety questions dogged a pair of Daiichi Sankyo oncology agents that came before ODAC on Tuesday, but a lack of available treatment options for tenosynovial giant cell tumor and a clear win on the primary endpoint for pexidartinib swayed the FDA panel in favor of approval 12-3. Quizartinib, to treat relapsed, refractory FLT3-ITD-positive AML, did not fare as well, with the Oncologic Drugs Advisory Committee snubbing the compound with a 3-8 vote.

Pexidartinib from Daiichi Sankyo Co. Ltd. (Tokyo:4568) met the primary endpoint of improving overall response rates at 39% vs. 0% for placebo in the Phase III ENLIVEN trial of 120 patients with giant cell tumor of the tendon sheath (p<0.0001). However, at the meeting and in briefing materials, FDA raised concerns over the high rate of liver toxicity seen in the trial as well as the large amount of missing data for three of the four key secondary endpoints. ...