US FDA Sought Patient Viewpoint On Hemlibra's Benefit/Risk Profile

27 Jan 2018

Hemophilia patient advocate expressed concerns to FDA about ability of hemophilia treatment centers to educate patients about thrombotic microangiopathy and thromboembolic events, unexpected but serious cases of which were seen in the clinical program.

The US FDA consulted with a hemophilia patient advocate about the benefit/risk profile of Genentech Inc.'s Hemlibra (emicizumab-kxwh) in light of serious cases of thrombotic microangiopathy and thromboembolic events that had temporarily halted clinical trial enrollment.

Although the patient advocate – Christopher Templin, a special government employee who has participated in FDA advisory committee meetings –...