FDA Grants Breakthrough Therapy Designation for Genentech's Hemlibra

The following is an excerpt from a press release from Genentech. Read the full press release here. 

Genentech, a member of the Roche Group, announced today the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to HEMLIBRA for people with hemophilia A without factor VIII inhibitors. Breakthrough Therapy Designation is designed to accelerate the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies.
This designation is based on data from the Phase III HAVEN 3 study in people 12 years or older with hemophilia A without inhibitors. In the study, HEMLIBRA prophylaxis dosed subcutaneously every week or every two weeks showed a statistically significant and clinically meaningful reduction in treated bleeds compared to no prophylaxis. In an intra-patient comparison, once-weekly HEMLIBRA prophylaxis was superior to prior factor VIII prophylaxis as demonstrated by a statistically significant and clinically meaningful reduction in treated bleeds. The most common adverse events with HEMLIBRA were injection site reactions, and no new safety signals were observed. No thrombotic microangiopathy or thrombotic events occurred in this study.
Read the entire press release here.