SOUTH SAN FRANCISCO (dpa-AFX) - Genentech, a member of the Roche Group (RHHBY), said that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to HEMLIBRA or emicizumab-kxwh 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.
The 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.
HEMLIBRA was granted its first Breakthrough Therapy Designation in September 2015 and was approved by the FDA in November 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors based on results from the HAVEN 1 and HAVEN 2 studies.
Copyright RTT News/dpa-AFX
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