Emicizumab BLA gets Priority Review for hemophilia A

The Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHHBY) said FDA accepted and granted Priority Review to a BLA for emicizumab (ACE910) as a once-weekly subcutaneous preventative treatment for children, adolescents and adults with hemophilia A with Factor VIII inhibitors. Its PDUFA date is Feb. 23, 2018.

The BLA is based on data from the Phase III HAVEN 1 and HAVEN 2 trials. In HAVEN 1, emicizumab as prophylaxis met the primary endpoint of reducing annualized bleeding rate vs. no prophylaxis, on-demand treatment with bypassing agents (2.9 vs. 23.3 treated bleeds, p<0.0001). Roche reported interim data from the pediatric HAVEN 2 trial in June (see BioCentury Extra, June 26)...