FDA accepts Hemlibra sBLA for hemophilia A patients without factor VIII inhibitors for priority review

The US Food and Drug Administration has accepted the supplemental Biologics License Application (sBLA) and granted Priority Review for Hemlibra (emicizumab-kxwh) for adults and children with hemophilia A without factor VIII inhibitors, an indication that would attract a much wider patient population than currently available.

The sBLA, filed by Roche (ROG: SIX) subsidiary Genentech, is based on data from the Phase III HAVEN 3 study. The FDA is expected to make a decision on approval by October 4, 2018. Results from the HAVEN 3 study were presented at the World Federation of Hemophilia (WFH) 2018 World Congress in May and at the time wowed investors and analysts.

Analysts at Jeffries have predicted that Hemlibra could generate $5 billion in annual sales – provided its use for hemophilia A is widened to the larger, non-inhibitor population. However, other analysts forecast a more modest $2 billion in peak revenues.