FDA Grants Priority Review to Genentech’s HEMLIBRA (emicizumab-kxwh) for People with Hemophilia A without Factor VIII Inhibitors

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) and granted Priority Review for HEMLIBRA^® (emicizumab-kxwh) for adults and children with hemophilia A without factor VIII inhibitors. The sBLA 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.

As quoted in the press release:

“People with hemophilia A can face significant challenges in managing their condition and may need to adapt their daily lives to avoid bleeds and accommodate treatment,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We believe the FDA’s decision to grant Priority Review to HEMLIBRA underscores its potential to improve the standard of care for people without factor VIII inhibitors and to help reduce treatment burden by offering more flexible subcutaneous dosing options. We look forward to working with the FDA to hopefully bring HEMLIBRA to all people with hemophilia A as quickly as possible.”

In the HAVEN 3 study, adults and adolescents aged 12 years or older with hemophilia A without factor VIII inhibitors who received HEMLIBRA prophylaxis every week or every two weeks showed a 96 percent (p<0.0001) and 97 percent (p<0.0001) reduction in treated bleeds, respectively, compared to those who received no prophylaxis.

Click here to read the full press release.