Genentech, a member of the Roche Group, announced the U.S. Food and Drug Administration has accepted the company’s supplemental Biologics License Application and granted Priority Review for HEMLIBRA Â 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 byÂ Oct. 4, 2018.
“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 and 97 percent reduction in treated bleeds, respectively, compared to those who received no prophylaxis. In an additional arm of the study, people who had previously received factor VIII prophylaxis in a non-interventional study switched to HEMLIBRA prophylaxis, allowing for an intra-patient comparison of two prophylaxis regimens. Based on the intra-patient comparison, HEMLIBRA demonstrated a statistically significant reduction of 68 percent in treated bleeds, making it the first medicine to show superior efficacy to prior treatment with factor VIII prophylaxis, the standard of care. There were no unexpected or serious adverse events (AEs) related to HEMLIBRA in the HAVEN 3 study, and the most common AEs were consistent with previous studies. The most common AEs occurring in 5 percent or more of people in the HAVEN 3 study were injection site reactions, joint pain (arthralgia), common cold symptoms (nasopharyngitis), headache, upper respiratory tract infection and influenza. Results from the HAVEN 3 study were presented at the World Federation of Hemophilia (WFH) 2018 World Congress in May.
Source: Genentech Press Release