FDA Grants Breakthrough Therapy Designation to Hemlibra for Hemophilia A Without Inhibitors

Emicizumab-kxwh (Hemlibra, Genentech) is the first medication to show superior efficacy compared with prior factor VIII prophylaxis in intra-patient comparisons.

Officials with the FDA have granted breakthrough therapy designation to emicizumab-kxwh (Hemlibra, Genentech) for individuals with hemophilia A without factor VIII inhibitors, according to a press release.

Emicizumab-kxwh was previously granted 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.

The latest designation is based on data from a phase 3 HAVEN 3 study, which included 152 patients 12 years or older with hemophilia A who were previously treated with factor VIII therapy either on-demand or for prophylaxis. The HAVEN 3 study evaluated the efficacy, safety, and pharmacokinetics of emicizumab-kxwh prophylaxis versus no prophylaxis.

In the study, patients received emicizumab-kxwh prophylaxis dosed subcutaneously every week or every 2 weeks. Those in the prophylaxis group showed a statistically significant and clinically meaningful reduction in treated bleeds compared with the no prophylaxis group. Once-weekly emicizumab-kxwh prophylaxis was superior to prior factor VIII prophylaxis.

“Hemlibra is the first medicine to show superior efficacy compared to factor VIII prophylaxis, the standard of care for people with hemophilia A without inhibitors, in an intra-patient comparisons,” Sandra Horning, MD, chief medical officer and head of Global Product Development, said in the press release.

Emicizumab-kxwh is a bispecific factor IXa- and factor X-directed antibody designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for hemophilia A patients, according to the press release.

The most common adverse events associated with emicizumab-kxwh were injection site reactions and no new safety signals were observed.


FDA Grants Breakthrough Therapy Designation For Genentech’s HEMLIBRA (emicizumab-kxwh) in Hemophilia A Without Inhibitors [news release]. Genentech’s website. https://www.gene.com/media/press-releases/14713/2018-04-16/fda-grants-breakthrough-therapy-designat. Accessed April 18, 2018.