Genentech, a member of Roche Group, and Chugai Pharmaceutical announced the FDA approval of Hemlibra (U.S. generic name: emicizumab-kxwh), a treatment for hemophilia A created by Chugai, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors. Hemlibra is now the only prophylactic treatment for people with hemophilia A with and without factor VIII inhibitors that can be administered subcutaneously (under the skin) and at multiple dosing options (once weekly, every two weeks, or every four weeks).
The U.S. application was submitted by Genentech.
“Many preventative treatment options for people with hemophilia A without factor VIII inhibitors require intravenous infusions several times a week. Even then, people can still experience bleeds, and there has been a need for more treatment options,” said Michael Callaghan, M.D., hematologist, Children’s Hospital of Michigan. “The approval of Hemlibra is an important advancement for the entire hemophilia A community, as we now have a new class of medicine for the first time in nearly 20 years.
This regulatory approval is based on results from two Phase lll studies HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160), conducted jointly with Roche and Genentech. In the Phase III HAVEN 3 study, adults and adolescents aged 12 years or older with hemophilia A without factor VIII inhibitors who received Hemlibra prophylaxis once weekly (n=36) or every two weeks (n=35) experienced a 96 percent (95 percent CI: 92.5; 98.0, p<0.0001) and 97 percent (95 percent CI: 93.4; 98.3, p<0.0001) reduction in treated bleeds, respectively, compared to those who received no prophylaxis (n=18).
Hemlibra is the first medicine to significantly reduce treated bleeds compared to prior factor VIII prophylaxis, which has been the recommended standard of care, as demonstrated by a statistically significant reduction of 68 percent (95 percent CI: 48.6; 80.5, p<0.0001) in treated bleeds in a prospective intra-patient comparison (n=48) of people who previously received factor VIII prophylaxis in a non-interventional study and switched to Hemlibra prophylaxis. In the single-arm Phase III HAVEN 4 study of adults and adolescents aged 12 years or older with hemophilia A with factor VIII inhibitors (n=5) and without factor VIII inhibitors (n=36), Hemlibra prophylaxis every four weeks (n=41) led to clinically meaningful control of bleeding. The most common adverse reactions occurring in 10 percent or more of people treated with Hemlibra in pooled studies (n=391) were injection site reactions (n=85), headache (n=57) and joint pain (arthralgia; n=59).
Hemlibra was granted Priority Review and Breakthrough Therapy Designation by the FDA in hemophilia A without inhibitors, following the prior designations in hemophilia A with inhibitors. Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the safety and effectiveness of the treatment, prevention or diagnosis of a serious disease.
In Japan and the EU, applications have been filed to regulatory authorities and are currently under review for an additional indication of prophylactic treatment for people with hemophilia A without inhibitors, as well as for additional dosage and administration as a biweekly or every four-week treatment for people with hemophilia A with inhibitors to factor VIII.