FDA Grants Breakthrough Therapy Designation for Genentech?s HEMLIBRA (emicizumab-kxwh) in Hemophilia a Without Inhibitors
? Designation based on Phase III HAVEN 3 study demonstrating HEMLIBRA prophylaxis significantly reduced bleeds compared to no prophylaxis ?
? First medicine to show superior efficacy compared to prior factor VIII prophylaxis in an intra-patient comparison ?
April 17, 2018 01:00 AM Eastern Daylight Time
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to HEMLIBRA??(emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Breakthrough Therapy Designation is designed to accelerate the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies.
?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 comparison,? said Sandra Horning, M.D., chief medical officer and head of Global Product Development. ?We look forward to working with health authorities to make HEMLIBRA available to people without inhibitors as soon as possible, and we are excited to share this news with the community as we join in celebrating World Hemophilia Day.?
This designation is based on data from the Phase III HAVEN 3 study in people 12 years or older with hemophilia A without inhibitors. In the study, HEMLIBRA prophylaxis dosed subcutaneously every week or every two weeks showed a statistically significant and clinically meaningful reduction in treated bleeds compared to no prophylaxis. In an intra-patient comparison, once-weekly HEMLIBRA prophylaxis was superior to prior factor VIII prophylaxis as demonstrated by a statistically significant and clinically meaningful reduction in treated bleeds. The most common adverse events with HEMLIBRA were injection site reactions, and no new safety signals were observed. No thrombotic microangiopathy or thrombotic events occurred in this study.
HEMLIBRA was granted its first 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 based on results from the HAVEN 1 and HAVEN 2 studies.
The HEMLIBRA development program reflects Genentech?s commitment to address clinical unmet needs in the hemophilia A community. Roche and Genentech are proud to support the World Federation of Hemophilia and the global bleeding disorders community as sponsors of World Hemophilia Day. To learn more about World Hemophilia Day and the World Federation of Hemophilia visit?http://www.wfh.org/en/whd.
About HAVEN 3 (NCT02847637)
HAVEN 3 is a randomized, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of HEMLIBRA prophylaxis versus no prophylaxis (episodic/on-demand factor VIII treatment) in people with hemophilia A without inhibitors to factor VIII. The study included 152 patients with hemophilia A (12 years of age or older) who were previously treated with factor VIII therapy either on-demand or for prophylaxis. Patients previously treated with on-demand factor VIII were randomized in a 2:2:1 fashion to receive subcutaneous HEMLIBRA prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study (Arm A), subcutaneous HEMLIBRA prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 3 mg/kg/2wks until the end of study (Arm B), or no prophylaxis (Arm C). Patients previously treated with factor VIII prophylaxis received subcutaneous HEMLIBRA prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study (Arm D). Episodic treatment of breakthrough bleeds with factor VIII therapy was allowed per protocol.
About HEMLIBRA
HEMLIBRA is a bispecific factor IXa- and factor X-directed antibody. It is 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. HEMLIBRA is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once weekly. HEMLIBRA was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed by Chugai, Roche and Genentech.
HEMLIBRA U.S. Indication
HEMLIBRA is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia?A with factor VIII inhibitors.
Important Safety Information
HEMLIBRA increases the potential for blood to clot. Discontinue prophylactic use of bypassing agents the day before starting HEMLIBRA prophylaxis. Carefully follow the healthcare provider?s instructions regarding when to use an on-demand bypassing agent, and the dose and schedule one should use. Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was administered for 24 hours or more to patients receiving HEMLIBRA prophylaxis.
HEMLIBRA may cause the following serious side effects when used with aPCC (FEIBA?), including:
- Thrombotic microangiopathy (TMA).?This is a condition involving blood clots and injury to small blood vessels that may cause harm to one?s kidneys, brain, and other organs. Patients should get medical help right away if they have any of the following signs or symptoms during or after treatment with HEMLIBRA:
- confusion
- weakness
- swelling of arms and legs
- yellowing of skin and eyes
- stomach (abdomen) or back pain
- nausea or vomiting
- feeling sick
- decreased urination
- Blood clots (thrombotic events).?Blood clots may form in blood vessels in one?s arm, leg, lung or head. Patients should get medical help right away if they have any of these signs or symptoms of blood clots during or after treatment with HEMLIBRA:
- swelling in arms or legs
- pain or redness in the arms or legs
- shortness of breath
- chest pain or tightness
- fast heart rate
- cough up blood
- feel faint
- headache
- numbness in the face
- eye pain or swelling
- trouble seeing
- are pregnant or plan to become pregnant. It is not known if HEMLIBRA may harm their unborn baby. Females who are able to become pregnant should use birth control (contraception) during treatment with HEMLIBRA.
- are breastfeeding or plan to breastfeed. It is not known if HEMLIBRA passes into a female?s breast milk.