SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today positive results from the Phase III HAVEN 3 study evaluating HEMLIBRA® (emicizumab-kxwh) in adults and adolescents (aged 12 years or older) with hemophilia A without inhibitors to factor VIII. The study met its primary endpoint, showing a statistically significant and clinically meaningful reduction in the number of treated bleeds over time in people receiving HEMLIBRA prophylaxis every week compared to those receiving no prophylaxis. The study also met key secondary endpoints, including a statistically significant and clinically meaningful reduction in the number of treated bleeds over time with HEMLIBRA prophylaxis dosed every two weeks compared to no prophylaxis. Importantly, once-weekly HEMLIBRA prophylaxis was superior to factor VIII prophylaxis, as demonstrated by a statistically significant and clinically meaningful reduction in treated bleeds in an intra-patient comparison of patients receiving HEMLIBRA prophylaxis compared to their prior factor VIII prophylaxis. The most common adverse events with HEMLIBRA were injection site reactions, with no new safety signals observed. No thrombotic microangiopathy or thrombotic events occurred in this study.
“HEMLIBRA is the first product to show superior efficacy to factor VIII prophylaxis. These results in people with hemophilia A without inhibitors represent the next step forward in our clinical trial program, which includes the positive HAVEN 1 and interim HAVEN 2 data in people with inhibitors,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We look forward to working with health authorities to make this treatment available for all people with hemophilia A as soon as possible.”
“It is well established that prophylaxis is the preferred approach for treatment of hemophilia A, but this can require frequent intravenous infusions, and some patients on prophylaxis can still experience bleeds, while others prefer on-demand treatment,” said Johnny Mahlangu, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa. “Given its potential to be dosed through subcutaneous injection only once weekly or every other week, HEMLIBRA may provide a further effective prophylactic treatment option for more people with hemophilia A and help alleviate some of the administration burden associated with current treatment.”
Data from the HAVEN 3 study will be presented at an upcoming medical meeting and submitted to health authorities around the world for approval consideration. These results add to the growing body of evidence shown in the inhibitor population and support that HEMLIBRA may benefit all people with hemophilia A regardless of inhibitor status. Data from the HAVEN 1 and HAVEN 2 studies supported the recent U.S. Food and Drug Administration (FDA) approval of HEMLIBRA for adults and children with hemophilia A with inhibitors, and are being reviewed under accelerated assessment by the European Medicines Agency.
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.
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:
- 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
- numbness in the face
- eye pain or swelling
- trouble seeing
If aPCC (FEIBA®) is needed, patients should talk to their healthcare provider in case they feel they need more than 100 U/kg of aPCC (FEIBA®) total.
How should patients use HEMLIBRA?
HEMLIBRA may interfere with laboratory tests that measure how well blood is clotting and may cause a false reading. Patients should talk to their healthcare provider about how this may affect their care.
What are the other possible side effects of HEMLIBRA?
The most common side effects of HEMLIBRA include: redness, tenderness, warmth, or itching at the site of injection; headache; and joint pain.
These are not all of the possible side effects of HEMLIBRA. Patients should call their doctor for medical advice about side effects.
Side effects may be reported to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Side effects may also be reported to Genentech at (888) 835-2555.
About hemophilia A
Hemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Hemophilia affects around 20,000 people in the United States, with hemophilia A being the most common form and approximately 50-60 percent of people living with a severe form of the disorder.
People with hemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their disorder, people with hemophilia A can bleed frequently, especially into their joints or muscles. These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damage.
About Genentech in hemophilia
In 1984, Genentech scientists were the first to clone recombinant factor VIII in response to the contaminated hemophilia blood supply crisis of the early 1980s. For more than 20 years, Genentech has been developing medicines to bring innovative treatment options to people with diseases of the blood within oncology, and in hemophilia A. Genentech is committed to improving treatment and care in the hemophilia community by delivering meaningful science and clinical expertise. For more information visit http://www.gene.com/hemophilia.
Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.