Genentech announced that its Phase III HAVEN 3 study of Hemlibra in adults and adolescents with hemophilia A without inhibitors to factor VIII had positive results.
Genentech, a Roche company, announced that its Phase III HAVEN 3 study of Hemlibra (emicizumab-kxwh) in adults and adolescents with hemophilia A without inhibitors to factor VIII had positive results.
The study’s primary endpoint was statistical significance and clinically meaningful reduction in the number of treated bleeds over time in patients receiving the drug every week compared to those not receiving prophylaxis. It also met key secondary endpoints, including a statistically significant and clinically meaningful cut in the number of treated bleeds over time with Hemlibra prophylaxis when it was dosed every two weeks compared to no prophylaxis.
The company also emphasized that the once-weekly Hemlibra prophylaxis was superior to factor VIII prophylaxis.
Hemophilia A is an inherited disorder where an individual’s blood doesn’t clot properly. This can lead to uncontrolled and often spontaneous bleeding. It affects about 20,000 people in the U.S., with hemophilia A being the most common form. Affected individuals either have no or not enough of a clotting protein called factor VIII. Some people develop resistance to factor VIII treatment, which is called “inhibitors.” Some people do not, which is the population the HAVEN 3 study evaluated.
Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is engineered to bring together factor IXa and factor X, which are proteins needed to activate the natural coagulation cascade and restore blood clotting to normal. Hemlibra is a preventative (prophylactic) treatment. It was created by Chugai Pharmaceutical Co., and is co-developed by Chugai, Roche and Genentech.
“Hemlibra is the first product to show superior efficacy to factor VIII prophylaxis,” said Sandra Horning, Genentech’s chief medical officer and head of Global Product Development, in a statement. “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. We look forward to working with health authorities to make this treatment available for all people with hemophilia A as soon as possible.”
On Nov. 16, the U.S. Food and Drug Administration (FDA) approved Hemlibra for routine prophylaxis to prevent or reduce the frequency of bleeding events in adults and children with hemophilia A with factor VIII inhibitors. About a third of patients with hemophilia A that is severe develop inhibitors to replacement therapies that use factor VIII, which increases their risk of life-threatening bleeds or repeated events that can result in long-term joint damage. In clinical trials, HAVEN 1 and Haven 2, Hemlibra showed it could substantially cut bleeds in adults and children in this subpopulation of the disease.
“People with hemophilia A who develop inhibitors face significant challenges preventing bleeds and typically require infusions of medicine multiple times a week, which can be especially difficult for young children and their families,” said Guy Young, director of Hemostasis and Thrombosis Program at Children’s Hospital Los Angeles, in a statement. “This new medicine has been shown to reduce the frequency of bleeds compared to the currently available medicines and only needs to be injected once a week. This could make a meaningful difference for these children.”
Genentech plans to release data from the HAVEN 3 study at a medical meeting in the near-future as well as submit it to global regulatory agencies for approval.
