Hemophilia A, also called factor VIII (FVIII) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective clotting protein known as factor VIII. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation in a gene, according to the National Hemophilia Foundation.
According to the Centers for Disease Control and Prevention, hemophilia occurs in approximately 1 in 5,000 live births and affects all races and ethnic groups. There are about 20,000 people with hemophilia in the US. Hemophilia A is four times as common as hemophilia B. Patients may experience repeated episodes of serious bleeding, primarily into their joints, which can be severely damaged as a result. Some patients develop an immune response known as a FVIII inhibitor or antibody. The antibody interferes with the effectiveness of currently available treatments for hemophilia.
Yesterday the FDA approved a medication called Hemlibra as the first newly approved medicine in nearly 20 years to treat hemophilia A with inhibitors. It’s the only treatment option that can be self-administered once-weekly by subcutaneous injection. Approval was granted to Genentech, part of the Roche (OTCQX: RHHBY) group of companies. Hemlibra was created by Chugai Pharmaceutical (OTC: CHGCY) and is being co-developed by Chugai, Roche and Genentech.
Today’s approval of Hemlibra represents an important advancement for people with hemophilia A with inhibitors, who have struggled to manage their bleeding disorder and haven’t had a new medicine in almost 20 years. We believe Hemlibra will improve protection against bleeds and reduce the treatment administration burden for people with hemophilia A with inhibitors, and we are committed to helping them access this medicine.
– Sandra Horning, MD, Chief Medical Officer and head of Global Product Development, Genentech
Hemlibra therapy works by bridging other Factors in the blood to restore blood clotting for these patients. The safety and efficacy of Hemlibra was based on data from two clinical trials. The first was a trial that included 109 males aged 12 and older with hemophilia A with FVIII inhibitors. The randomized portion of the trial compared Hemlibra to no prophylactic treatment in 53 patients who were previously treated with on-demand therapy with a bypassing agent before enrolling in the trial. Patients taking Hemlibra experienced approximately 2.9 treated bleeding episodes per year compared to approximately 23.3 treated bleeding episodes per year for patients who did not receive prophylactic treatment. This represents an 87% reduction in the rate of treated bleeds.
The second trial was a single arm trial of 23 males under the age of 12 with hemophilia A with FVIII inhibitors. During the trial, again, 87% of the patients taking Hemlibra did not experience a bleeding episode that required treatment.
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. 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.
– Guy Young, MD, director of Hemostasis and Thrombosis Program, Children’s Hospital Los Angeles, and professor of Pediatrics, University of Southern California Keck School of Medicine
Hemlibra was reviewed by the FDA under Priority Review and granted Breakthrough Therapy Designation in people 12 years of age or older with hemophilia A with inhibitors in September 2015. Clinical data are being reviewed under accelerated assessment by the European Medicines Agency (EMA) and submissions to health authorities around the world are ongoing.
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