FDA Grants Priority Review to Genentech’s Hemlibra

Rare Daily Staff

The U.S. Food and Drug Administration granted Genentech’s Hemlibra Priority Review for the company’s supplemental biologics license application for adults and children with hemophilia A without factor VIII inhibitors, a rare, genetic clotting disorder.

Hemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. People with the condition either lack or do not have enough of a clotting protein called factor VIII. 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.

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.

Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease. The FDA is expected to decide on approval by October 4, 2018.

“People with hemophilia A can face significant challenges in managing their condition and may need to adapt their daily lives to avoid bleeds and accommodate treatment,” said Sandra Horning, chief medical officer and head of Global Product Development. “We believe the FDA’s decision to grant Priority Review to Hemlibra underscores its potential to improve the standard of care for people without factor VIII inhibitors and to help reduce treatment burden by offering more flexible subcutaneous dosing options.”

The application is supported by the company’s HAVEN 3 study, which included adults and adolescents aged 12 years or older with hemophilia A without factor VIII inhibitors who received Hemlibra prophylaxis. It showed a 96 percent in reduction in treated bleeds, in patients who received Hemlibra every week and a 97 percent reduction in patients who received Hemlibra every two weeks, compared to those who received no prophylaxis.

Hemlibra 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. Hemlibra was also recently approved by regulatory authorities in other countries around the world, including by the European Commission in February 2018 for routine prophylaxis of bleeding episodes in people with hemophilia A with factor VIII inhibitors.

June 6, 2018
Photo: Sandra Horning, chief medical officer and head of Global Product Development