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FDA Accepts License Application and Grants Priority Review for Emicizumab

September 8, 2017
FDA Accepts License Application and Grants Priority Review for Emicizumab

The U.S. Food and Drug Administration has accepted the Biologics License Application (BLA) and granted priority review for emicizumab, as a once-weekly treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors. Emicizumab, which is being co-developed by Chugai, Roche and Genentech, is unlike existing treatments in that it is administered subcutaneously via an injection just under the skin, instead of intravenously.

Emicizumab is a laboratory-engineered protein that works by performing a key function in the clotting cascade that is normally carried out by the FVIII protein, which is deficient in individuals with hemophilia A. The “cascade” is an intricate series of chemical and molecular reactions between clotting factors that lead to clot formation. In this case, emicizumab binds to and bridges two other key clotting proteins, activated factor IX and factor X, important components of the cascade normally performed by FVIII.

The BLA for emicizumab is based on positive results from the Phase III HAVEN 1 study and interim results from the Phase III HAVEN 2 study, which were presented at the 26th International Society on Thrombosis and Haemostasis Meeting, July 10, 2017, in Berlin, Germany.

“Genentech has a history of developing innovative antibody therapies to address some of the highest unmet medical needs,” said Sandra Horning, MD, chief medical officer and head of Global Product Development. “Results of our Phase III study in adults and adolescents as well as early Phase III results in children showed that emicizumab has significant potential to help people with hemophilia A with inhibitors, who face major challenges in preventing and treating bleeds. We are working with the FDA to hopefully bring this new prophylactic treatment option to the hemophilia A inhibitor community as soon as possible.”

Source: Genentech press release dated August 23, 2017