Rare Disease Report

Genentech's Hemophilia A Data Presented at 2016 WFH

JULY 27, 2016
Andrew Black
Exciting data was presented at the 2016 World Federation of Hemophilia in Orlando, Florida last week on Genentech’s hemophilia treatment drug, emicizumab. In the Phase 1/2 trial of emicizumab, the drug continued to demonstrate promising safety and prophylactic efficacy for people with severe hemophilia A, with or without inhibitors to factor VIII.
The Phase 1/2trial assessed emicizumab in an extension of a 21 to 32-month period which included Japanese patients with severe hemophilia A ages 12 to 58 years that were organized in three different dosing groups receiving 0.3 mg, 1mg, or 3mg of emicizumab. 
Emicizumab successfully reduced bleeding events by more than 95% in each group that was administered the drug. The successfulness of the Phase 1/2 trial in the treatment of hemophilia showed progression of the drug which excited Genentech.
The next step is a Phase 3 trial that is currently underway. The trial will be an open-label study will evaluate the safety, efficacy and pharmacokinetics of prophylactic emicizumab treatment in patients previously treated with episodic or prophylactic bypassing agents. Phase III will enroll patients 12 years of age or older with hemophilia A with factor VIII inhibitors. Genentech also plans for additional pivotal studies that are planned to commence soon.


Emicizumab is an investigational humanized, bispecific monoclonal antibody. It is designed to support the interaction between factors IXa and X which are proteins that work together in the blood clotting process, thought to restore clotting function and thereby prevent spontaneous bleeding. Emicizumab can be administered subcutaneously once a week and can stay in the body for four to five weeks, which could allow for weekly or potentially less frequent administration.

Hemophilia A

Hemophilia A is a rare, chronic, genetic disorder that results in impaired clotting mechanisms due to missing or reduced levels of factor VIII. People with hemophilia A experience recurrent and extended bleeding episodes that cause pain and irreversible joint damage. Some of these bleeding episodes can be life threatening. There are an estimated 16,000 – 20,000 people in the United States with hemophilia A (and 142,000 world wide).

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