23 August 2017 - Application based on positive results of Phase III study in adults and adolescents with haemophilia A with inhibitors and interim Phase III results in children.

Genentech announced today that the U.S. FDA has accepted the company’s biologics license application and granted priority review for emicizumab prophylaxis (preventative) as a once-weekly subcutaneous treatment for adults, adolescents and children with haemophilia A with factor VIII inhibitors. Nearly one in three people with haemophilia A develop inhibitors to standard factor VIII replacement therapies, which limits treatment options and increases the risk of life-threatening bleeds and repeated bleeds, particularly in joints, that cause long-term damage.

The application for emicizumab is based on results from the Phase III HAVEN 1 study in adults and adolescents 12 years of age and older, as well as interim results from the Phase III HAVEN 2 study in children younger than 12 years of age. Results from HAVEN 1 were published in The New England Journal of Medicine and results from both studies were presented at the 26th International Society on Thrombosis and Haemostasis Congress in July 2017.

Read Genentech press release