FDA grants priority review to hemophilia drug

FDA grants priority review to emicizumab for hemophilia A

FDA grants priority review to emicizumab for hemophilia A

The FDA is expected to make a decision on approval by February 23, 2018, Roche said, adding that almost a third of hemophilia A patients developed inhibitors limiting treatment options and increasing the risk of life-threatening bleeds. It binds the proteins factor IXa and factor X together to activate the natural coagulation cascade and restore the body's normal blood clotting process.

Granting priority review means the FDA will make a decision on approval within six months.

The BLA 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.

Emicizumab, a once weekly, ready-to-use solution for SC injection, is an investigational bispecific monoclonal antibody created to bring together factors IXa and X, proteins required to activate the natural coagulation cascade and restore the blood clotting process.

Shire also claims that Roche has been misleadingly offering a look at "treated bleeds" - a secondary endpoint - instead of the "number of bleeds over time", originally laid out as the primary endpoint in HAVEN 1. Patients previously treated with prophylactic bypassing agents received emicizumab prophylaxis. The median age was 28 years.

Results of the study revealed that patients taking once-weekly emicizumab prophylaxis had annualized bleeding rate of 2.9 events compared to 23.3 events in patients not receiving prophylactic treatment. There are still studies ongoing that are evaluating the drug in haemophilia A patients both with and without inhibitors. HAVEN 1 was a randomized, multicenter, open-label trial evaluating the efficacy, safety and pharmacokinetics of emicizumab prophylaxis compared to on-demand bypassing agents (BPAs) [no prophylaxis; episodic use only] in adults and adolescents ≥12yrs old. The most common adverse events were local injection site reactions, headache, fatigue, upper respiratory infection, and joint pain.

In a news release, Sandra Horning, M.D., chief medical officer and head of Global Product Development at Genentech said, "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".

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