FDA Grants Priority Review to Emicizumab for Haemophilia A Treatment

Barsaba Taglieri
Agosto 25, 2017

The U.S. Food and Drug Administration (FDA) has accepted Genentech's Biologics License Application (BLA) and granted Priority Review for emicizumab to prophylactically treat patients with hemophilia A with factor VIII inhibitors.

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. "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". The FDA granted Breakthrough Therapy Designation for emicizumab in adults and adolescents with hemophilia A with inhibitors in September 2015. The study met its primary endpoint, showing that prophylaxis with emicizumab demonstrated a clinically meaningful and statistically significant reduction in treated bleeds of 87% (risk rate [RR]=0.13, P 0.0001) compared to on-demand BPAs. Patients previously treated with prophylactic bypassing agents received emicizumab prophylaxis. Researchers also evaluated safety and pharmacokinetics.

The intrapatient analysis that compared the two prophylaxis regimens showed a 79% reduction in treated bleeds among a subset of patients treated with emicizumab. The most common adverse events were local injection site reactions, headache, fatigue, upper respiratory infection, and joint pain.

HAVEN 2 is a single-arm, multicenter, open-label trial evaluating the efficacy, safety and pharmacokinetics of once-weekly SC emicizumab in children 12yrs. Interim results were consistent with the positive results from the HAVEN1 study.

One of 19 children reported a treated bleed, and no joint or muscle bleeds were reported. The only AE that was severe in intensity was a moderate event of injection-site hematoma, which occurred on trial day 2 and resolved on trial day 28. Results from HAVEN 1 were published in The New England Journal of Medicine (NEJM) and results from both studies were presented at the 26th International Society on Thrombosis and Haemostasis (ISTH) Congress in July 2017.

"We are working with the FDA to hopefully bring this new prophylactic treatment option to the hemophilia A inhibitor community as soon as possible", Horning said in the release. Additional studies for emicizumab in hemophilia A with and without inhibitors are ongoing.

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