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Medical Session Spotlight: Alternative therapies for the management of inhibitors

On July 25, Professor Midori Shima will give an important lecture on alternative therapies for the management of inhibitors.

Midori-Shima

On Monday, July 25, Professor Midori Shima, Department of Pediatrics, Nara Medical University, will give an important lecture on alternative therapies for the management of inhibitors. The lecture will focus on recent developments regarding the bispecific antibody emicizumab—a treatment that was designated as a Breakthrough Therapy by the US Food and Drug Administration (FDA) in September 2015.

Emicizumab is a therapy of great interest to the bleeding disorders community because it may be a way to prevent bleeds without infusing clotting factor concentrates and in particular could prove to be an effective treatment for patients with inhibitors.

Phase 1 study results, published in May 2016, demonstrated promising results for the drug: a once-weekly subcutaneous injection of emicizumab demonstrated a clinically acceptable safety profile and a potential benefit for preventing bleeding in hemophilia A patients (with and without FVIII inhibitors). The molecule is currently in a Phase III global study in hemophilia A patients with FVIII inhibitors. Phase III global studies in patients without inhibitors and pediatric patients are also planned to begin in 2016.

The potential of emicizumab is intriguing because the development of inhibitors to FVIII or factor IX (FIX) is a major and costly challenge when treating hemophilia. Not all hemophilia patients with the same severity and genotype develop inhibitors suggesting the presence of a tolerance mechanism against FVIII- or FIX-derived immunity. Immune tolerance induction (ITI) therapy is the essential treatment for patients with inhibitors. Approximately 30% of the patients in hemophilia A and over half of hemophilia B patients do not respond to ITI, however, and the mechanism of immune tolerance induction remains unknown.

Now is an exciting time in the world of bleeding disorders, as there are multiple new therapeutics based on innovative concepts in development. While emicizumab uses a humanized asymmetric antibody that mimics FVIIIa function by maintaining a suitable interaction between FIXa and FX, concizumab is a humanized anti-TFPI monoclonal antibody restoring coagulation through inhibiting TFPI and another drug – fitusiran – uses small interfering RNAs (siRNA, ALN-AT3) to suppress liver production of AT through post-transcriptional gene silencing.

Keep on top of these exciting developments by attending Dr. Shima’s lecture on Monday, July 25th from 16:30 to 18:00. To find out more, consult our Congress program page.

Content adapted from:
– Chugai news release dated May 26, 2016
– Alternative therapies for the management of inhibitors, M. Shima, Department of Pediatrics, Nara Medical University, Kashihara, Japan; D. Lillicrap, Department of Pathology and Molecular Medicine, Queen’s University, Kingston, ON, Canada; and R. Kruse- Jarres, Washington Center for Bleeding Disorders at Bloodworks NW University of Washington, Seattle, WA, USA.