New inhibitor therapies have the potential to change hemophilia treatment

Considering that the treatment of hemophilia with inhibitors remains problematic, and that inhibitors may be an important consideration for new approaches such as gene therapy, it’s particularly relevant that a session during the Congress examined alternative therapies in the management of inhibitors. Midori Shima, Nara Medical University, Japan, chaired this session, which was designed to help attendees understand the next generation of treatments for people with hemophilia and inhibitors. “The session aims at summarizing the current concepts of inhibitor development and eradication from the immunological aspect, and presenting emerging novel therapies,” Shima said. David Lillicrap, Queen’s University, Canada, presented new hypotheses of causes of inhibitor and their elimination. Rebecca Kruse- Jarres, University of Washington, U.S.A., discussed how recombinant porcine FVIII constitutes a potent hemostatic therapeutic.

Shima summarized details of clinical trials of three novel therapeutics: an anti-FIXa/X bispecific antibody (emicizumab)—in which he is directly involved—an anti-antithrombin siRNA (fitusiran) and an anti-TFPI antibody (concizumab).