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ASH 2020 | Fitusiran prophylaxis shows encouraging results in hemophilia patients

Steven Pipe, MD, University of Michigan, Ann Arbor, MO, discusses the results of the Phase II, open-label extension study (NCT02554773) aiming to assess the long-term durability, efficacy and safety of fitusiran prophylaxis in hemophilia A or B (HA/HB) patients, with or without inhibitors. Fitusiran, a small interfering RNA, is designed to lower antithrombin level, thus improving thrombin generation and resulting in fewer and milder bleeds. In patients treated with fitusiran, the antithrombin blood levels were reported to fall below 20% of the norm. Monthly prophylaxis provided sustained antithrombin lowering in both HA and HB patients, with or without inhibitors, resulting in a low bleeding rate over the follow-up period. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.

Disclosures

Consulting: Apcintex, Bayer, Biomarin, Catalyst Biosciences, CSL Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sangamo Therapeutics, Sanofi, Takeda, Spark Therapeutics, uniQure

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