19 Jun, 2020 New Positive Results for Fitusiran Presented at WFH
New positive results from an interim analysis of the Phase 2 open-label extension (OLE) study of fitusiran, in development for the treatment of hemophilia A or B, with or without inhibitors, were presented at the World Federation of Hemophilia (WFH) Virtual Summit, held June 14-19, 2020.
In the data presented at WFH, monthly subcutaneous dosing with fitusiran in patients (N = 34) with moderate or severe hemophilia A and B, with and without inhibitors, demonstrated a durable therapeutic effect, including sustained lowering of antithrombin, increased thrombin generation, and low overall annualized bleed rate (median 0.84) in the median observation period of 36 months.
Long-term exposure to fitusiran in the Phase 2 OLE study reveals a safety profile that is supportive of continued evaluation of fitusiran in the ongoing Phase 3 ATLAS program.
Sanofi Genzyme is leading and funding the global development of fitusiran and will commercialize fitusiran, if successful.