The Food and Drug Administration (FDA) has approved protocol amendments and updated clinical materials for fitusiran (Sanofi Genzyme and Alnylam), lifting the hold on clinical studies of the investigational hemophilia treatment so they can proceed.
Fitusiran, an RNAi therapeutic targeting antithrombin (AT), is designed to lower levels of AT in order to further sufficient thrombin generation to restore hemostasis and prevent bleeding. With the FDA’s decision to lift the hold on fitusiran, the Phase 2 open-label extension study and the Phase 3 ATLAS program are expected to resume clinical trial dosing around the end of 2017.
Previously, the FDA and Alnylam had reached an agreement on new clinical risk mitigation measures for protocol-specified guidelines and additional investigator and patient education regarding the treatment of any breakthrough bleeds that should arise in studies.
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