Note: The following is edited from a press release from Alnylam Pharmaceuticals. Read the full press release in its entirety here.
Alnylam Pharmaceuticals Inc., a RNAi therapeutics company, andÂ SanofiÂ Genzyme, the specialty care global business unit ofÂ Sanofi, announced Dec. 15, 2017, theÂ U.S. Food and Drug AdministrationÂ (FDA) has lifted the hold on clinical studies with fitusiran, including the Phase 2 open-label extension (OLE) study and the ATLAS Phase 3 program.
Alnylam and theÂ FDAÂ had previously reached alignment on new clinical risk mitigation measures, including protocol-specified guidelines and additional investigator and patient education concerning reduced doses of replacement factor or bypassing agent to treat any breakthrough bleeds in fitusiran studies. TheÂ FDAÂ has now approved the protocol amendments and other updated clinical materials for fitusiran studies. Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of patients with hemophilia A and B. It is designed to lower levels of AT with the goal of promoting sufficient thrombin generation