Alnylam announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to revusiran for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis).

Familial Amyloidotic Cardiomyopathy (FAC) patients are currently being enrolled into the ENDEAVOUR Phase 3 trial, a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of revusiran in patients with FAC. The co-primary endpoints of the study are the change compared to baseline in six-minute walk distance (6-MWD) and the percent reduction in serum TTR between placebo- and revusiran-treated patients at 18 months.

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The trial is designed to enroll up to 200 FAC patients with a documented TTR mutation, including V122I or other mutations, in addition to amyloid deposits as identified by biopsy. Patients are being randomized 2:1, revusiran:placebo, with revusiran administered subcutaneously at 500mg daily for five days then weekly for 18 months. An unblinded interim analysis for futility may be conducted when 50% of patients reach 18 months. All patients completing the ENDEAVOUR Phase 3 study will be eligible to enroll in a Phase 3 open-label extension (OLE) study.

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