Rare Neurologic Disorder Therapy Designated Orphan Drug

Rare Neurologic Disorder Therapy Designated Orphan Drug
Rare Neurologic Disorder Therapy Designated Orphan Drug

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.

RELATED: Neurologic Disorders Resource Center

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.

For more information visit Alnylam.com.

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