FDA grants orphan drug status for Alnylam’s Revusiran to treat TTR-mediated amyloidosis

Currently, Revusiran is being evaluated in a Phase III trial to treat Familial Amyloidotic Cardiomyopathy (FAC), one of the predominant clinical manifestations of ATTR amyloidosis.

Patient enrollment is currently being carried out in the randomized, double-blind, placebo-controlled, global Phase III trial (Endeavour), which is designed to evaluate the efficacy and safety of revusiran in FAC patients.

Alnylam Regulatory Affairs and Quality Assurance senior vice-president Saraswathy Nochur said: "We believe RNAi therapeutics represent a promising new approach for the treatment of ATTR amyloidosis, with the potential to make a meaningful impact for patients with this progressive and debilitating disease.

"We look forward to the continued advancement of revusiran, including enrollment in our Endeavour Phase III trial in ATTR amyloidosis patients with FAC.

"In addition, we continue dosing TTR cardiac amyloidosis patients in our Phase II open-label extension study with revusiran, and plan to present initial data from that study in late 2015."

The co-primary endpoints of the Endeavour Phase III trial are the change compared to baseline in 6-minute walk distance (6-MWD) and the percent reduction in serum TTR between placebo- and revusiran-treated patients at 18 months.

The trial’s secondary endpoints include a composite endpoint of cardiovascular mortality and cardiovascular hospitalization, New York Heart Association (NYHA) class, Kansas City Cardiomyopathy Questionnaire (KCCQ), CV mortality, CV hospitalization and all-cause mortality.

Around 200 FAC patients with a documented TTR mutation, including V122I or other mutations, in addition to amyloid deposits as identified by biopsy will be enrolled in the Phase III trial.