Alnylam Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its revusiran to treat transthyretin (TTR) mediated amyloidosis (ATTR amyloidosis).
Revusiran is an investigational RNAi therapeutic that is currently in Phase III development to treat familial amyloidotic cardiomyopathy (FAC), one of the predominant clinical manifestations of ATTR amyloidosis.
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The company is currently enrolling FAC patients in the Endeavour Phase III trial, which is a randomised, double-blind, placebo-controlled and global study designed to assess the efficacy and safety of revusiran in patients with FAC.
Alnylam regulatory affairs and quality assurance senior vice-president Dr 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."
According to the company, the co-primary endpoints of the Endeavour Phase III trial 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.
The trial will enrol up to 200 FAC patients with a documented TTR mutation, including V122I or other mutations, in addition to amyloid deposits as identified by biopsy.
In April 2014, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) recommended revusiran for designation as an orphan medicinal product to treat ATTR amyloidosis.
In January 2014, Alnylam and Sanofi company Genzyme partnered to accelerate and expand the development and commercialisation of RNAi therapeutics.
