WASHINGTON (dpa-AFX) - Alnylam Pharmaceuticals, Inc. (ALNY) announced that the U.S. Food & Drug Administration, or FDA, has provided Orphan Drug Designation to ALN-TTR02 as a therapeutic for the treatment of familial amyloidotic polyneuropathy, or FAP, one of the predominant clinical manifestations of transthyretin (TTR)-mediated amyloidosis, or ATTR.
Saraswathy Nochur, vice president, Regulatory Affairs and Quality Assurance at Alnylam, stated, 'We believe RNAi therapeutics represent a novel and exciting approach for ATTR patients and have great potential to make a meaningful impact in the treatment of this devastating disease. We look forward to sharing Phase I clinical data from our ALN-TTR02 program early in the third quarter, and, assuming continued positive results, we plan to advance to a pivotal trial in 2013. Alnylam is committed to bringing this high impact medicine to patients afflicted with ATTR.'
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