Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has provided Orphan Drug Designation to ALN-TTR02 as a therapeutic for the treatment of familial amyloidotic polyneuropathy (FAP), one of the predominant clinical manifestations of transthyretin (TTR)-mediated amyloidosis (ATTR).
"We are very pleased to have received Orphan Drug Designation from the FDA for ALN-TTR02, the lead effort in our 'Alnylam 5x15' product strategy. 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," said Saraswathy (Sara) Nochur, Ph.D., Vice President, Regulatory Affairs and Quality Assurance at Alnylam. "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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