Alnylam Pharmaceuticals Receives Positive Opinion for Orphan Drug Designation from European Medicines Evaluation Agency for ALN-TTR01, an RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that The Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA) adopted a positive opinion for ALN-TTR01 designation as an orphan medicinal product for the treatment of familial amyloidotic polyneuropathy (FAP), one of the predominant forms of transthyretin (TTR)-mediated amyloidosis (ATTR). ALN-TTR01 is a systemically delivered RNAi therapeutic being developed for the treatment of all forms of ATTR, which includes FAP and familial amyloidotic cardiomyopathy (FAC). A positive opinion by the COMP precedes official designation of ALN-TTR01 as an orphan drug by the European Commission.
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