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Alnylam ATTR Drug Receives Orphan Drug Status in Europe


Alnylam Pharmaceuticals this week announced that its transthyretin-mediated amyloidosis drug ALN-TTRsc has received orphan drug status from European regulators.

ALN-TTRsc is a subcutaneously administered version of the company's ATTR drug patisiran, an intravenous therapy currently in Phase III testing in patients with a manifestation of the disease called familial amyloidotic polyneuropathy.

The subcutaneous version recently entered Phase II testing for a version of ATTR called cardiac amyloidosis. That trial is assessing the tolerability of the drug, as well as its effect on knocking down serum levels of its target protein, in 15 patients. Data from the trial is expected later this year.