Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), announced on 4/23/18 that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending ALN-TTRsc02, an investigational, subcutaneously administered RNAi therapeutic, for designation as an orphan medicinal product for the treatment of transthyretin (TTR)-mediated (ATTR) amyloidosis.
ALN-TTRsc02 is an investigational, subcutaneously administered RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to reduce the deposition and facilitate the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues. The safety and efficacy of ALN-TTRsc02 have not been evaluated by the U.S. Food and Drug Administration, European Medicines Agency or any other health authority.
Transthyretin (TTR)-mediated (ATTR) amyloidosis is a rare, progressively debilitating, and often fatal disease caused by misfolded TTR proteins that accumulate as amyloid deposits in multiple tissues including the nerves, heart, and GI tract. Prevalence of ATTR amyloidosis is uncertain, however estimates suggest fewer than 200,000 patients across the U.S. and Europe.
ALN-TTRsc02 is currently in Ph II development in the US.