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Isis Announces Positive Opinion On European Orphan Drug Designation For ISIS-TTR Rx

CARLSBAD, Calif., Feb. 14, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending ISIS-TTR Rx for designation as an orphan medicinal product for the treatment of ATTR-Amyloidosis to the European Commission (EC). The opinion will be subject to review by the EC, which ultimately grants the decision on orphan drug designation.  ATTR-Amyloidosis, or TTR amyloidosis, is a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.    ISIS-TTR Rx is an antisense drug in development with GlaxoSmithKline (GSK), which has an option to exclusively license the ISIS-TTR Rx program.  ISIS-TTR Rx is currently being evaluated in a Phase 2/3 study in familial amyloid polyneuropathy (FAP) patients.

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"We are encouraged by COMP's continued recognition of the need for new treatments for rare and orphan diseases and the designation of our drugs as potential therapeutic options.  Since the beginning of the year, we have received two positive opinions on European Orphan Drug Designation, first for ISIS-APOCIII Rx, our novel triglyceride-lowering drug, and now for ISIS-TTR Rx," said B. Lynne Parshall, chief operating officer at Isis.  "Our Phase 2/3 study for ISIS-TTR Rx is underway with some patients treated for more than six months. FAP is a devastating life-threatening disease and patients with FAP have limited therapeutic options.  We remain encouraged about the potential for ISIS-TTR Rx to provide therapeutic benefit for these patients in need."     

The COMP, a committee of the European Medicines Agency, adopts an opinion on the granting of orphan drug designation, after which the opinion is submitted to the European Commission for endorsement of the opinion.  Orphan drug designation is granted to products designed to diagnose, prevent or treat life-threatening or very serious conditions that affect not more than five in 10,000 persons in the European Union.  

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