FDA grants ISIS TTRRx (Isis Pharmaceuticals) fast track designation for treating Familial Amyloid Polyneuropathy

The FDA has granted ISIS TTRRx, from Isis Pharmaceuticals, fast track designation for the treatment of Familial Amyloid Polyneuropathy (FAP). ISIS TTRRx is an antisense drug in development with GlaxoSmithKline (GSK) for the treatment of transthyretin (TTR) amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues. In a Phase I trial, subjects treated with ISIS TTRRx generally tolerated the drug well. Isis Pharmaceuticals now plans to initiate a Phase II/III study evaluating the efficacy of ISIS TTRRx in patients with FAP in 2012.