CAMBRIDGE, Mass.--(BUSINESS WIRE)--FoldRx Pharmaceuticals, Inc. (FoldRx) today announced positive results from its pivotal Phase II/III clinical study of the company’s lead compound, tafamidis (Fx-1006A), in patients suffering from TTR amyloid polyneuropathy (ATTR-PN), a fatal orphan disease also known as Familial Amyloid Polyneuropathy (FAP). Liver transplantation is the only currently available treatment option for this progressive neurodegenerative disease. Preliminary results from the first randomized controlled trial ever completed in this disease demonstrate that tafamidis treatment significantly halts disease progression in ATTR-PN, reduces the burden of disease after 18 months compared to placebo, and appears to be safe and well-tolerated.
