News ) Monday said it received a ‘Refusal to File’ letter from FDA, rejecting its New Drug Application for new oral investigational compound tafamidis, as it was not sufficiently complete to permit a substantive review.
The company expects that no additional clinical studies are required for refiling the application.Tafamidis is intended for the treatment of patients with Transthyretin Familial Amyloid Polyneuropathy or TTR-FAP, a progressively fatal genetic neurodegenerative disease caused by the mutation of the TTR gene.
TTR is an amyloidogenic protein secreted by the liver. TTR-FAP is characterized by pain, paresthesia, muscular weakness and autonomic dysfunction.
Liver transplant is the only treatment option that is currently available.Tafamidis is being developed by FoldRx Pharmaceuti…