Amyloid | 2019
Patient profile with ATTR-FAP and evaluation of the safety and efficacy of tafamidis meglumine in Japan – interim analysis in post-marketing surveillance
Abstract
Transthyretin familial amyloid polyneuropathy (ATTR-FAP) is a fatal hereditary amyloidosis characterized by extracellular deposition of amyloid fibrils composed of transthyretin (TTR) [1,2]. Tafamidis meglumine (VyndaqelR capsules, Pfizer, Tokyo, Japan; hereinafter called tafamidis) is a TTR stabilizer and was approved in Japan in 2013 for delaying peripheral neurologic impairment in patients with ATTRFAP [2,3]. An all-case, post-marketing surveillance (PMS) study is being conducted in Japan to assess the long-term, real-world safety and effectiveness of tafamidis in patients with ATTR-FAP (study period, November 2013 to May 2021). Here, we report the results from the interim analysis (data cut-off date: 15 May 2017).