US FDA refuses to file Pfizer's tafamidis for TTR-FAP
This article was originally published in Scrip
Executive Summary
Pfizer has suffered a blow in its bid to crack the rare diseases market after receiving a refusal to file letter from the US FDA for its NDA for tafamidis, the company's investigational oral compound for patients with transthyretin familial amyloid polyneuropathy (TTR-FAP).