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US FDA rejects Pfizer rare neurodegenerative disease drug

This article was originally published in Scrip

Executive Summary

Pfizer revealed on 18 June after the markets closed that it received a complete response letter (CRL) from the US FDA for the firm's new drug application (NDA) for Vyndaqel (tafamidis meglumine), which the company is seeking to market as a therapy for transthyretin familial amyloid polyneuropathy (TTR-FAP), a rare, genetic progressive neurodegenerative disease, in which there is a buildup of amyloid, primarily in peripheral nerves, but also in the organs.


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