Pfizer Vyndaqel Advisory Committee Pits Orphan Flexibility Vs. Data Shortcomings

FDA finds much to question in a single pivotal trial of Pfizer’s orphan drug Vyndaqel (tafamidis meglumine) for familial amyloid polyneuropathy, but the ultra-orphan nature of the disease may outweigh those concerns.

Pfizer Inc. must be hoping to appeal to FDA’s flexibility on orphan drug reviews as the advisory panel tasked with reviewing Vyndaqel (tafamidis meglumine) weighs a dispute between agency and sponsor over the statistical reading of the pivotal trial results, and the agency notes ominously that the bulk of the efficacy seems to come from a single, foreign site.

Most of FDA’s approval decisions for orphan products use unconventional efficacy standards, according to a report released last year by the National Organization for Rare Disorders, which helps make the...

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