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Shire’s Replagal Withdrawn: A “Non-Traditional” Review Ends Badly

This article was originally published in RPM Report

Executive Summary

Shire’s decision to take its Fabry Disease therapy and go home sets up two possible narratives. One accentuates FDA’s apparent inflexibility and is sure to reinforce calls for legislative changes to the approval standard. The other highlights a recalcitrant or even petulant-sounding sponsor. Neither is likely to sound good to Fabry’s patients.

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