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Persistence Pays Off For Amicus: Positive Migalastat Data In Hand

This article was originally published in The Pink Sheet Daily

Executive Summary

Amicus will return to FDA with positive data on its oral small molecule chaperone migalastat in Fabry patients after a Phase III trial showed the oral drug had comparable efficacy to existing enzyme replacement therapies.

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Amicus' Oral Fabry Drug Priced To Compete Against Traditional ERT

FDA approved Galafold under accelerated approval, paving the way for the launch of the first oral therapy to treat a subset of patients with the rare condition Fabry disease. Amicus priced Galafold at $315,000 per year.

FDA Reversal Gives Amicus Renewed Hope For US Oral Fabry Launch

Amicus plans to file migalastat for Fabry disease in the US in the fourth quarter, after FDA gave the greenlight, reversing a decision last year to require an additional Phase III study.

FDA Reversal Gives Amicus Renewed Hope For US Oral Fabry Launch

Amicus plans to file migalastat for Fabry disease in the US in the fourth quarter, after FDA gave the greenlight, reversing a decision last year to require an additional Phase III study. Additional information includes European submission data and post-launch RWE.

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