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Amicus' Fabry drug 'accelerated' in Europe

This article was originally published in Scrip

Executive Summary

The EMA's Committee for Medicinal Products for Human Use (CHMP) has granted accelerated assessment to migalastat, Amicus Therapeutics' experimental oral small-molecule monotherapy for Fabry patients who have amenable genetic mutations. This follows recent news that the company plans to seek an accelerated approval of its NDA in the US, which sent Amicus' share price soaring. The firm said it would submit the NDA in the second half of this year based on positive information from the FDA. Under accelerated assessment, the CHMP may shorten the marketing authorization assessment (MAA) review period from 210 days to 150 days. The EC generally issues a final decision on EU approval within three months of the CHMP's recommendation. If approved, Amicus will then pursue country-by-country reimbursement. Amicus says it is on track to submit the MAA to request full approval for migalastat monotherapy in the EU in the second quarter of 2015. Amicus previously reported positive Phase III migalastat data in its FACETS and ATTRACT trials, which demonstrated reductions in disease substrate, stability of kidney function, reduction in cardiac mass and a positive impact on patient-reported outcomes in patients with amenable mutations.

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