Volume 95 Issue 29 | p. 14
Issue Date: July 17, 2017

FDA shifts on rare-disease drug approval

Amicus Therapeutics needn’t conduct a new trial for Fabry disease treatment, agency says
Department: Business
Keywords: regulation, rare disease, migalastat, Amicus, Trump

In a change of heart, the U.S. Food & Drug Administration has told Amicus Therapeutics it can seek approval for its Fabry disease treatment migalastat. Shares of the rare-disease-focused biotech firm soared by more than 25% on news of the regulatory shift, which trims at least three years off the development time for the drug.

Migalastat stabilizes the enzyme responsible for breaking down globotriaosylceramide (GL-3), a fatty substance that accumulates in the lysosomes of people . . .

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