Issue Date: July 17, 2017
FDA shifts on rare-disease drug approval
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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