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After 12 years, millions of dollars, and a major reversal of fortune, Amicus Therapeutics won Food and Drug Administration approval on Friday for its first therapy, a treatment for a rare and sometimes deadly disease.

The drug, called Galafold, targets Fabry disease, an inherited disorder in which the lack of a key enzyme leads to buildups up fats that can cause fatal organ damage. As it stands, Fabry patients rely on costly, bimonthly injections of synthetic enzymes to keep symptoms at bay. Amicus’s drug is an every-other-day pill that works not by replacing the missing enzyme but by boosting the effects of what patients already produce.


The FDA approval clears Galafold for use in patients with certain genetic variants, accounting for up to 50 percent of the roughly 3,000 people with Fabry in the U.S., according to Amicus.

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  • This article fails to point out that the US phase 3 trial failed to hit statistical significance and the EU phase 3 trial had a serious patient imbalance where the Galafold arm had a bunch of patients with a less sick genotype.

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