This Tiny Biotech Pins Hope on U.S. Approval

This Tiny Biotech Pins Hope on U.S. Approval

Amicus Therapeutics (NASDAQ: FOLD) already markets its Fabry disease drug Galafold overseas, and it hopes it will soon be able to help patients in the U.S., too. Yesterday, management reported that it had completed the application for Galafold's Food and Drug Administration (FDA) approval.

The FDA has 60 days to accept or reject the application. If it's accepted, the FDA will set a date to announce an official go/no-go decision on Galafold. An eventual approval could significantly improve this company's financial position, so let's take a closer look at Galafold's opportunity. 

Fabry disease patients don't produce enough of a protein that's responsible for breaking down a fatty substance called GL-3. As a result, GL-3 builds up in vital organs, including the heart and kidneys. Over time, the buildup increases the risk of kidney failure, stroke, and heart attack.

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Source: Fool.com