What happened

Shares of Amicus Therapeutics (NASDAQ:FOLD), a biopharmaceutical company focused on the development rare-disease drugs, exploded higher by 29% during the month of July, according to data from S&P Global Market Intelligence. The culprit for the decisive move higher is a press release from the company on July 11.

So what

Back in the fall of 2015, the U.S. Food and Drug Administration (FDA) delivered a crushing blow to Amicus' shareholders by requesting more data from the drugmaker regarding its leading drug candidate (at the time), migalastat (brand name, Galafold), as a treatment for Fabry disease. Fabry causes a fatty substance called GL-3 to build up in vital organs, which can lead to life-threatening problems like stroke, heart attack, or kidney failure. The FDA requested Amicus to include data from previous clinical studies before it would consider allowing the company to file for a new drug application. 

A biotech lab researcher holding a blood sample and reading notes on a clipboard.

Image source: Getty Images.

Fast forward to July 11, 2017 and a press release from Amicus Therapeutics stating that the FDA had changed its tune. Per the release, the FDA now deemed the data sufficient for a new drug filing, which the company plans to complete during the fourth quarter. Migalastat is already approved in Europe.

What's more, the submission will be filed under "Subpart H," which is a fancy way of saying that it'll be given accelerated approval, pushing a potentially game-changing drug to market a bit quicker after its previous lengthy delay. An estimated 3,000 people are diagnosed with Fabry disease in the U.S., and while it won't be the only drug in the U.S. to treat the disease, the substantial pricing power bestowed on orphan disease drugs should allow Amicus to capture healthy recurring revenue, if approved. Amicus' drug is specifically designed for patients who have amenable genetic mutations, of which 35% to 50% of Fabry patients around the world do have.

Now what

Earlier this week, Amicus Therapeutics reported its second-quarter operating results and pointed out that 179 Fabry disease patients were on Galafold as of July 31, with a year-end target of approximately 300 patients. Overall, this wound up leading to $7.2 million in quarterly revenue, up 71% from the sequential first quarter. Meanwhile, net loss shrank by $3 million year-over-year to $48.1 million. That may not sound like much progress, but it's baby steps in the right direction toward profitability. An approval in the U.S. would go a long way to helping stem these losses.

A doctor having a discussion with his patient.

Image source: Getty Images.

Furthermore, a recent public offering that raised a whopping $243.2 million gives Amicus a little over $470 million in cash and cash equivalents, which the company estimates should be enough of a runway to last into at least the second-half of 2019. Ergo, funding isn't an immediate concern.

As an additional catalyst, phase 3 data for SD-101 in epidermolysis bullosa is expected in the third quarter, along with top-line phase 1/2 data for ATB2000/AT2221 for Pompe disease. There are plenty of catalysts, and sales are trending in the right direction, but investors may want to wait for a significant reduction in operating losses before they consider dipping their toes in the water.

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