=Amicus Therapeutics (FOLD) to re-file for rare disease drug

• The Food & Drug Administration confirmed the company's plans to submit a new drug application for Amicus' migalastat, an oral treatment for Fabry disease. The company expects to submit during the fourth quarter. 
• The FDA could still reject the drug.

Last November,  tiny Amicus Therapeutics said that the Food and Drug Administration would not even consider approving migalastat, a treatment for a rare disorder called Fabry disease, until more data were collected, a process that was expected to take until 2019 or 2020.

Today, Amicus says that the FDA is reversing the decision, and that there is a clear path to approval.  Amicus hopes to file a new drug application by the end of the year, and approval would take six months after that. That will shave three or four years off the drug's path to market if all goes well.

In Fabry disease, a substance called GL-3 builds up in the body, causing pain, kidney failure, heart disease and stroke. About 3,000 Americans have been diagnosed with the condition, according to Amicus. Migalastat is thought to work only in about half the patients with the disease, whose disorder is linked to certain genetic mutations.

The drug was approved in the European Union a year ago, but the FDA was declining to consider Amicus' application, in part because one of two clinical trials of the drug had failed to meet its main prespecified goal. But John Crowley, Amicus' chief executive, says that the company convinced the FDA to look at its data using new analyses, including some that had been asked for by European regulators, and patient testimony. "We didn’t ask them to lower the bar," he says. "We just asked them to review the existing data."

Crowley is famous in biotech circles for his work developing a drug for Pompe disease, another rare disease from which his daughter, Megan, suffers. The story was the subject of a book, The Cure, and a movie, Extraordinary Measures, which starred Harrison Ford. Earlier this year, President Donald Trump highlighted Megan Crowley's story in a speech to congress as an example of the need for a more flexible FDA.