UPDATE: FOLD received approval from the FDA for migalastat HCI. Our subscribers saw it coming when we sent this post 5 full days before FDA approval!

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There’s a major catalyst coming for Amicus Therapeutics, Inc. (NASDAQ: FOLD) relatively soon. The company’s PDUFA date surrounding migalastat HCI for the treatment of patients 16 years of age and older with Fabry disease ho have amenable mutations. That date is August 13, 2018. After reviewing various information, we expect that the treatment will be approved.

What Is Migalastat HCI?

Migalastat HCI is a treatment under development by Amicus Therapeutics. The treatment is a first-in-class chaperone therapy that has been approved in the European Union as well as Japan as a monotherapy for Fabry disease in patients with amenable mutations.

The treatment works by stabilizing the body’s own dysfunctional enzyme. Through thsi method of action, it can clear the accumulation of disease substrate in patients who have amenable mutations. The approval in the EU includes 348 GLA mutations that have been identified and determined to be amenable based on the Galafold Amenability Assay. This means that the treatment is deemed to be effective in the EU in between 35% and 50% of the diagnosed Fabry population.

Why The Expectation Is That The FDA Will Approve Migalastat HCI

At the end of the day, there are several reasons that we are expecting to see Migalastat HCI be approved for the treatment of Fabry disease in the United States. Among the most important are:

Positive Clinical Data – First and foremost, it’s always important to take a look at the clinical data when determining whether or not you believe a treatment is likely to be approved. In the case of Migalastat, the data proved to be incredibly positive. In fact, the data proved to be so positive that the Journal of Medical Genetics picked it up for publication.

Regulatory Activity – Another key factor in my conclusion that Migalastat will be approved by the FDA has to do with regulatory activity abroad. At the moment, Migalastat is actually a commercial-stage treatment with approvals in both Europe and Japan. The company has even launched the product in both regions, with the Japanese launch being the most recent in May of this year.

Priority Review – The New Drug Application surrounding Migalastat wasn’t simply accepted, it was accepted for priority review. This means that the FDA will focus more efforts on the review to bring the treatment to the market more quickly as it addresses a large unmet medical need in the United States. Priority review is only granted to treatments with promising data from clinical trials. Therefore, there’s a strong indication that the FDA has a positive view of the treatment.

Be Prepared For This Catalystic Event

With approvals in the EU and Japan, strong clinical data, and priority review from the FDA, chances are that Migalastat’s New Drug Application will end in approval. Considering that the United States is the largest market for the treatment, it only makes sense that approval will prove to be a positive catalystic event for FOLD. I believe that, not only will approval be announced, but when it is, the stock will see a dramatic rise in valuation as a result of excited investors making their moves.

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