Shares of Amicus Therapeutics (FOLD) are down 40% today after the company provided an FDA update for its Fabry Disease drug Migalastat. The company has received the FDA minutes from the pre-NDA meeting that was held in September.

The strategy that Amicus is using is to conduct additional studies for different pathways for approval of its Migalastat drug. For instance, the company is contemplating performing a study to determine the effect of migalastat on gastrointestinal symptoms of patients with Fabry disease.

The FDA requested that the company complete additional studies with further clinical data supporting the package of an application for Amicus’ Migalastat. These studies will take a long time to complete and many investors were expecting a pathway for FDA approval to take place before the end of this year (as recently as last month, the company had said it expected to be able to file an NDA in Q4). Because of this delay, investors now realize that filing an NDA won’t happen anytime soon. 

This isn’t a doom and gloom scenario, it is just a delay in the program. While short term traders will exit, those who have a longer term horizon in investing in Amicus have nothing to worry about. The results to date in phase 3 have been good, now all that is required is additional evidence as requested by the FDA. Fabry disease is characterized by faulty enzymes that patients have in the body that are unable to break down lipids – fat substances – in the body. 

Patients with Fabry disease experience a range of symptoms from gastrointestinal problems all the way to other more severe complications, such as kidney damage and stroke. Fabry is a rare disease and there are hardly any treatment options The only treatment option currently available is enzyme replacement therapy but it doesn’t help very much. All the enzyme replacement therapy does is slow down the progression of the disease, but does not improve upon the symptoms experience by patients. 

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