Shares Of Amicus Therapeutics Are Up 30% After Positive Meeting With FDA And EMA On Rare Disease Drug

Shares of Amicus Therapeutics (FOLD) are up about 30% today after the company reported positive meetings with both the EMA and FDA regulatory authorities for its Fabry drug known as migalastat. Fabry disease is an inherited lysosomal disorder caused by a deficiency of an enzyme known as a-galactosidase A -- a-Gal A -- which is responsible for degrading lipids. In addition this enzyme causes progressive accumulation in the body of globotriaosylceramide -- GL3. This excessive build up of GL3 leads to the symptoms of Fabry disease which includes pain, kidney failure, and heart problems. 

In terms of EMA -- European Medicines Agency -- approval for migalastat, the company's Chief Medical Officer Dr. Jay Barth met with Rapporteurs to discuss MMA -- Marketing Authorization Application -- options for migalastat as a monotherapy for Fabry disease in Europe. The EMA body assigns Rapporteurs to analyze the data in the MAA and provide specific guidance to sponsors through the approval process. After the positive meeting with the Rapporteurs Amicus will be filing its MAA a lot sooner than expected. The company was going to file by the 2nd half of 2015 but after this positive meeting it will file the MAA in Q2 2015 instead. 

Chief Medical Officer Dr. Jay Barth also met with the FDA about a week ago to discuss an Accelerated Approval pathway for migalastat. The FDA wants to approve migalastat for Fabry disease since it is an unmet medical need but is considering only approving on certain Surrogate endpoints. Surrogate endpoints are clinical endpoints that the FDA establishes that prove drug efficacy against the disease.

In the case of this meeting the FDA has agreed accelerated approval on these surrogate endpoints:

1. Stabilization of Kidney function
2. Reduction in Cardiac mass
3. Substrate Reduction -- meaning reduction of GL3 lipid

In addition to the above, Amicus Therapeutics must run an additional confirmatory Phase 4 study. This study can take place after approval but it must be done to satisfy the FDA requirements to keep the drug marketed. With all this Amicus expects to have a pre-NDA meeting with the FDA by the second half of 2015. 

These two upcoming catalysts will provide investors with additional catalysts to look forward to. Shares of Amicus are trading 11x greater than average volume at the time of this article and it seems the momentum may continue in the coming days. We tend to like companies like Amicus which treat rare diseases because they tend to get more benefits in terms of approval as seen above. In addition prices of approved rare disease drugs can be set higher for greater profit because of the lack of competitive medicines. We think that Amicus Therapeutics is a great long-term biotech to own especially now that the company has already succeeded in phase 3 and the only hurdle left now is regulatory approval.