In the news yesterday was FDA approval of the Fabry disease treatment Migalastat (Galafold). Here's a couple articles on it:

FDA approves new treatment for a rare genetic disorder, Fabry disease

Once stiff-armed at the FDA, new management offers Amicus a warm embrace and marketing OK for Galafold

Before anyone jumps to conclusions about Resverlogix missing the boat, I suggest you read up on the history this drug. The Wikipedia page on Migalastat (Galafold) is pretty good. This drug has been in clinical trials for several years now. According to the Wikepedia page, two Phase 3 trials were completed between 2009 and 2015. The drug was approved in Europe over two years ago. So it is not a new therapy for everyone, just the US and others that may follow FDA guidance. 

Migalastat (Galafold) is not for every Fabry patient. According to the Wikipedia page:

"Amicus Therapeutics has published a list of 269 amenable and nearly 600 non-amenable mutations. About 35 to 50% of people with Fabry have an amenable mutation."

Therefore, only ~50% at most of Fabry patients could potentially benefit from this drug. 

It's mechanism of action is actually pretty cool. Many mutations of the α-GalA enzyme result in protein misfolding in the endoplasmic reticulum (ER) of the cell. Migalastat is a pharmacological chaperone that binds to the faulty enzyme. If it is an amenable mutation, this binding facilitates the proper protein folding in the ER, resulting in an increase in functional enzyme levels. Misfolded proteins are often destroyed, so this chaperone stabilizes the protein during cellular transport. As a later part of the cellular transport process, the enzyme is transported from the ER to the lysosome of the cell (its final destination), where the low pH and abudance of enzymatic substrates for α-GalA causes migalastat to dissociation and permit α-GalA to do its thing.

I haven't reviewed the clinical results yet for past Migalastat trials, so I can't comment on the Migalastat clinical findings and how they may compare to what apabetalone can do for these Fabry patients. However, my initial take is that the excitement shown by Resverlogix and the Fabry community for the potential of apabetalone to treat Fabry disease is a strong statment that even with Migalastat available there is still huge unmet need for novel Fabry treatments. 50% or more of Fabry patients won't benefit from Migalastat. And for those that may derive some benefit, the magnitude and duration of benefit may be limited. There is still very much room for apabetalone or other novel treatments for Fabry disease, IMO.

BearDownAZ