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GSK Pursues Fabry Disease Drug as Orphan Indications Are All The Rage

Amicus Therapeutics has just signed a deal with GSK giving the large pharma worldwide rights to their Fabry disease drug, Amigal, in return for $60 million in upfront payments and $170 million in milestones. Patients are currently enrolling in a Phase III trial of Amigal, with preliminary results expected by the end of this year.
 
This is fantastic news for Amicus, which has been developing its Fabry disease drug since its inception in 2002. Instead of enzyme replacement, the company designed inhibitor-like compounds that act like “molecular chaperones”, helping to stabilize the defective protein. In this case, the protein is an enzyme called alpha-galactosidase A. The technology has also been applied to other disorders including Gaucher’s disease and Pompe disease.
 
This new agreement with GSK is a redemption of sorts for Amicus after being dumped by Shire in 2009 only two years after their partnership began when the company’s Gaucher’s disease candidate suffered a setback in the clinic. It is also a sign of pharma’s intense interest in the ultra-rare disease space.
 
There are now at least four major players elbowing with treatments for Fabry disease: Genzyme (soon to be part of Sanofi); Shire; GSK via the Amicus deal, and Pfizer through a partnership with Protalix. Keep in mind peak sales for Fabrazyme from Genzyme, the leading treatment, never surpassed $500 million annually and there are a mere 2500 diagnosed patients in the US.
 
I understand the allure of selling treatments costing well over $100,000, but this sort of herd mentality simply defies logic. 


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