Previously, GSK announced its intention to focus on new treatments for up to 200 rare diseases. Last week, it announced agreements with Fondazione Telethon and Fondazione San Raffaele for a novel gene therapy treatment for ADA Severe Combined Immune Deficiency.
Yesterday, GSK announced another deal with Amicus Therapeutics for its treatment, Amigal, for Fabry's Disease. Fabry's is an inherited disease that affects between 5000 and 10,000 patients worldwide. Fabry's has been in the news a lot in the past year due to Genzyme's inability to fully supply its enzyme-replacement treatment for the disease because of manufacturing problems in its torubled Allston plant.
GSK will pay Amicus $60 M upfront and the deal included milestone payments up to another $170 M. GSK also took the opportunity to bring its stock position with Amicus up to 19.9% ownership. Amigal is now in phase III trials and GSK gains global rights to develop, manufacture and distribute. Amicus-GSK will explore combining Amigal with enzyme replacment therapy as part of their study. Amigal represents a first in class attempt of an oral medication for Fabry's. See Fierce Biotech.
Posted by Bruce Lehr October 30th 2010.