GSK, Amicus ink $230M deal for Fabry disease

Continuing its push into the rare disease market, GlaxoSmithKline has agreed to pay $60 million up front and up to $170 million in milestone payments for Amicus Therapeutics' Fabry disease drug Amigal (migalastat HCl). Fabry is an inherited disease that affects just 5,000 to 10,000 people worldwide. GSK also purchased 6.9 million shares of Amicus stock for $4.56 per share. That gives the pharma giant 19.9% ownership of Amicus.

GSK will collaborate on development of the Phase III drug, and gains worldwide rights to develop, manufacture and commercialize Amigal. GSK and Amicus will also explore co-administration of Amigal with enzyme replacement therapy (ERT) for Fabry disease. A Phase III study of the drug began last year; GSK and Amicus expect preliminary results from that trial in the second half of 2011. A separate late-stage trial will begin this year.

"This strategic collaboration is another significant milestone in delivering our vision for GSK Rare Diseases. Amicus' scientific and clinical expertise in human genetic diseases is among the best in the industry, and we are pleased to be collaborators and investors in this exceptional company" said Marc Dunoyer, Global Head of GSK Rare Diseases, in a statement. "Our focus now is to continue to advance Amigal for Fabry disease and it is our hope to deliver a first-in-class, oral medicine to the thousands of people worldwide living with this devastating rare disease."

- here's the GSK release