GSK Collaborates With Amicus On A Rare Disease

GlaxoSmithKline and Amicus Therapeutics have agreed to develop and commercialize Amicus' investigative treatment for Fabry disease, migalastat, which is currently in Phase III clinical trials.

Under the terms of the agreement, GSK will receive an exclusive worldwide license to develop, manufacture, and commercialize migalastat. The two companies will also launch new clinical studies on coadministration of migalastat with enzyme replacement therapy for treatment of the disease.

Fabry disease is a rare inherited lysosomal storage disorder caused by deficiency of α-galactosidase A, which in turn leads to buildup of a lipid called globotriaosylceramide. This accumulation is believed to cause symptoms associated with the disease, including pain, kidney failure, and increased risk of heart attack and stroke.

Amicus, based in Cranbury, N.J., will receive an upfront license payment of $30 million from GSK and may receive an additional $170 million in development and commercialization milestones. GSK is also purchasing a 19.9% ownership stake in Amicus for $31 million.

Migalastat is a small-molecule "pharmaceutical chaperone" designed to selectively bind to and stabilize α-galactosidase A, its target enzyme. "This strategic collaboration is another significant milestone in delivering our vision for GSK Rare Diseases," says Marc Dunoyer, head of GSK's rare diseases unit, which launched in February. Dunoyer recently outlined his strategy for the unit (C&EN, Oct. 25, page 18). It will focus on 200 diseases in metabolism and inherited disorders, central nervous system and muscle disorders, immunoinflammation, and rare malignancies and hematology.