[an error occurred while processing this directive]
Skip to Main Content

Latest News

Advertise Here
November 1, 2010

GSK Collaborates With Amicus On A Rare Disease

Pharmaceuticals: The partners will develop a small-molecule treatment for Fabry disease

Rick Mullin

  • Print this article
  • Email the editor

Latest News

October 28, 2011

Speedy Homemade-Explosive Detector

Forensic Chemistry: A new method could increase the number of explosives detected by airport screeners.

Solar Panel Makers Cry Foul

Trade: U.S. companies complain of market dumping by China.

Novartis To Cut 2,000 Jobs

Layoffs follow similar moves by Amgen, AstraZeneca.

Nations Break Impasse On Waste

Environment: Ban to halt export of hazardous waste to developing world.

New Leader For Lawrence Livermore

Penrose (Parney) Albright will direct DOE national lab.

Hair Reveals Source Of People's Exposure To Mercury

Toxic Exposure: Mercury isotopes in human hair illuminate dietary and industrial sources.

Why The Long Fat?

Cancer Biochemistry: Mass spectrometry follows the metabolism of very long fatty acids in cancer cells.

Text Size A A

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.

Chemical & Engineering News
ISSN 0009-2347
Copyright © 2011 American Chemical Society
  • Print this article
  • Email the editor

Services & Tools

ACS Resources

ACS is the leading employment source for recruiting scientific professionals. ACS Careers and C&EN Classifieds provide employers direct access to scientific talent both in print and online. Jobseekers | Employers

» Join ACS

Join more than 161,000 professionals in the chemical sciences world-wide, as a member of the American Chemical Society.
» Join Now!