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CRANBURY, N.J. British drug maker GlaxoSmithKline will work with U.S.-based Amicus Therapeutics to develop a drug for a rare genetic disease.
The two companies announced a deal to develop and commercialize Amigal (migalastat hydrochloride), a treatment for Fabry disease. Under the deal, GSK will pay Amicus $30 million upfront, as well as milestone payments of up to $170 million and royalties on future sales.
Fabry disease is a lysosomal storage disorder resulting from deficiencies of the enzyme alpha-galactosidase A. Lack of the enzyme results in buildup of a lipid called globotriaosylceramide, or GL-3, which is believed to cause the disease’s symptoms, such as pain, kidney failure and increased risk of heart attack and stroke. The disease affects 5,000 to 10,000 people worldwide.