- Walgreens partners with United Nations Foundation's Shot@Life campaign
- Crossmark names Janet Carter-Smith VP
- Study: Cough-cold manufacturers' voluntary label change on pediatric products reduced ER admissions
- Cardinal Health invites independent pharmacy owners to ‘discover new horizons’ at RBC 2013
- Walgreens partners with Chicago Public Schools and city health department on Tdap vaccination project
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.