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Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide.
A small number of adult patients who metabolize Cerdelga more quickly or at an undetermined rate, as detected by an established genetic laboratory test, will not be eligible for Cerdelga treatment.
Cerdelga was approved by the USFDA in August 2014, and is under review by other regulatory authorities around the world.
It is expected that Cerdelga will be available commercially in EU countries beginning in 2015 and over the next few years.
Cerdelga is a potent, highly specific ceramide analogue inhibitor of glucosylceramide synthase with broad tissue distribution including to bone marrow.
It reduces the production of glucosylceramide, the substance that builds up in the cells and tissues of people with Gaucher disease type 1.