Drug developer Shire PLC said Feb. 11 its potential Gaucher disease drug showed positive results in a late-stage study.
Gaucher is a rare enzyme disorder that harms the body’s ability to process fat, and causes liver and possible neurological problems.
Its most common form, Type 1, is found in about one out of every 50,000 to 100,000 people worldwide, according to the National Institutes of Health. The genetic condition occurs more frequently in people of Ashkenazi Jewish heritage, affecting about one in 500 to 1,000 people, according to the NIH.
If approved, Shire’s drug would likely compete directly with Genzyme Corp.’s Cerezyme in the market. The Food and Drug Administration is scheduled to make a regulatory decision about Shire’s drug candidate, velaglucerase alfa, by Feb. 28.
Genzyme is developing another potential Gaucher disease drug, called eliglustat tartrate, formerly Genz-112638; it is in late-stage development. Another Gaucher drug on the market is Actelion Pharmaceuticals Ltd.’s Zavesca. — ap