Alexion Pharmaceuticals Inc. Release: Improved Survival Observed In Pediatric Patients With Severe Hypophosphatasia (HPP) Who Were Treated With Investigational Asfotase Alfa For Up To Five Years

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CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that researchers presented new data from an integrated analysis of survival from two open-label, Phase 2 studies of asfotase alfa in pediatric patients (ages =5 years at enrollment) with hypophosphatasia (HPP) compared with data from a retrospective natural history study of untreated historical control patients matched for age and disease severity. In this analysis, survival in patients with HPP at high risk of death who were treated with asfotase alfa for up to five years was significantly improved (89% vs. 27%, p<0.0001) compared with untreated historical control patients.1 These late-breaking results were presented today at the American Society for Bone and Mineral Research (ASBMR) 2014 Annual Meeting in Houston, where researchers also presented new data from the ongoing open-label extension phases of two Phase 2 clinical studies in which sustained gains in physical function and reductions in disability and pain were observed in pediatric patients receiving asfotase alfa treatment for up to three years.2,3

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