WASHINGTON (dpa-AFX) - Alexion Pharmaceuticals, Inc. (ALXN) Sunday said researchers presented new data from an integrated analysis of survival from two open-label, Phase 2 studies of asfotase alfa in pediatric patients, aged up to 5 years at enrollment, with hypophosphatasia or HPP compared with data from a retrospective natural history study of untreated historical control patients matched for age and disease severity.
Survival in patients with HPP at high risk of death who were treated with asfotase alfa for up to five years was significantly improved compared with untreated historical control patients.
These late-breaking results were presented at the American Society for Bone and Mineral Research 2014 Annual Meeting in Houston.
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.
HPP is a genetic, chronic and progressive ultra-rare metabolic disease that can lead to progressive damage to multiple vital organs, destruction and deformity of bones and premature death. Asfotase alfa is an investigational enzyme replacement therapy for the treatment of HPP.
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