Alexion Reports Better Muscle Strength In HPP Children On Asfotase Alfa

(RTTNews) - Alexion Pharmaceuticals, Inc. (ALXN) said researchers presented new data from a retrospective, multinational natural history study of children with hypophosphatasia or HPP.

HPP is a genetic, chronic and progressive ultra-rare metabolic disease characterized by defective bone mineralization that can lead to repeated fractures and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.

In this study, which included 32 patients with juvenile-onset HPP, children with HPP had a substantial disease burden, particularly with regard to musculoskeletal abnormalities and growth deficiencies.

The children from this cohort experienced HPP-related skeletal problems, other disease complications and morbidity that persisted despite standard efforts to control symptoms.

Researchers reported results from the extension phase of a multinational, open-label Phase 2 study in children with HPP who were treated with asfotase alfa for at least three years. New data showed hip and knee strength improved over time.

Dawn Phillips, Physical Therapist and Functional Outcomes Specialist, UNC Division of Physical Therapy, Chapel Hill, N.C., said, "At the start of this study, children with HPP had substantial muscle weakness and impaired function compared with healthy peers. During the treatment period with asfotase alfa, patients experienced rapid and sustained improvements in muscle strength and physical function, allowing them to engage in physical activity more typical of healthy children their age."

The most common adverse events were injection site reactions.

These data were presented in a late-breaking oral session at the Endocrine Society's 97th Annual Meeting and Expo or ENDO in San Diego.