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Strensiq improves bone healing in children with hypophosphatasia

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Children with perinatal- and infantile-onset hypophosphatasia treated with Strensiq saw sustained improvements in bone healing and physical function over 3 years, according to findings from an ongoing trial presented at the Endocrine Society Annual Meeting in Boston.

Hypophosphatasia (HPP) is a rare metabolic disease characterized by low alkaline phosphatase activity and defective bone mineralization that can lead to bone deformity and other skeletal abnormalities, as well as muscle weakness, seizures, pain and respiratory failure.

In presenting findings from an ongoing, open-label, phase 2 trial of Strensiq (asfotase alfa, Alexion Pharmaceuticals), researchers said children aged 11 to 17 years with HPP treated with asfotase alfa therapy for up to 5 years (n = 12) experienced improvements in both bone healing (assessed radiographically) and physical function. In addition, new interim results indicated a reduction in two key tissue nonspecific alkaline phosphatase (TNSALP) substrates that accumulate in HPP, as well as improvements in physical function in both adolescent and adult patients with HPP (ages 13 to 66 years at baseline).

“The study results presented at ENDO show clinically significant and sustained improvements across all key measures of pediatric-onset HPP, including bone healing, physical function and pain in patients who were treated with Strensiq,” Martin Mackay, PhD, executive vice president and global head of research and development at Alexion, said in a press release. “The data provide additional information on the long-term efficacy and safety of Strensiq, a highly innovative enzyme replacement therapy that treats the underlying cause of HPP by replacing the missing TNSALP enzyme.”

Strensiq is approved in the United States as a treatment for patients with perinatal-, infantile- or juvenile-onset HPP.

Reference :

Liese J, et al. PP26-3. Presented at: The Endocrine Society Annual Meeting; April 1-4, 2016; Boston.

Disclosure: The authors are all employees, clinical researchers or speakers for Alexion Pharmaceuticals.