Alexion announced that the Food and Drug Administration (FDA) has approved Strensiq (asfotase alfa) for the treatment of patients with perinatal-, infantile-, and juvenile-onset hypophosphatasia (HPP). Strensiq is the first treatment approved for the treatment of HPP, a genetic and chronic ultra-rare metabolic disorder.

Strensiq is a bone-targeted enzyme replacement therapy that works by replacing the missing TNSALP enzyme.

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The FDA’s approval was based on data from four clinical trials and supporting extension trials comprising patients with perinatal-, infantile-, and juvenile-onset HPP who were treated with Strensiq for up to 6.5 years (n=99). In the study, patients with HPP treated with Strensiq experienced significant survival benefit vs. historical control patients with similar clinical characteristics. At Week 48, overall survival was 97% for treated patients (n=68) vs. 42% for historical control patients (n=48). Estimated invasive ventilator-free survival was 96% for treated patients (n=54) vs. 31% for historical control patients (n=48). Also, substantial improvements in skeletal manifestations of HPP, as assessed by RGI-C scale, as well as improvements in height and weight, as measured by z-score were also seen in patients treated with Strensiq.

Strensiq was approved under Priority Review and was granted Breakthrough Therapy designation. The FDA has also issued a Rare Pediatric Disease Priority Review Voucher.

Strensiq will be available as 18mg/0.45mL, 28mg/0.7mL, 40mg/mL, and 80mg/0.8mL strengths in 1- or 12-count single-use vials. Strensiq is preservative-free and will launch by October 27, 2015.

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