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.
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.
For more information call (888) 765-4747 or visit Strensiq.com.