By replacing alkaline phosphatase, Strensiq® treats the underlying cause of hypophosphatasia (HPP).1
The underlying cause of hypophosphatasia is deficient tissue-nonspecific alkaline phosphatase (TNSALP) activity.1
Hypophosphatasia is a lifelong disease caused by loss-of-function mutations in the ALPL gene, which encodes the tissue-nonspecific form of alkaline phosphatase (TNSALP).3-5 Deficient TNSALP leads to elevations in several TNSALP substrates (eg, inorganic pyrophosphate), causing rickets and bone deformation in infants and children and osteomalacia (softening of bones) once growth plates close, along with muscle weakness.1
Patients with hypophosphatasia suffer from a variety of skeletal manifestations.3 In a historical cohort of untreated patients with life-threatening perinatal/infantile-onset hypophosphatasia, >50% of patients died by the first year of life. The primary cause of death in the cohort was respiratory complications and/or failure.6 Patients with hypophosphatasia who survive into childhood and adolescence can suffer from skeletal issues, growth delays, and limited mobility.1,3,4,7,8
Strensiq® replaces the deficient TNSALP enzyme and reduces the enzyme substrate levels.1
In Strensiq clinical trials, mean alkaline phosphatase levels varied across populations studied and increased typically well above the upper limit of normal9
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The levels remained elevated during the treatment period, reflecting ongoing treatment with Strensiq9
Treatment with Strensiq lowered plasma levels of the alkaline phosphatase substrates inorganic pyrophosphate (PPi) and pyridoxal 5’-phosphate (PLP) within 6 to 12 weeks of treatment1
- Reductions in PPi and PLP did not correlate with clinical outcomes1