Drug class

Metabolic Disease Enzyme Replacement - Hypophosphatasia

Metabolic disease enzyme replacement therapies for hypophosphatasia represent a specialized pharmacologic category of recombinant enzymes indicated for patients with hypophosphatasia. Read more about Metabolic Disease Enzyme Replacement - Hypophosphatasia

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STRENSIQ (asfotase alfa)

METABOLIC DISEASE ENZYME REPLACEMENT - HYPOPHOSPHATASIA · CHILDHOOD HYPOPHOSPHATASIA ·+1
ALEXION PHARMAC
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About Metabolic Disease Enzyme Replacement - Hypophosphatasia.

Metabolic disease enzyme replacement therapies for hypophosphatasia represent a specialized pharmacologic category of recombinant enzymes indicated for patients with hypophosphatasia.

These agents—most notably bone-targeted tissue-nonspecific alkaline phosphatase fusion proteins such as asfotase alfa—work by directly replacing the deficient endogenous alkaline phosphatase enzyme.

By hydrolyzing extracellular inorganic pyrophosphate, an inhibitor of bone mineralization, they restore physiological calcium and phosphate deposition into hydroxyapatite crystals.

Clinically, this class is utilized to treat perinatal-, infantile-, and juvenile-onset hypophosphatasia to improve skeletal mineralization, reduce rickets and fracture risk, and prevent systemic metabolic complications.