Asfotase alfa (Strensiq)

概览

Asfotase alfa is an enzyme replacement therapy used for the treatment of perinatal/infantile and juvenile onset hypophosphatasia (HPP).

作用机制

HPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.

适应症

Indicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).

• Infantile-onset Hypophosphatasia
• Juvenile-onset Hypophosphatasia
• Perinatal-onset Hypophosphatasia