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asfotase alfa
- Development stage
- Approved
- Lead developer
- Alexion Pharmaceuticals
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Subcutaneous
01
Overview
Asfotase alfa is a recombinant human enzyme used as an enzyme replacement therapy for the treatment of perinatal, infantile, and juvenile-onset hypophosphatasia (HPP), a rare genetic disorder characterized by deficient activity of tissue-nonspecific alkaline phosphatase (TNSALP). The drug is designed to replace the deficient TNSALP enzyme, thereby reducing elevated levels of its substrates such as inorganic pyrophosphate (PPi) and pyridoxal 5'-phosphate (PLP), which interfere with bone mineralization. By restoring TNSALP activity, asfotase alfa improves bone mineralization and reduces skeletal abnormalities associated with HPP. It is administered via subcutaneous injection and was developed by Alexion Pharmaceuticals[1][3][6][7][8].
- Brand names
- Strensiq
- Other names
- asfotase alfa
02
Targets
ALPL (Tissue-nonspecific alkaline phosphatase)
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