Medication leaflets

Active substance

Results for the substance asfotase alfa

4 results

About this active substance

Asfotase alfa is an enzyme replacement therapy used in the treatment of hypophosphatasia (HPP), a rare metabolic disorder caused by genetic mutations affecting the activity of tissue-nonspecific alkaline phosphatase (TNSALP). This enzyme plays a crucial role in the mineralization of bones and teeth, and its deficiency leads to bone deformities, fractures, and other severe complications.

Asfotase alfa is administered as subcutaneous injections, usually 3-6 times per week, depending on the severity of the disease and the patient's response to treatment. It is indicated for patients with perinatal, infantile, or juvenile-onset HPP to improve bone mineralization and reduce associated complications.

Common side effects include injection site reactions, fever, skin irritation, and bone pain. In rare cases, severe allergic reactions or ectopic calcifications may occur. Regular monitoring of bone and metabolic parameters is essential during treatment.

Asfotase alfa represents an innovative therapy that has significantly improved the prognosis for patients with hypophosphatasia, offering a chance for a healthier and more active life.