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efzimfotase alfa
- Development stage
- Phase 3
- Lead developer
- Alexion Pharmaceuticals
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Administration
- Subcutaneous
01
Overview
Efzimfotase alfa (ALXN1850) is a second-generation tissue-nonspecific alkaline phosphatase (TNSALP) enzyme replacement therapy developed for the treatment of hypophosphatasia (HPP), a rare inherited metabolic disorder characterized by deficient activity of TNSALP leading to impaired bone mineralization. Efzimfotase alfa is a recombinant fusion protein designed to increase drug exposure and enhance enzyme activity compared to existing therapies such as asfotase alfa. It is administered via subcutaneous injection and is currently in phase 3 clinical trials in adolescents and adults with HPP who have not previously been treated with asfotase alfa[1][2][6][8].
- Other names
- efzimfotase alfa
02
Targets
ALPL (Tissue-nonspecific alkaline phosphatase)
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