Summary

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• Strensiq (asfotase alfa) is indicated for the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).
• This summary is based on the review of three systematic review(s)/meta-analysis(es). ^[1-3]
• Asfotase alfa has demonstrated effectiveness in treating Hypophosphatasia (HPP) across various age groups, including infants, children, and adults, with normalization of TNSALP substrate concentrations (inorganic pyrophosphate [PPi] and pyridoxal 5'-phosphate [PLP]) observed over 5 years of treatment.
• Functional outcomes improved with asfotase alfa treatment, with the percentage predicted distance walked on the 6-Minute Walk Test remaining within the normal range for monoallelic patients and improving from below normal to normal for biallelic patients, with no clear differences in effectiveness between these genetic subgroups.
• The evidence suggests that pharmacologic intervention, including asfotase alfa, can lead to significant clinical improvement in HPP symptoms, particularly in managing the disease across different populations and genetic subgroups.
• The reviewed studies provide information on the effectiveness of asfotase alfa in treating Hypophosphatasia (HPP) across various population types and subgroups, including infants, children, adults, and genetic subgroups (biallelic and monoallelic ALPL variants). Variability in baseline characteristics (e.g., PPi and PLP concentrations) and clinical manifestations (e.g., fractures, delayed walking, bone deformities) was noted, but overall treatment effectiveness was consistent across these groups.