Drugdocs | Strensiq®

Drug updated on 4/17/2024

Dosage Form	Injection (subcutaneous; 18 mg/0.45 mL, 28 mg/0.7 mL, 40 mg/mL, 80 mg/0.8 mL)
Drug Class	Tissue nonspecific alkaline phosphatases
Ongoing and Completed Studies	ClinicalTrials.gov

Indication

For the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP)

Asfotase alfa (Strensiq) is used for the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).
Three systematic reviews/meta-analyses were reviewed, providing insights into the effectiveness and safety of asfotase alfa in treating HPP.
The first study found that enzyme replacement therapy using asfotase alfa effectively treats infants, children, and adults suffering from HPP by increasing their lifespan.
A post hoc analysis pooled data from two phase 2 studies to investigate clinical outcomes with asfotase alfa in relation to ALPL variant state; it was found that both biallelic and monoallelic variants showed similar pretreatment baseline clinical characteristics but those with biallelic variants had significantly higher Baseline PPi and PLP levels than monoallelic variants.
Despite these differences at baseline between biallelic or monoallelic variant states, treatment with asfotase alfa for up to 5 years normalized TNSALP substrate concentrations and improved functional outcomes without clear differences between these groups.
The third review emphasized the significant morbidity associated with HPP due to loss of function mutations in the ALPL gene causing reductions in alkaline phosphatase activity; pharmacologic intervention such as Strensiq can result in significant clinical improvement across various manifestations including musculoskeletal, renal, dental respiratory,and neurologic symptoms related to this disorder.

Document Title	Year	Source
Strensiq (asfotase alfa) Prescribing Information.	2020	FDA

Document Title	Year	Source
Effect of Asfotase Alfa in the Treatment of Hypophosphatasia- A Systematic Review	2023	Journal of pharmacy & bioallied sciences
Investigation of ALPL variant states and clinical outcomes: An analysis of adults and adolescents with hypophosphatasia treated with asfotase alfa	2021	Molecular genetics and metabolism
Hypophosphatasia: Canadian update on diagnosis and management	2019	Osteoporosis international

Document Title	Year	Source
Clinical practice guidelines for hypophosphatasia.	2020	Clinical Pediatric Endocrinology