Strensiq logo Study design STRENSIQ® (asfotase alfa) is indicated for long-term enzyme replacement therapy in patients with paediatric-onset hypophosphatasia (HPP) to treat the bone manifestations of the disease.1 To date, this is the first and only approved treatment in this indication,2–4 supported by up to 7 years of clinical data.2–7

This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Please report any adverse events via your national reporting system. Adverse events can also be reported to Alexion Pharmaceuticals by contacting:
https://contactazmedical.astrazeneca.com.
Please refer to the Summary of Product Characteristics for further information.
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The efficacy and safety profile of STRENSIQ® was assessed in clinical trials with patients, of various ages, who had HPP2–6
Icon of a conical flask next to a medical certificate Icon of a clock Icon of a skeleton torso Icon of a person being measured
Icon of a conical flask next to a medical certificate Icon of a clock Icon of a skeleton torso Icon of a person being measured
Icon of a conical flask next to a medical certificate Icon of a clock Icon of a skeleton torso Icon of a person being measured
Icon of a conical flask next to a medical certificate Icon of a clock Icon of a skeleton torso Icon of a person being measured
* 10 patients completed a 6-month treatment period and entered the extension phase of this study.2

Nine patients reached adolescence (13–17 years old) during the study.1

After 1 month of treatment, one patient withdrew from the study for elective scoliosis surgery.6

§ Exploratory efficacy measures included motor function, muscle strength, patient reported functional disability and patient reported pain.7
Adapted from Whyte MP, et al. J Bone Miner Res. 2018;33(5):868–874.10
  • The Radiographic Global Impression of Change (RGI-C) scale is used to assess changes in the skeletal burden of paediatric patients with HPP, and quantifies change in mineralisation and bone structure over time10
  • The 7-point scale ranges from -3 (severe deterioration) to +3 (complete or near-complete healing)10
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BOT-2, Bruininks-Oseretsky Test of Motor Proficiency, 2nd Edition; HPP, hypophosphatasia; PLP, plasma/serum pyridoxal 5′-phosphate; PPi, inorganic pyrophosphate; RGI-C, Radiographic Global Impression of Change; RSS, Rickets Severity Scale; TEAE, treatment-emergent adverse event.
This medicinal product has been authorised under ‘exceptional circumstances’. This means that due to the rarity of the disease it has not been possible to obtain complete information on this medicinal product.

▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Please report any adverse events via your national reporting system. Adverse events can also be reported to Alexion Pharmaceuticals by the following link: https://contactazmedical.astrazeneca.com/
STRENSIQ® Summary of Product Characteristics. Alexion Europe SAS. Available at https://www.ema.europa.eu/en/documents/product-information/strensiq-epar-product-information_en.pdf. Last accessed: June 2025. Whyte MP, et al. Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial. Lancet Diabetes Endocrinol. 2019;7(2):93–105. European Medicines Agency. EPAR for Strensiq. 2024. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/strensiq#product-info. Last accessed: June 2025. Jaswanthi N, et al. Effect of Asfotase Alfa in the Treatment of Hypophosphatasia- A Systematic Review. J Pharm Bioallied Sci.2023;15(Suppl 1):S101-S104. Hofmann CE, et al. Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study. J Clin Endocrinol Metab. 2019;104(7):2735–2747.. Whyte MP, et al. Asfotase alfa therapy for children with hypophosphatasia. JCI Insight. 2016;1(9):e85971. Kishnani PS, et al. Five-year efficacy and safety of asfotase alfa therapy for adults and adolescents with hypophosphatasia. Bone. 2019;121:149–162. Whyte MP, et al. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012;366(10):904–913. Asfotase Alfa (Strensiq). Ottawa (ON): Canadian Agency for Drugs and Technologies in Health. 2017. Appendix 4, Additional Clinical Evidence. Available at: https://www.ncbi.nlm.nih.gov/books/NBK476066/. Last accessed: June 2025. Whyte MP, et al. Validation of a Novel Scoring System for Changes in Skeletal Manifestations of Hypophosphatasia in Newborns, Infants, and Children: The Radiographic Global Impression of Change Scale. J Bone Miner Res. 2018;33(5):868–874. Seefried L, et al. Burden of Illness in Adults With Hypophosphatasia: Data From the Global Hypophosphatasia Patient Registry. J Bone Miner Res. 2020;35(11):2171–2178.