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Asfotase Alfa in Perinatal/Infantile-Onset and Juvenile-Onset Hypophosphatasia: A Guide to Its Use in the USA

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Abstract

Subcutaneous asfotase alfa (Strensiq™), a first-in-class bone-targeted human recombinant tissue-nonspecific alkaline phosphatase (TNSALP) replacement therapy, is approved in the USA for the treatment of patients with perinatal/infantile- or juvenile-onset hypophosphatasia (HPP). In clinical trials, asfotase alfa was an effective and generally well tolerated treatment for perinatal/infantile- and juvenile onset-HPP through at least 3 and 5 years’ treatment, respectively. Relative to untreated age-matched, juvenile-onset-HPP historical control cohorts, survival and ventilation-free survival were significantly prolonged in asfotase alfa-treated patients, consequent to preceding improvements in bone mineralization.

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Acknowledgments

The review was adapted and updated from Drugs 2016;76(2);255–262 [1]. During the peer review process, the manufacturer of asfotase alfa was offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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  1. Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand

    Lesley J. Scott

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  1. Lesley J. Scott
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Correspondence to Lesley J. Scott.

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The preparation of this review was not supported by any external funding.

Conflicts of interest

Lesley Scott is a salaried employee of Adis/Springer, is responsible for the article content and declares no relevant conflicts of interest.

Additional information

The manuscript was reviewed by: M. L. Bianchi, Experimental Laboratory for Children’s Bone Metabolism Research, Bone Metabolism Unit, Istituto Auxologico Italiano IRCCS, Milan, Italy; C. Hofmann, Children’s Hospital University of Wurzburg, Wurzburg, Germany; C. R. Rockman - Greenberg, Department of Paediatrics and Child Health, University of Manitoba, Winnipeg, Canada.

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Scott, L.J. Asfotase Alfa in Perinatal/Infantile-Onset and Juvenile-Onset Hypophosphatasia: A Guide to Its Use in the USA. BioDrugs 30, 41–48 (2016). https://doi.org/10.1007/s40259-016-0161-x

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