title: Asfotase Alfa in Perinatal/Infantile-Onset and Juvenile-Onset Hypophosphatasia: A Guide to Its Use in the USA.
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.
authors.affiliation: Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand. demail@springer.com.
authors.forename: Lesley J
authors.initials: LJ
authors.lastname: Scott
mesh_terms: D000469:Alkaline Phosphatase / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D000818:Animals; D056947:Enzyme Replacement Therapy / Q000379:methods*; D006801:Humans; D007014:Hypophosphatasia / Q000188:drug therapy*; D007074:Immunoglobulin G / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D013534:Survival; D014481:United States
chemical_list: D007074:Immunoglobulin G; D011993:Recombinant Fusion Proteins; D000469:Alkaline Phosphatase; C570710:asfotase alfa