Strensiq (asfotase alfa) — Medica

Hypophosphatasia – perinatal/infantile- and juvenile-onset

Initial criteria

Patient meets ALL of the following (A, B, C, and D):
A) Diagnosis is supported by ONE of the following (i, ii, or iii):
i. Molecular genetic testing documenting pathogenic tissue non-specific alkaline phosphatase (ALPL) gene variants; OR
ii. Low baseline serum alkaline phosphatase activity; OR
iii. An elevated level of a tissue non-specific alkaline phosphatase substrate (e.g., serum pyridoxal 5’-phosphate, serum or urinary inorganic pyrophosphate, urinary phosphoethanolamine); AND
B) Patient meets ONE of the following (i or ii):
i. Patient currently has, or had a history of, clinical manifestations consistent with hypophosphatasia (e.g., skeletal abnormalities, premature tooth loss, muscle weakness, poor feeding, failure to thrive, respiratory problems, vitamin B6-dependent seizures); OR
ii. Patient has a family history (parent or sibling) of hypophosphatasia without current clinical manifestations of hypophosphatasia; AND
C) Disease onset < 18 years of age; AND
D) The medication is prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder sub-specialist, or a physician who specializes in the treatment of hypophosphatasia or related disorders.

1 year