Strensiq (asfotase alfa) — CareFirst (Caremark)

Perinatal/Infantile-onset hypophosphatasia (HPP)

Initial criteria

Member has clinical signs and/or symptoms of hypophosphatasia.
The onset of the disease was perinatal/infantile or juvenile.
If member age ≥ 18 years, documentation of presence of the condition before age 18 must be provided.
Diagnosis confirmed by either of the following:
— Pathological mutation in the ALPL gene as detected by ALPL molecular genetic testing OR
— Radiographic imaging demonstrating skeletal abnormalities, AND serum alkaline phosphatase (ALP) level below gender- and age-specific reference range, AND elevated tissue-nonspecific alkaline phosphatase (TNSALP) substrate level (e.g., serum PLP, urine PEA, or urinary/plasma PPi).
Member’s weekly dose will not exceed 9 mg/kg weekly for perinatal/infantile-onset HPP or 6 mg/kg weekly for juvenile-onset HPP.

Member is currently receiving the medication and is experiencing benefit from therapy as demonstrated by one or more of the following:
— Improvement in skeletal manifestations from baseline on the Radiographic Global Impression of Change (RGI-C) scale.
— For members age < 18 years, improvement in height and weight compared to baseline as measured by z-scores.
— Improvement in step length by at least 1 point in either foot compared to baseline on the Modified Performance Oriented Mobility Assessment-Gait (MPOMA-G) scale.
— Improvement in 6 Minute Walk Test (6MWT) compared to baseline.
— Improvement in Timed Up & Go (TUG) Test compared to baseline.
— Improvement in Chair Rise Test compared to baseline.
— Improvement in Lower Extremity Function Scale (LEFS) compared to baseline.
Member’s weekly dose will not exceed 9 mg/kg weekly for perinatal/infantile-onset HPP or 6 mg/kg weekly for juvenile-onset HPP.

12 months