asfotase alfa — Blue Cross Blue Shield of Illinois

perinatal/infantile-onset hypophosphatasia (HPP)

Initial criteria

Diagnosis of perinatal/infantile- OR juvenile-onset hypophosphatasia (HPP)
Patient age at onset < 18 years [chart notes required]
Patient is experiencing active disease (e.g., bone pain, fractures, gait problems)
Patient has/had clinical manifestations consistent with HPP at age of onset prior to age 18 (e.g., vitamin B6-dependent seizures, fractures, lost teeth with roots, skeletal abnormalities like rachitic chest deformity leading to respiratory problems or bowed limbs, failure to thrive) [chart notes required]
ONE of the following [chart notes required]: Molecular genetic testing confirming mutations in ALPL gene encoding tissue nonspecific alkaline phosphatase (TNSALP) OR ALL of the following: radiographic imaging confirming HPP diagnosis at age of onset prior to age 18, reduced unfractionated serum ALP activity (below normal reference range without bisphosphonate therapy), and ONE of elevated serum pyridoxal 5'-phosphate (PLP) (without vitamin supplements within one week before test) OR elevated urine phosphoethanolamine (PEA) OR elevated urinary inorganic pyrophosphate (PPi)
Prescriber is a specialist in area of patient’s diagnosis (e.g., endocrinologist, geneticist) or has consulted with such specialist
Patient has had ophthalmology examination and renal ultrasound at baseline (before therapy)
Patient has no FDA-labeled contraindications to requested agent
Requested dose within FDA labeling based on patient weight OR supported higher dose for requested indication

Patient previously approved for requested agent through plan’s Prior Authorization process
Clinical improvement from baseline in at least ONE of: respiratory status (e.g., level of respiratory support required) OR growth (improvement in length/height, weight, or head circumference z-scores) OR radiographic findings (improvement in skeletal manifestations by RSS or RGI-C, or fewer fractures) OR level of activity (improvement in motor function or daily living activities) [chart notes required]
Prescriber is a specialist in patient’s diagnosis area (e.g., endocrinologist, geneticist) or has consulted with such specialist
Patient monitored for ophthalmic/renal calcifications and for changes in vision or renal function
No FDA-labeled contraindications to requested agent
Requested dose within FDA labeling based on patient weight OR supported higher dose for requested indication

12 months