miglustat (gaa deficiency) — Blue Cross Blue Shield of Illinois

Initial criteria

• ONE of the following: (A) The requested agent is eligible for continuation of therapy AND prescriber states the patient has been treated with the requested agent (not on samples) within the past 90 days AND is at risk if therapy is changed OR (B) ALL of the following:
• Diagnosis of late-onset Pompe disease confirmed by ONE of the following: (A) Genetic analysis confirms biallelic mutation (two pathogenic variants) in the GAA gene OR (B) Deficient acid alpha-glucosidase enzyme activity in dried blood spots, leukocytes, skin fibroblasts, and/or skeletal muscle tissue
• Patient is not improving on their current enzyme replacement therapy (ERT)
• Patient weight ≥ 40 kg
• Requested agent will be taken in combination with Pombiliti
• If the patient has an FDA labeled indication, ONE of the following: (A) Patient’s age is within FDA labeling for indication OR (B) There is support for using the requested agent for the patient’s age for the requested indication