miglustat (gaa deficiency) — Blue Cross Blue Shield of New Mexico

Initial criteria

• Requested agent eligible for continuation of therapy AND prescriber states patient treated with requested agent within past 90 days AND at risk if therapy changed OR all the following:
• Diagnosis of late-onset Pompe disease confirmed by genetic analysis with biallelic mutation in GAA gene OR deficient acid alpha-glucosidase glycogen enzyme activity in dried blood spots, leukocytes, skin fibroblasts, and/or skeletal muscle tissue
• Patient is not improving on current enzyme replacement therapy (ERT)
• Patient weighs ≥40 kg
• Requested agent will be taken in combination with Pombiliti
• If FDA-labeled indication THEN age within labeling OR support for use for patient’s age