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

late-onset Pompe disease (acid maltase deficiency; glycogen storage disease type II)

Initial criteria

  • ONE of the following: A. continuation of therapy if patient treated within past 90 days and at risk if changed OR B. ALL of the following:
  • Diagnosis of late-onset Pompe disease confirmed by genetic analysis showing biallelic pathogenic GAA variants OR deficient acid alpha-glucosidase enzyme activity
  • Patient not improving on current enzyme replacement therapy (ERT)
  • Patient weight ≥ 40 kg
  • Requested agent taken in combination with Pombiliti
  • Patient's age within FDA labeling OR supported for use at that age