Opfolda (miglustat) for Pompe disease
Prior authorization policy describing coverage criteria for Opfolda (miglustat capsules) as enzyme stabilizer therapy in combination with Pombiliti for late-onset Pompe disease in adults >40 kg, including required diagnostic evidence, prescriber specialty, duration of approval, and non-covered indications.
Genetic testing language rephrased to specify 'biallelic pathogenic or likely pathogenic acid alpha-glucosidase gene variants'.
Annual revision on 05/07/2025 noted; 'No criteria changes' for that revision.
Coverage Summary
Prior authorization policy describing coverage criteria for Opfolda (miglustat capsules) as an enzyme stabilizer to be used in combination with Pombiliti for late-onset Pompe disease (acid alpha-glucosidase deficiency) in adults weighing > 40 kg. Approvals are provided for 1 year. The policy requires diagnostic evidence and prescriber specialty involvement and specifies that Opfolda has no monotherapy activity for Pompe disease; indications other than the FDA-approved use (for example, Gaucher disease) are not covered.
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