Opfolda (miglustat capsules – Amicus) — Cigna

Initial criteria

• Patient age ≥ 18 years; AND
• Patient weighs > 40 kg; AND
• Medication will be used in combination with Pombiliti; AND
• Patient has not demonstrated an improvement in objective measures after receiving ONE of the following for at least one year (Lumizyme [alglucosidase alfa] intravenous infusion OR Nexviazyme [avalglucosidase alfa-ngpt] intravenous infusion); AND
• Patient has late-onset acid alpha-glucosidase deficiency (late-onset Pompe disease) with diagnosis established by ONE of the following (laboratory test demonstrating deficient acid alpha-glucosidase activity in blood, fibroblasts, or muscle tissue OR molecular genetic test demonstrating biallelic pathogenic or likely pathogenic acid alpha-glucosidase [GAA] gene variants); AND
• Medication is prescribed by or in consultation with a geneticist, neurologist, metabolic disorder sub-specialist, or physician who specializes in the treatment of lysosomal storage disorders

Approval duration

1 year