Opfolda (miglustat) — Medica

Preferred products

• Lumizyme (alglucosidase alfa)
• Nexviazyme (avalglucosidase alfa-ngpt)

Initial criteria

• Patient is age ≥ 18 years
• Patient weighs > 40 kg
• The medication will be used in combination with Pombiliti (cipaglucosidase alfa)
• Patient has not demonstrated an improvement in objective measures (e.g., forced vital capacity, six-minute walk test) after receiving ONE of the following for at least 1 year: Lumizyme (alglucosidase alfa) intravenous infusion OR Nexviazyme (avalglucosidase alfa-ngpt) intravenous infusion
• 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 GAA gene variants
• Medication is prescribed by or in consultation with a geneticist, neurologist, metabolic disorder sub-specialist, or physician who specializes in treatment of lysosomal storage disorders

Approval duration

1 year