Opfolda — Medical Mutual

Acid Alpha-Glucosidase Deficiency (Pompe Disease)

Initial criteria

Patient is age ≥ 18 years; AND
Patient weighs > 40 kg; AND
The 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 IV OR Nexviazyme IV). Note: Examples of objective measures include forced vital capacity (FVC) and six-minute walk test (6MWT); AND
Patient has late-onset acid alpha-glucosidase deficiency (late-onset Pompe disease) with diagnosis established by either: laboratory test demonstrating deficient acid alpha-glucosidase activity in blood, fibroblasts, or muscle tissue OR molecular genetic test demonstrating acid alpha-glucosidase gene mutation; AND
The medication is prescribed by or in consultation with a geneticist, neurologist, a metabolic disorder sub-specialist, or a physician who specializes in the treatment of lysosomal storage disorders

Absence of unacceptable toxicity from the drug (examples include anaphylaxis and severe hypersensitivity reactions, severe infusion-associated reactions, acute cardiorespiratory failure, etc.); AND
Patient has demonstrated a beneficial response to therapy compared to pretreatment baseline in one or more of the following: stabilization or improvement in FVC and/or 6-MWT; AND
Patient is being monitored for antibody formation (including neutralizing antibodies)

1 year