Pombiliti — Medical Mutual

Acid Alpha-Glucosidase Deficiency (Pompe Disease)

Initial criteria

Patient age ≥ 18 years; AND
Patient weighs > 40 kg; AND
The medication will be used in combination with Opfolda; AND
Patient has not demonstrated an improvement in objective measures after receiving one of the following for at least one year (Lumizyme intravenous infusion OR Nexviazyme intravenous infusion); AND
Patient has late-onset acid alpha-glucosidase deficiency (late-onset Pompe disease) with diagnosis established by a laboratory test demonstrating deficient acid alpha-glucosidase activity in blood, fibroblasts, or muscle tissue OR a molecular genetic test demonstrating acid alpha-glucosidase gene mutation; AND
The medication is prescribed by or in consultation with a geneticist, neurologist, metabolic disorder sub-specialist, or physician specializing in lysosomal storage disorders.

Absence of unacceptable toxicity from the drug (e.g., anaphylaxis, 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 forced vital capacity (FVC) and/or six-minute walk test (6-MWT); AND
Patient is being monitored for antibody formation (including neutralizing antibodies).

1 year