Pombiliti (cipaglucosidase alfa-atga) — Medica
Acid Alpha-Glucosidase Deficiency (Pompe Disease)
Preferred products
- Lumizyme (alglucosidase alfa)
- Nexviazyme (avalglucosidase alfa-ngpt)
Initial criteria
- Patient is age ≥ 18 years
- Patient weighs > 40 kg
- Medication will be used in combination with Opfolda (miglustat capsules)
- Patient has not demonstrated improvement in objective measures 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 acid alpha-glucosidase (GAA) gene variants
- 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