POMBILITI ATGA (cipaglucosidase alfa-atga)

POMBILITI ATGA (cipaglucosidase alfa-atga)

Office-Administration – intravenous (IV) infusion

Diagnosis considered for coverage:

• Indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT)

Coverage Criteria:

For diagnosis of Late-Onset Pompe disease:

• Diagnosis of late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency); AND
• Disease is confirmed by one of the following:
  • Absence or deficiency (less than 40% of the lab specific normal mean) of GAA enzyme activity in lymphocytes, fibroblasts, or muscle tissues as confirmed by an enzymatic assay 
  • Molecular genetic testing confirms mutations in the GAA gene; AND
• Presence of clinical signs and symptoms of the disease (e.g., respiratory distress, skeletal muscle weakness, etc.); AND
• Medication is used in combination with Opfolda (miglustat); AND
• Patient weight is greater than or equal to 40 kg; AND
• Trial and inadequate response to one of the following:
  • Lumizyme 
  • Nexviazyme; AND
• Not to be used in combination with other miglustat products (i.e., Zavesca, Yargesa)

Reauthorization Criteria:

For diagnosis of Late-Onset Pompe disease:

• Patient demonstrates positive clinical response to therapy (e.g., improvement in FVC, improvement in 6-minute walk distance [6MWD]); AND
• Medication is used in combination with Opfolda (miglustat); AND
• Not to be used in combination with other miglustat products (i.e., Zavesca, Yargesa)

Dosing:

Late-Onset Pompe disease:

• 20 mg/kg (of actual body weight) administered every other week as an intravenous infusion
• Pombiliti must be administered in combination with Opfolda. If the Opfolda dose is missed, Pombiliti should not be administered

Coverage Duration:

• Initial: 1 year
• Reauthorization: 2 years

Authorization is not covered for the following:

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Additional Information: 

• Consensus recommendation based on current clinical guidelines indicate that treatment should be started in patients with late onset Pompe disease when they become symptomatic and/or show signs of disease progression
• Prior to Pombiliti administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. If premedication was used with previous enzyme replacement therapy (ERT), prior to Pombiliti administration, pretreat with antihistamines, antipyretics, and/or corticosteroids

Policy Updates:

• Effective 6/01/2024 – New policy approved by WHA P&T Committee. (P&T, 05/21/2024)

References:

• Pombiliti Prescribing Information. Amicus Therapeutics US, LLC. Philadelphia, PA. Sept 2023. 
• Diaz, C., Diaz-Manera, J. Therapeutic Options for the Management of Pompe Disease: Current Challenges and Clinical Evidence in Therapeutics and Clinical Risk Management. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9759116/. Accessed November 2, 2023. 
• Cleveland Clinic - Pompe Disease. Available at: https://my.clevelandclinic.org/health/diseases/15808-pompe-disease. Accessed November 2, 2023. 
• Cupler, E., Berger, K., Leshner, R., et al. Consensus Treatment Recommendations for Late-Onset Pompe Disease. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3534745/. Accessed November 2, 2023. 
• Barba-Romero MA, Barrot E, Bautista-Lorite J, et al. Clinical guidelines for late-onset Pompe disease. Available at: https://www.orpha.net/data/patho/Cpg/en/PompeLateOnset_ES_en_CPG_ORPHA420429.pdf. Accessed November 2, 2023.