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.
Last review date: June 1, 2024