Vol. 5 No. 8 (2025)
Reimbursement Recommendations

Cipaglucosidase Alfa (Pombiliti) With Miglustat (Opfolda)

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Published August 7, 2025

Key Messages

  • Canada’s Drug Agency (CDA-AMC) recommends that Pombiliti with Opfolda be reimbursed by public drug plans for the treatment of adult patients with late-onset Pompe disease (LOPD) weighing at least 40 kg if certain conditions are met.
  • Pombiliti with Opfolda should only be covered to treat patients who have a confirmed diagnosis of LOPD, are able to walk, and have a sitting forced vital capacity measurements of at least 30% of the predicted value for healthy adults. Pombiliti with Opfolda should not be covered to treat patients who have severe disease.
  • Pombiliti with Opfolda should only be reimbursed if prescribed by a clinician experienced in treating lysosomal storage diseases or other types of neuromuscular diseases, it is not used in combination with other enzyme replacement therapies for Pompe disease, and the cost of Pombiliti with Opfolda is reduced.