Vol. 5 No. 12 (2025)
Reimbursement Reviews

Cipaglucosidase Alfa (Pombiliti) With Miglustat (Opfolda)

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Published December 22, 2025

Key Messages

  • Reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class.
  • The assessments inform nonbinding recommendations that help guide the reimbursement decisions of Canada’s federal, provincial, and territorial governments, with the exception of Quebec.
  • This review assesses cipaglucosidase alfa for injection (Pombiliti), lyophilized powder, 105 mg/vial, IV infusion in combination with miglustat capsules (Opfolda), 65 mg, oral capsules.
  • Indication: Cipaglucosidase alfa: Indicated in combination with the enzyme stabilizer Opfolda (65 mg miglustat capsule) for the treatment of adult patients with late-onset Pompe disease (acid alpha-glucosidase [GAA] deficiency) weighing ≥ 40 kg. Miglustat: An enzyme stabilizer indicated in combination with Pombiliti (cipaglucosidase alfa) for the treatment of adult patients with late-onset Pompe disease (acid alpha-glucosidase [GAA] deficiency) weighing ≥ 40 kg. Cipaglucosidase alfa must be used in combination with 65 mg miglustat capsules.