Canada’s Drug Agency | Drugs, Health Technologies and Systems.

cipaglucosidase alfa with miglustat

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Generic Name:
cipaglucosidase alfa with miglustat
Project Status:
Active
Therapeutic Area:
Pompe disease
Manufacturer:
Amicus Therapeutics Canada Inc.
Call for patient/clinician input open:
Brand Name:
Pombiliti with Opfolda
Project Line:
Reimbursement Review
Project Number:
SR0871-000
Call for patient/clinician input closed:
NOC Status at Filing:
Pre NOC
Biosimilar:
No
Manufacturer Requested Reimbursement Criteria1:
Pombiliti (cipaglucosidase alfa for injection) is indicated in combination with the enzyme stabiliser OPFOLDA (65 mg miglustat capsule) for the treatment of adult patients with late-onset Pompe disease (acid -glucosidase [GAA] deficiency) weighing 40 kg. Opfolda (miglustat capsules) is an enzyme stabilizer indicated in combination with POMBILITI (cipaglucosidase alfa) for the treatment of adult patients with late-onset Pompe disease (acid -glucosidase [GAA] deficiency) weighing 40 kg.
Submission Type:
Initial
Fee Schedule:
Schedule A
Indications:
Pombiliti (cipaglucosidase alfa for injection) is indicated in combination with the enzyme stabiliser Opfolda (65 mg miglustat capsule) for the treatment of adult patients with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency) weighing ≥ 40 kg. Opfolda (miglustat capsules) is an enzyme stabilizer indicated in combination with Pombiliti (cipaglucosidase alfa) for the treatment of adult patients with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency) weighing ≥40 kg.
  1. The requested reimbursement criteria are provided by the applicant and do not necessarily reflect the views of Canada's Drug Agency. Reimbursement criteria from Canada's Drug Agency will be documented in the final recommendation, if applicable.
Key Milestones2
Call for patient/clinician input openOctober 23, 2024
Call for patient/clinician input closedDecember 13, 2024
Submission receivedNovember 29, 2024
Submission acceptedDecember 13, 2024
Review initiatedDecember 16, 2024
Draft CADTH review report(s) provided to sponsor for commentApril 09, 2025
Deadline for sponsors commentsApril 22, 2025
CDA-AMC review report(s) and responses to comments provided to sponsorMay 15, 2025
Expert committee meeting (initial)May 28, 2025
Draft recommendation issued to sponsorJune 09, 2025
To
June 11, 2025
Draft recommendation posted for stakeholder feedbackJune 19, 2025
End of feedback periodJuly 04, 2025

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Last Updated : April 17, 2025