Product Details

Galafold

Migalastat
123 mg
Capsule


DIN/PIN/NPN

02468042

Manufacturer

Innomar Strategies, Inc.

Formulary Listing Date

2019-05-13  

Unit Price

1700.0000

Amount MOH Pays

1700.0000

Coverage Status

Exceptional Access Program Product

ODB Formulary Therapeutic Classification

Therapeutic Note

NO

ATC Code

A16AX14

Interchangeable Products

NO  

LU Clinical Criteria

NO  

EAP Criteria

Therapeutic Class Reimbursement Criteria
Metabolic/Genetic Modifiers

Migalastat

  • Brand(s): Galafold
  • Dosage Form/Strength: 123 mg capsule

EAP Initiation criteria: 

For the treatment of Fabry disease (FD) in adult patients who meet ALL the following criteria:

  1. Has a confirmed diagnosis of Fabry disease (deficiency of α-galactosidase A [α-Gal A]) and must be otherwise eligible for enzyme replacement therapy (ERT) for the treatment of FD as determined by a panel of Fabry disease experts consistent with criteria for diagnosis from the Canadian Fabry Disease Initiative (CFDI)1 

  1. Has an α-galactosidase A mutation that is determined to be amenable to migalastat by an in vitro assay2 

  1. Migalastat is not being used concomitantly with ERT3. 

  1. Patient is considered to be compliant/adherent to treatment 

  1. Prescriber must be an expert in genetic disorders or a clinician experienced in the diagnosis and management of Fabry disease. 

1Refer to Canadian Fabry Disease treatment guidelines 2017 at http://www.garrod.ca/wp-content/uploads/Canadian-FD-Treatment-Guidelines-2017.pdf
Information submitted with requests to support the diagnosis of FD should include: 

  1. Clinical features associated with FD 

  1. Biochemical markers (e.g., alpha-galactosidase activity in plasma or leukocytes, elevated plasma and/or urine biomarkers) 

  1. Molecular changes 

  1. Pathologic findings (e.g., biopsy results from involved tissues) 

2Definition of an amenable mutation: mutation that increases activity of alpha galactosidase A in an in vitro cell culture system (human embryonic kidney or HEK cells) by 1.2 times the baseline activity with an absolute value for enzyme activity of 3% or greater when compared with wildtype values.
You may refer to the following website http://canada.galafoldamenabilitytable.com/?validated=1&redirect=en&hcp=1&licensed=1  or other appropriate supplementary tables to determine amenable α-Gal A mutations to migalastat. 

3Enzyme replacement therapies (ERT) for Fabry disease (e.g., agalsidase alfa [Replagal, agalsidase beta [Fabrazyme]) 

Exclusion criteria: 

  • Individuals who do not have an amenable mutation to miglalastat 

  • If used concomitantly with an enzyme replacement therapy for FD 

  • Individuals with severe renal insufficiency (GFR below 30 mL/min/1.73m2) 

  • Individuals who are pregnant or nursing 

  • Individuals with poor adherence/compliance to treatment

Note: Patients should be monitored every 6 months or more frequently during the first 3 to 5 years of treatment. 

Recommended dose: 123 mg every other day

Approval duration: 6 months 

Renewal criteria: 

Renewals will be considered on a case-by-case basis in individuals who:

  • are adherent with treatment; and 

  • who demonstrate a response while on therapy as compared to baseline results; and 

  • who have not developed unacceptable toxicities to migalastat; and 

  • who continue not meet any of the exclusion criteria (see above) 

Requests for ongoing reimbursement should include information related to the patient’s renal and cardiac function, cerebrovascular events, hospitalizations or emergency room visits for FD-related issues, health related quality of life measures as evaluated through a valid HRQL test (SF-36), gastrointestinal symptoms, pain measures, and other relevant clinical outcomes from treatment. Please address the patient’s adherence/compliance with treatment and any adverse effects / toxicities from treatment with migalastat. The Fabry disease expert panel may be consulted for renewal recommendations. 

Approval duration of renewals: 1 year

EAP Drug Request Form:

Standard Form for EAP Drug Requests

Product Monograph

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