Product Details

Orkambi

Ivacaftor + Lumacaftor
125 mg + 100 mg
Tablet


DIN/PIN/NPN

02463040

Manufacturer

Vertex Pharmaceuticals (Canada) Incorporated

Formulary Listing Date

2021-06-25  

Unit Price

170.5357

Amount MOH Pays

170.5357

Coverage Status

Exceptional Access Program Product

ODB Formulary Therapeutic Classification

Therapeutic Note

NO

ATC Code

R07AX30

Interchangeable Products

NO  

LU Clinical Criteria

NO  

EAP Criteria

Therapeutic Class Reimbursement Criteria
Respirology Therapies

Ivacaftor/Lumacaftor

  • Brand(s): Orkambi
  • Dosage Form/Strength: 100 mg / 125 mg tablets; 200 mg/ 125 mg tablets
  • Effective date: February 20, 2019; Updated: June 25, 2021 and July 8, 2022

Initiation Criteria:

For the treatment of cystic fibrosis (CF) in patients who meet all of the following criteria:

  1. Patient is 2 years of age or older; AND 

  1. Has a confirmed diagnosis of cystic fibrosis and homozygous for F508del mutation in the cystic fibrosis transmembrane conductance regulator gene; AND 

  1. The patient has demonstrated adherence to their prescribed cystic fibrosis therapeutic regimen; AND 

  1. Prescribed by a clinical specialist affiliated with a Canadian cystic fibrosis centre; AND 

  1. Patient must meet one of the following criteria:
    i) Patient is 2 years of age or older and has experienced 1 or more pulmonary exacerbation(s) per year requiring therapy with IV antibiotics OR 3 or more pulmonary exacerbations per year requiring therapy with oral antibiotics.
    OR
    ii)     Patient is 6 to 11 years of age and has an absolute decline in FEV1 percent predicted equal to or greater than 5% within a 12-month period, sustained over at least 4 months, in spite of optimized medical therapies (For example drop from 90% to 85% predicted)
    OR
    iii)     Patient is 12 years of age or older and has a baseline FEV1 equal to or less than 70% predicted, who have an absolute decline in FEV1 of ≥5%, within a 12-month period, sustained over at least 4 months, in spite of optimized medical therapies, (For example, FEV1 decline from 60% predicted to 55% predicted in the last 6 months)
    OR
    iv)     Patient is 12 years of age and older and has a baseline FEV1 of greater than 70% predicted who have an absolute decline in FEV1 of ≥10% predicted within a 12-month period, sustained over at least 4 months, in spite of optimized medical therapies (For example, FEV1 decline from 80% predicted to 70% predicted in the last 6 months) 

Exclusion criteria:
(Patients meeting the following will not be funded)

  • Patient is currently receiving invasive mechanical ventilation via endotracheal tube or tracheostomy tube; OR 

  • Patient is the previous recipient of a double lung transplant; OR 

  • Patient receiving concomitant therapy with another cystic fibrosis transmembrane conductance regulator (CFTR) modulator.

Notes:

The following assessments should be made prior to initiating treatment:

  • Weight, height, and BMI; 

  • Number of CF related hospitalizations in the previous 6 months; 

  • Number of days treated with oral and IV antibiotics for pulmonary exacerbations in the previous 6 months (OR number of pulmonary exacerbations requiring oral and IV antibiotics in previous 6 months)

Additionally, for patients 6 years of age and older, all the following MUST be provided: 

  • Baseline measurement of FEV1 in litres and % predicted taken within 3 months of planned initiation of treatment or prior to commencing treatment; AND

  • Change in FEV1 demonstrating decline in FEV1 % predicted prior to starting therapy (as defined in initiation criteria); AND

  • Cystic Fibrosis Questionnaire Child (CFQ-C) and Cystic Fibrosis Questionnaire- Parent (CFQ-P) for those 6 to 13 years old OR Cystic Fibrosis Questionnaire Respiratory (CFQ-R) Domain score for those 14 years and older.

Approval duration of initials: 7 months

Initial Renewal Criteria: 

Renewal of funding will be considered in individuals meeting the following:

  1. The patient continues to demonstrate adherence to their prescribed cystic fibrosis therapeutic regimen; AND 

  1. The patient has demonstrated at least ONE of the following after six months of treatment:
    i) A decrease in the total number of days for which the patient received treatment with oral and/or IV antibiotics for pulmonary exacerbations compared with the six-month period prior to initiating treatment OR a decrease in the total number of pulmonary exacerbations requiring oral and IV antibiotics compared with the six-month period prior to initiating treatment;
    OR
    ii)     Decreased number of CF related hospitalizations at 6 months compared with the six-month period prior to initiating Orkambi treatment;
    OR
    iii)     No decline in BMI at six months compared with the baseline BMI assessment (those 2 to 5 years old may also use BMI percentile);
    OR
    iv) No decline in FEV1 at six months compared with the baseline FEV1 assessment (as applicable to initiation criteria)

  1. If the patient is 6 years of age or older, they must demonstrate improved or sustained quality of life through an age-appropriate test (i.e., CFQ-C and CFQ-P scores for those 6 to 13 years old and CFQ-R Respiratory Domain score if the patient is 14 years or older)

Subsequent renewal criteria:

For patients who have met the initiation criteria and initial renewal criteria.

Ongoing renewal of funding will be provided for those who are continuing to benefit from therapy with Orkambi.

Approval Duration of renewals: 1 year

Approved doses: 

  • Patients 2 to 5 years old weighing less than 14 kg: 1 packet of granules (containing lumacaftor 100 mg and ivacaftor 125* mg) every 12 hours*.
  • 2 to 5 years old weighing more than 14 kg: 1 packet of granules (containing lumacaftor 150 mg and ivacaftor 188* mg) every 12 hours*.
  • 6-11 years old: 200 mg/250mg lumacaftor/ivacaftor every 12 hours.
  • 12 years and older: 400 mg/250mg lumacaftor/ivacaftor every 12 hours. 

*Sachets may be considered case-by-case to accommodate dosing requirements

EAP Drug Request Form:

Standard Form for EAP Drug Requests

Product Monograph

View Monograph