Orkambi – NC Standard

Restricted Product(s):

• Orkambi® (lumacaftor/ivacaftor)

FDA Approved Use:

• For the treatment of cystic fibrosis (CF) in patients 1 year of age and older who are homozygous for the F508del mutation in the CFTR gene. If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene.
• Limitations of Use: The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation.

Criteria for Approval of Restricted Product(s):

1. The patient is 1 year of age or older; AND 
2. The patient has been diagnosed with cystic fibrosis; AND 
3. The patient is homozygous for the F508del mutation; AND 
4. An FDA-cleared CF mutation test has been used to detect the presence of a mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene; AND 
5. Please attach patient specific lab results and/or Cystic Fibrosis Foundation Patient Registry Report with confirmation of a mutation in the CFTR gene (medical record documentation required); AND 
6. Requests for granule packets are only authorized for patients under the age of 6 who weigh at least 7 kg and are medically unable to take tablet formulation by oral administration; AND 
7. Orkambi will not be used concurrently with other CFTR modulators (e.g., Alyftrek™ , Kalydeco®, Symdeko®, Trikafta®); AND 
8. The patient is being managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., pulmonologist); AND 
9. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.

Duration of Approval: 1 year (365 days)

Quantity Limitations:

Quantity limitations apply to brand and associated generic products.

Quantity Limit Exception Criteria:

1. The quantity (dose) requested is for documented titration purposes at the initiation of therapy (authorization for a 90 day titration period); AND 
2. The prescribed dose cannot be achieved using a lesser quantity of a higher strength; AND 
3. The quantity (dose) requested does not exceed the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert; OR 
4. If the quantity (dose) requested exceeds the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert, then the prescriber must submit documentation in support of therapy with a higher dose for the intended diagnosis (submitted documentation may include medical records OR fax form which reflects medical record documentation that shows the length of time the requested dose has been used, and what other medications and doses have been tried and failed).

Duration of Approval: 1 year (365 days)

References:

All information referenced is from FDA package insert unless otherwise noted below.

Policy Implementation/Update Information:

Criteria and treatment protocols are reviewed annually by the Blue Cross NC P&T Committee, regardless of change. This policy is reviewed in Q4 annually.

January 2025v2: Criteria update: Added Alyftrek to medications not to be used concurrently with requested medication. Added limitation of use section.

January 2025: Criteria change: Added requirement that Orkambi will not be used concurrently with other CFTR. Added specialist requirement.

September 2022v2: Criteria change: Added new to market 75-94 mg oral granules packet to quantity limitations.

September 2022: Criteria change: Added expanded age indication for the treatment of patients 1 year of age or older.

April 2022: Criteria change: Duration of approval added for 1 year. Quantity limits added.

August 2021: Criteria update: Annual criteria review. Formatting changes only.

September 2018: updated to address ages 2-5 and new to market packet formulation

August 2018: updated to address the expanded indication for those 2 years of age and older (previously 6 years of age and older).

October 2016: updated to address the expanded indication for those 6 years of age and older (previously 12 years of age and older).

August 2015: Updated the following requirement to add the CFF patient registry report: **Please attach patient-specific lab results and/or Cystic Fibrosis Foundation Patient Registry Report with confirmation of the presence of the F508del mutation on both alleles of the CFTR gene**

July 2015: Original utilization management criteria issued.