Kalydeco – NC Standard

Restricted Product(s)

• Kalydeco (ivacaftor)

FDA Approved Use

• For the treatment of cystic fibrosis (CF) in patients age 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data 
• If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

Criteria for Approval of Restricted Product(s)

1. The patient is 1 month of age or older; AND 
2. The patient has been diagnosed with cystic fibrosis; AND 
3. The patient has had an FDA-cleared CF mutation test that detected the presence of at least one of the CFTR genes listed below AND
   1.  The provider has submitted 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 
4. The patient is NOT homozygous for a F508del mutation; AND 
5. Kalydeco will not be used concurrently with other CFTR modulators (e.g., Alyftrek™, Orkambi®, Symdeko®, Trikafta ®); AND 
6. The patient is being managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., pulmonologist); AND 
7. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.

Duration of Approval: 1 year (365 days)

CFTR genes

• 711+3A→G *
• 2789+5G→A *
• 3272-26A→G *
• 3849+10kbC→T*
• A120T
• A234D
• A349V
• A455E *
• A1067T
• D110E
• D110H
• D192G
• D579G *
• D924N
• D1152H *
• D1270N
• E56K
• E193K
• E822K
• E831X *
• F311del
• F311L
• F508C
• F508C;S1251N†
• F1052V
• F1074L
• G178E
• G178R *
• G194R
• G314E
• G551D *
• G551S *
• G576A
• G907D
• G1069R
• G1244E *
• G1249R
• G1349D *
• H939R
• H1375P
• I148T
• I175V
• I807M
• I1027T
• I1139V
• K1060T
• L206W *
• L320V
• L967S
• L997F
• L1480P
• M152V
• M9521
• M952T
• P67L *
• Q237E
• Q237H
• Q359R
• Q1291R
• R74W
• R75Q
• R117C *
• R117G
• R117H *
• R117L
• R117P
• R170H
• R347H *
• R347L
• R352Q *
• R553Q
• R668C
• R792G
• R933G
• R1070Q
• R1070W *
• R1162L
• R1283M
• S549N *
• S549R *
• S589N
• S737F
• S945L *
• S977F *
• S1159F
• S1159P
• S1251N *
• S1255P *
• T338I
• T1053I
• V232D
• V562I
• V754M
• V1293G
• W1282R
• Y1014C
• Y1032C

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.

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

October 2023: Criteria update: added new to market Kalydeco 5.8 mg packet.

May 2023: Criteria update: Updated for expanded age indication in patients 1 month and older. Added new to market Kalydeco 13.4 mg packet.

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

December 2020: Criteria change: Updated CFTR genes eligible for approval.

October 2020: Criteria update: Updated to address ages 4 months and up.

June 2019: Criteria update: updated to address ages 6 months and up

September 2018: updated to address ages 12 months- 2 years.

May 2018: Reformatted criteria; updated with specific genes responsive to ivacaftor (under policy implementation)

Aug 2015: Added medical record documentation for generic testing

Mar 2015: Original utilization management criteria issued