Trikafta - NC Standard

Commercial Policy

Version Date: January 2025

Restricted Product(s)

Trikafta® (Elexacaftor/tezacaftor/ivacaftor; Ivacaftor)

FDA Approved Use

For the treatment of cystic fibrosis (CF) in patients 2 years and older who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on in vitro data
If the patients genotype is unknown, an FDA-cleared CF mutation test should be able to confirm the presence of at least one F508del mutation or a mutation that is responsive based on in vitro data.

Criteria for Approval of Restricted Product(s)

The patient is 2 years of age or older; AND
The patient has a diagnosis of cystic fibrosis confirmed by ONE of the following:
1. An FDA-cleared CF mutation test that detected at least one F508del mutation in the CFTR gene; OR
2. The presence of at least one of the CFTR genes listed in the table below; AND
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
Trikafta will not be used concurrently with other CFTR modulators (e.g., Alyftrek™, Orkambi®, Kalydeco®, Symdeko®); AND
The patient is being managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., pulmonologist); AND
Indications outside of FDA labeling will be subject to medical necessity review in accordance with specific strong endorsement or support by nationally recognized compendia, when such recommendation is based on strong/high levels of evidence, and/or uniform consensus of clinical appropriateness has been reached. [Medical records and references / evidence must be provided]; AND
For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.

Duration of Approval: 365 days (1 year)

Mutations responsive to TRIKAFTA based on clinical data*

27894+5G⁠→A
3272-26A⁠→G
3849+10kbC⁠→T
A455E†
D1152H†
F508del†
G85E†
L1077P†
L206W†
L997F†
M1101K†
P5L†
R1066H†
R117C†
R347H†
R347P†
S945L†
T3381†
V232D†

Mutations responsive to TRIKAFTA based on in vitro data•

11027T
1105N
111391V
1125T
11269N
11366N
1148N
1148T
1175V
1331N
1336K
14554E
1502T
1506L
1507 1515del9
1556V
1601F
1618T
1807M
1980K
2183A⁠→G
3141de19
546insCTA
A1006E
A1067P
A1067T
A107G
A120T
A234D
A26P
A309D
A349V
A46D
A554E
C491R
D110E
D110H
D1270N
D1445N
D192G
D443Y
D443Y;G576A;R668C
D565G
D579G
D614G
D836Y
D924N
D979V
D993Y
E116K
E116Q
E193K
E292K
E403D
E474K
E56K
E588V
E60K
E822K
E92K
F1016S
F1052V
F1074L
F1099L
F1107L
F191V
F2001
F311del
F311L
F508C
F508C;S1251N
F575Y
F5871
G1047R
G1061R
G1069R
G1123R
G1244E
G1247R
G1249R
G126D
G1349D
G178E
G178R
G194R
G194V
G27E
G27R
G314E
G424S
G463V
G480C
G480S
G551A
G551D
G551S
G576A
G576A;R668C
G622D
G628R
G970D
G970S
H1375P
H139R
H199Y
H620P
H620Q
H939R
H939R;H949L
Hl054D
Hl085P
Hl085R
K1060T
K162E
K464E
L1011S
L1324P
L1335P
L137P
L1480P
L15P
L165S
L320V
L333F
L333H
L346P
L441P
L453S
L619S
L967S
M1137V
M152V
M265R
M9521
M952T
Ml50K
N1088D
N13031
N1303K
N186K
N187K
N418S
P140S
P205S
P499A
P574H
P67L
P750L
Q1291R
Q1313K
Q237E
Q237H
Q359R
Q372H
Q493R
Q552P
Q98R
R1048G
R1070Q
R1070W
R1162L
R117C;G576A;R668C
R117G
R117H
R117L
R117P
R1283M
R1283S
R170H
R258G
R297Q
R31C
R31L
R334L
R334Q
R347L
R352Q
R352W
R516S
R553Q
R555G
R668C
R709Q
R74Q
R74W
R74W;D1270N
R74W;V201M
R74W;V201M;D1270N
R751L
R75L
R75Q
R792G
R933G
S1045Y
S108F
S1118F
S1159F
S1159P
S1235R
S1251N
S1255P
S13F
S341P
S364P
S492F
S5491
S549N
S549R
S589N
S737F
S912L
S977F
T1036N
T1053I
T1246I
T1299I
T3511
Tl086I
V1153E
V1240G
V1293G
V201M
V392G
V456A
V456F
V562I
V603F
V754M
W1098C
W1282R
W361R
Y1014C
Y1032C
Y109N
Y161D
Y161S
Y301C
Y563N

Mutations responsive to TRIKAFTA based on extrapolation from Trial 5

1341G⁠→A
1898+3A⁠→G
2752-26A⁠→G
2789+2insA
296+28A⁠→G
3041-15T⁠→G
3141del9
3600G⁠→A
3849+40A⁠→G
3849+4A⁠→G
3850-3T⁠→G
4005+2T⁠→C
546insCTA
5T;TG12
5T;TG13
621+3A→G
711+3A⁠→G
A1006E
A1067T
A120T
A234D
A349V
A455E
A46D
A554E
D110E
D110H
D1152H
D1270N
D192G
D443Y
D443Y;G576A;R668C†
D579G
D614G
D836Y
D924N
D979V
E116K
E193K
E403D
E474K
E56K
E588V
E60K
E822K
E831X
E92K
F1016S
F1052V
F1074L
F1099L
F191V
F311L
F508C
F508C;S1251N†
F508del*
F575Y
G1061R
G1069R
G1244E
G1249R
G126D
G1349D
G178E
G178R
G194R
G194V
G27R
G314E
G463V
G480C
G551D
G551S
G576A
G576A;R668C†
G622D
G628R
G85E
G970D
H1054D
H1085P
H1085R
H1375P
H139R
H199Y
H939R
I1027T
I1139V
I1269N
I1366N
I148T
I175V
I336K
I502T
I601F
I618T
I807M
I980K
K1060T
L1077P
L1324P
L1335P
L1480P
L15P
L165S
L206W
L320V
L346P
L453S
L967S
L997F
M1101K
M152V
M265R
M952I
M952T
P205S
P574H
P5L
P67L
Q1291R
Q237E
Q237H
Q359R
Q98R
R1066H
R1070Q
R1070W
R1162L
R117C
R117G
R117H
R117L
R117P
R1283M
R1283S
R170H
R258G
R31L
R334L
R334Q
R347H
R347L
R347P
R352Q
R352W
R553Q
R668C
R74Q
R74W
R74W;D1270N†
R74W;V201M;D1270N†
R74W;V201M†
R751L
R75Q
R792G
R933G
S1159F
S1159P
S1251N
S1255P
S13F
S341P
S364P
S492F
S549N
S549R
S589N
S737F
S912L
S945L
S977F
T1036N
T1053I
T338I
V1153E
V1240G
V1293G
V201M
V232D
V456A
V456F
V562I
V754M
W1098C
W1282R
W361R
Y1014C
Y1032C
Y109N
Y161D
Y161S
Y563N

*F508del is a responsive CFTR mutation based on both clinical and in vitro [see Clinical Studies (14)]

^†Complex/compound mutations where a single allele of the CFTR gene has multiple mutations; these exist independent of the presence of mutations on the other allele.

Quantity Limitations

Quantity limitations apply to brand and associated generic products.

Medication	Quantity per Day (unless specified)
Trikafta 50-25-37.5-75 mg tablet	3 tablets
Trikafta 100-50-75-150 mg tablet	3 tablets
Trikafta 59.5 mg packet	56 packets / 28 days
Trikafta 75 mg packet	56 packets / 28 days

Quantity Limit Exception Criteria

The quantity (dose) requested is for documented titration purposes at the initiation of therapy (authorization for a 90 day titration period); AND
The prescribed dose cannot be achieved using a lesser quantity of a higher strength; AND
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
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: 365 days (1 year)

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 change: Updated with new FDA approved mutation chart. Added Alyftrek to medications not to be used concurrently with requested medication.

January 2025: Criteria change: Added requirement that Trikafta will not be used concurrently with other CFTR. Added specialist requirement. Added P&T statement.

May 2023: Criteria change: Expanded age indication to 2 years of age and added new to market Trikafta packets.

February 2023: Criteria update: Criteria review, formatting, simplified language (clarified) documentation required for diagnosis.

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

June 2021: Criteria change: Expanded indication to 6 years of age or older. Changed duration of approval to 365 days.

December 2020: Criteria change: Indication update to include mutations in the CFTR gene that is responsive based on in vitro data.

June 2020: Criteria change: Removed Orkambi and Symdeko as step therapy in members homozygous for the F508del mutation.

November 2019: Original utilization management criteria issued.

Disclosures:

BLUE CROSS®, BLUE SHIELD® and the Cross and Shield Symbols are registered marks of the Blue Cross and Blue Shield Association, an association of independent Blue Cross and Blue Shield Plans. Blue Cross NC is an independent licensee of the Blue Cross and Blue Shield Association. All other marks are the property of their respective owners.

Blue Cross and Blue Shield of North Carolina does not discriminate on the basis of race, color, national origin, sex, age or disability in its health programs and activities. Learn more about our non-discrimination policy and no-cost services available to you.

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