Treatment Eligibility

ARE YOUR PATIENTS ELIGIBLE FOR SYMDEKO® (tezacaftor/ivacaftor and ivacaftor)?

Enter your patient’s mutations below to see if they are eligible for SYMDEKO.
You may determine eligibility for up to 5 patients at once.

Enter your patient’s mutations below to see if they are eligible for SYMDEKO.

SYMDEKO is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.

SYMDEKO is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.

Mutation 1
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Mutation 2 (optional)
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    Most patients have 1 CFTR mutation on each allele. However, in rare instances, a single allele can have more than 1 mutation. This is called a compound, or complex, mutation.

    To enter 2 or more mutations into 1 entry field, separate them with a semicolon, space, comma, or forward slash. For example, if your patient's genotype is R74W/V201M/D1270N and W1282R, use the Mutation 1 field to enter: R74W/V201M/D1270N and the Mutation 2 field to enter: W1282R.

    Alternatively, please see the responsive mutations below:

    Bold=Mutations approved in December 2020

    CFTR Mutations That Produce CFTR Protein and Are Responsive to SYMDEKO 1-3

    546insCTA

    F575Y

    L346P

    R553Q

    711+3A→G*

    F1016S

    L967S

    R668C

    2789+5G→A*

    F1052V

    L997F

    R751L

    3272-26A→G*

    F1074L

    L1324P

    R792G

    3849+10kbC→T*

    F1099L

    L1335P

    R933G

    A120T

    G126D

    L1480P

    R1066H

    A234D

    G178E

    M152V

    R1070Q

    A349V

    G178R

    M265R

    R1070W*

    A455E*

    G194R

    M952I

    R1162L

    A554E

    G194V

    M952T

    R1283M

    A1006E

    G314E

    P5L

    R1283S

    A1067T

    G551D

    P67L*

    S549N

    D110E

    G551S

    P205S

    S549R

    D110H*

    G576A

    Q98R

    S589N

    D192G

    G576A;
    R668C

    Q237E

    S737F

    D443Y

    G622D

    Q237H

    S912L

    D443Y;G576A;R668C

    G970D

    Q359R

    S945L*

    D579G*

    G1069R

    Q1291R

    S977F*

    D614G

    G1244E

    R31L

    S1159F

    D836Y

    G1249R

    R74Q

    S1159P

    D924N

    G1349D

    R74W

    S1251N

    D979V

    H939R

    R74W;
    D1270N

    S1255P

    D1152H*

    H1054D

    R74W;V201M

    T338I

    D1270N

    H1375P

    R74W;V201M;D1270N

    T1036N

    E56K

    I148T

    R75Q

    T1053I

    E60K

    I175V

    R117C*

    V201M

    E92K

    I336K

    R117G

    V232D

    E116K

    I601F

    R117H

    V562I

    E193K

    I618T

    R117L

    V754M

    E403D

    I807M

    R117P

    V1153E

    E588V

    I980K

    R170H

    V1240G

    E822K

    I1027T

    R258G

    V1293G

    E831X

    I1139V

    R334L

    W1282R

    F191V

    I1269N

    R334Q

    Y109N

    F311del

    I1366N

    R347H*

    Y161S

    F311L

    K1060T

    R347L

    Y1014C

    F508C

    L15P

    R347P

    Y1032C

    F508C;
    S1251N

    L206W*

    R352Q*

        

    F508del

    L320V

    R352W

         

    *Clinical data for these mutations appear in Clinical Studies, and can be found here, and here, or within section 14.1 and 14.2 of the Prescribing Information.
    †A patient must have 2 copies of the F508del mutation or at least 1 copy of a responsive mutation listed above in this table to be indicated for SYMDEKO.
    ‡Complex/compound mutations in which a single allele of the CFTR gene has multiple mutations; these exist independent of the presence of mutations on the other allele.

    INDICATIONS AND USAGE

    SYMDEKO is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.

    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.


    INDICATIONS AND USAGE

    SYMDEKO is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.

    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.


    IMPORTANT SAFETY INFORMATION

    WARNINGS AND PRECAUTIONS

    Transaminase (ALT or AST) Elevations

    • Elevated transaminases have been observed in patients with CF receiving SYMDEKO, as well as with ivacaftor monotherapy. Assessments of transaminases (ALT and AST) are recommended prior to initiating SYMDEKO, every 3 months during the first year of treatment, and annually thereafter. For patients with a history of transaminase elevations, more frequent monitoring should be considered
    • Dosing should be interrupted in patients with significant elevations of transaminases (e.g., ALT or AST >5x upper limit of normal [ULN], or ALT or AST >3x ULN with bilirubin >2x ULN) and laboratory tests should be closely followed until abnormalities resolve. Following resolution of transaminase elevations, consider the benefits and risks of resuming treatment

    Hypersensitivity Reactions, Including Anaphylaxis

    • Hypersensitivity reactions, including cases of anaphylaxis, have been reported in the postmarketing setting. If signs or symptoms of serious hypersensitivity reactions develop during treatment, discontinue SYMDEKO and institute appropriate therapy. Consider the benefits and risks for the individual patient to determine whether to resume treatment with SYMDEKO

    Concomitant Use With CYP3A Inducers

    • Exposure to ivacaftor is significantly decreased and exposure to tezacaftor may be reduced by concomitant use of CYP3A inducers, which may reduce the therapeutic effectiveness of SYMDEKO. Co-administration of SYMDEKO with strong CYP3A inducers, such as rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, and St. John’s wort, is not recommended

    Cataracts

    • Cases of non-congenital lens opacities have been reported in pediatric patients treated with SYMDEKO, as well as with ivacaftor monotherapy. Baseline and follow-up ophthalmological examinations are recommended in pediatric patients initiating treatment with SYMDEKO

    ADVERSE REACTIONS

    Serious Adverse Reactions

    • Serious adverse reactions, whether considered drug-related or not by the investigators, that occurred more frequently in patients treated with SYMDEKO compared to placebo included distal intestinal obstruction syndrome, 3 (0.6%) patients treated with SYMDEKO vs. 0 placebo patients

    Most Common Adverse Reactions

    • The most common adverse reactions in Trials 1 and 3 occurring in ≥3% of patients treated with SYMDEKO (N=334) and at a higher rate than for placebo (N=343) were headache, nausea, sinus congestion, and dizziness
    • The safety profile in patients age 6 to less than 12 years from an open-label Phase 3 trial (N=70) was similar to that observed in Trials 1 and 3

    USE IN SPECIFIC POPULATIONS

    Pediatric Use

    • The safety and effectiveness of SYMDEKO in patients with CF younger than 6 years of age have not been studied

    Click here to access full Prescribing Information.

    References:
    1. SYMDEKO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; August 2023. 2. The Clinical and Functional Translation of CFTR (CFTR2); Available at http://cftr2.org. List of CFTR2 mutations. https://www.cftr2.org/mutations_history/CFTR2_11March2019.xlsx. Accessed September 1, 2022. 3. National Center for Biotechnology Information. ClinVar. Available at https://www.ncbi.nlm.nih.gov/clinvar/. Accessed September 1, 2022.

     

     

    INDICATIONS AND USAGE

    SYMDEKO is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.

    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.


    IMPORTANT SAFETY INFORMATION

    WARNINGS AND PRECAUTIONS

    Transaminase (ALT or AST) Elevations

    • Elevated transaminases have been observed in patients with CF treated with SYMDEKO, as well as with ivacaftor monotherapy. Assessments of transaminases (ALT and AST) are recommended prior to initiating SYMDEKO, every 3 months during the first year of treatment, and annually thereafter. For patients with a history of transaminase elevations, more frequent monitoring should be considered
    • Dosing should be interrupted in patients with significant elevations of transaminases (e.g., ALT or AST >5x upper limit of normal [ULN], or ALT or AST >3x ULN with bilirubin >2x ULN) and laboratory tests should be closely followed until abnormalities resolve. Following resolution of transaminase elevations, consider the benefits and risks of resuming treatment

    Hypersensitivity Reactions, Including Anaphylaxis

    • Hypersensitivity reactions, including cases of anaphylaxis, have been reported in the postmarketing setting. If signs or symptoms of serious hypersensitivity reactions develop during treatment, discontinue SYMDEKO and institute appropriate therapy. Consider the benefits and risks for the individual patient to determine whether to resume treatment with SYMDEKO

    Concomitant Use With CYP3A Inducers

    • Exposure to ivacaftor is significantly decreased and exposure to tezacaftor may be reduced by concomitant use of CYP3A inducers, which may reduce the therapeutic effectiveness of SYMDEKO. Co-administration of SYMDEKO with strong CYP3A inducers, such as rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, and St. John’s wort, is not recommended

    Cataracts

    • Cases of non-congenital lens opacities have been reported in pediatric patients treated with SYMDEKO, as well as with ivacaftor monotherapy. Baseline and follow-up ophthalmological examinations are recommended in pediatric patients initiating treatment with SYMDEKO

    ADVERSE REACTIONS

    Serious Adverse Reactions

    • Serious adverse reactions, whether considered drug-related or not by the investigators, that occurred more frequently in patients treated with SYMDEKO compared to placebo included distal intestinal obstruction syndrome, 3 (0.6%) patients treated with SYMDEKO vs. 0 placebo patients

    Most Common Adverse Reactions

    • The most common adverse reactions in Trials 1 and 3 occurring in ≥3% of patients treated with SYMDEKO (N=334) and at a higher rate than for placebo (N=343) were headache, nausea, sinus congestion, and dizziness
    • The safety profile in patients age 6 to less than 12 years from an open-label Phase 3 trial (N=70) was similar to that observed in Trials 1 and 3

    USE IN SPECIFIC POPULATIONS

    Pediatric Use

    • The safety and effectiveness of SYMDEKO in patients with CF younger than 6 years of age have not been studied

    Click here to access full Prescribing Information.

    References:
    1. SYMDEKO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; August 2023. 2. The Clinical and Functional Translation of CFTR (CFTR2); Available at http://cftr2.org. List of CFTR2 mutations. https://www.cftr2.org/mutations_history/CFTR2_11March2019.xlsx. Accessed September 1, 2022. 3. National Center for Biotechnology Information. ClinVar. Available at https://www.ncbi.nlm.nih.gov/clinvar/. Accessed September 1, 2022.