Treatment Eligibility

ARE YOUR PATIENTS ELIGIBLE FOR KALYDECO®(ivacaftor)?

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

Enter your patient's mutations below to see if they are eligible for KALYDECO.

KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 4 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.

Mutation 1
Mutation 2 (optional)
1.

Mutation(s) not recognized. Please check to see if they were entered correctly.

Mutation(s) not recognized. Please check to see if they were entered correctly.

    Most patients have 2 CFTR mutations, 1 for 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 R74WN201M/D1270N and W1282R, use the Mutation 1 field to enter: R74WN201M/D1270N and the Mutation 2 field to enter: W1282R.

    Please see the responsive mutations below:

    CFTR Mutations Responsive to KALYDECO Based on Clinical and/or In Vitro Data¹⁻³
    A1067T G1349D* P67L* S1159P
    A120T G178E Q1291R S1251N*
    A234D G178R* Q237E S1255P*
    A349V G194R Q237H S549N*
    A455E* G314E Q359R S549R*
    D110E G551D* R1070Q S589N*
    D110H G551S* R1070W* S737F
    D1152H* G576A R1162L S945L*
    D1270N G970D R117C* S977F*
    D192G H1375P R117G T1053I
    D579G* H939R R117H* T338I
    D924N I1027T R117L V1293G
    E193K I1139V R117P V232D
    E56K I148T R1283M V562I
    E822K I175V R170H V754M
    E831X* I807M R347H* W1282R
    F1052V K1060T R347L Y1014C
    F1074L L1480P R352Q* Y1032C
    F311del L206W* R553Q 2789+
    5G→A*
    F311L L320V R668C 3272-
    261→G*
    F508C L967S R74W 3849+10kbC→T*
    F508C;S1251N L997F R75Q 711+3A→G*
    G1069R M152V R792G  
    G1244E* M952I R933G  
    G1249R M952T S1159F  
    CFTR Mutations Responsive to KALYDECO Based on Clinical and/or In Vitro Data¹⁻³
    A1067T G1349D* P67L* S1159P
    A120T G178E Q1291R S1251N*
    A234D G178R* Q237E S1255P*
    A349V G194R Q237H S549N*
    A455E* G314E Q359R S549R*
    D110E G551D* R1070Q S589N*
    D110H G551S* R1070W* S737F
    D1152H* G576A R1162L S945L*
    D1270N G970D R117C* S977F*
    D192G H1375P R117G T1053I
    D579G* H939R R117H* T338I
    D924N I1027T R117L V1293G
    E193K I1139V R117P V232D
    E56K I148T R1283M V562I
    E822K I175V R170H V754M
    E831X* I807M R347H* W1282R
    F1052V K1060T R347L Y1014C
    F1074L L1480P R352Q* Y1032C
    F311del L206W* R553Q 2789+
    5G→A*
    F311L L320V R668C 3272-
    261→G*
    F508C L967S R74W 3849+
    10kbC→T*
    F508C;
    S1251N
    L997F R75Q 711+3A→G*
    G1069R M152V R792G  
    G1244E* M952I R933G  
    G1249R M952T S1159F  

    *Clinical data exist for these mutations.1
    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.1

    Back to top

    Important Safety Information

    Transaminase (ALT or AST) Elevations

    • Elevated transaminases have been reported in patients with CF receiving KALYDECO. Transaminase elevations were more common in patients with a history of transaminase elevations or in patients who had abnormal transaminases at baseline. ALT and AST should be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. For patients with a history of transaminase elevations, consider more frequent monitoring of liver function tests
    • Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal (ULN). Following resolution of transaminase elevations, consider the benefits and risks of resuming KALYDECO

    Indications and Usage

    KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 4 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation 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.

    Indications and Usage

    KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 4 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation 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.

    Important Safety Information

    Transaminase (ALT or AST) Elevations

    • Elevated transaminases have been reported in patients with CF receiving KALYDECO. Transaminase elevations were more common in patients with a history of transaminase elevations or in patients who had abnormal transaminases at baseline. ALT and AST should be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. For patients with a history of transaminase elevations, consider more frequent monitoring of liver function tests
    • Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal (ULN). Following resolution of transaminase elevations, consider the benefits and risks of resuming KALYDECO

    Concomitant Use With CYP3A Inducers

    • Use of KALYDECO with strong CYP3A inducers, such as rifampin, substantially decreases the exposure of ivacaftor, which may reduce the therapeutic effectiveness of KALYDECO. Co-administration of KALYDECO 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/cataracts have been reported in pediatric patients treated with KALYDECO. Baseline and follow-up ophthalmological examinations are recommended in pediatric patients initiating KALYDECO treatment

    Pediatric Use

    • The safety and effectiveness of KALYDECO in patients with CF younger than 4 months of age have not been studied. The use of KALYDECO in children under the age of 4 months is not recommended

    Serious Adverse Reactions

    • Serious adverse reactions, whether considered drug-related or not by the investigators, which occurred more frequently in patients treated with KALYDECO included abdominal pain, increased hepatic enzymes, and hypoglycemia

    Most Common Adverse Reactions

    • The most common adverse reactions in patients with a G551D mutation in the CFTR gene (Trials 1 and 2) with an incidence of ≥8% and at a higher incidence for patients treated with KALYDECO (N=109) than for placebo (N=104) were headache, oropharyngeal pain, upper respiratory tract infection, nasal congestion, abdominal pain, nasopharyngitis, diarrhea, rash, nausea, and dizziness
    • The safety profiles for patients with additional approved mutations enrolled in Trials 4, 5, and 7; and for patients aged 2 to less than 6 years enrolled in Trial 6; and for patients aged 4 months to less than 24 months enrolled in Trial 8; were similar to that observed in Trials 1 and 2

    Click here to access full Prescribing Information.

    References:
    1.  KALYDECO [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; December 2020. 2. The Clinical and Functional Translation of CFTR (CFTR2); http://cftr2.org. Accessed October 1, 2021. 3. Cystic Fibrosis Genetic Analysis Consortium, The Hospital for Sick Children. Cystic Fibrosis Mutation Database (CFTR1). http://www. genet.sickkids.on.ca/app. Accessed October 1, 2021.