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.
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
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.