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Kalydeco
Cystic Fibrosis (CF), at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor, homozygous for F508del mutation in the CFTR gene
Treatment
3 FDA approvals
0 Active Studies for Kalydeco
What is Kalydeco
IvacaftorThe Generic name of this drug
Treatment SummaryIvacaftor (Kalydeco or VX-770) is a drug used to treat Cystic Fibrosis (CF). It was approved by the FDA in 2012 and by Health Canada in late 2012. It is used as a monotherapy and in combination with other drugs to help manage the symptoms of CF. Cystic Fibrosis is an inherited condition caused by mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, which affects the transport of chloride and sodium ions across cell membranes. Ivacaftor is the first medication designed to treat the underlying causes of CF
Kalydecois the brand name
Kalydeco Overview & Background
Brand Name
Generic Name
First FDA Approval
How many FDA approvals?
Kalydeco
Ivacaftor
2012
13
Approved as Treatment by the FDA
Ivacaftor, commonly known as Kalydeco, is approved by the FDA for 3 uses including Cystic Fibrosis (CF) and homozygous for F508del mutation in the CFTR gene .Cystic Fibrosis (CF)
Helps manage Cystic Fibrosis (CF)
homozygous for F508del mutation in the CFTR gene
Used to treat homozygous for F508del mutation in the CFTR gene in combination with Tezacaftor
at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor
Used to treat at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor in combination with Tezacaftor
Effectiveness
How Kalydeco Affects PatientsIvacaftor helps to improve symptoms and underlying causes of cystic fibrosis. It works by increasing the chance that the CFTR protein can open, which is different from other cystic fibrosis medications that help prevent the protein from misfolding. Taking ivacaftor has been linked to better lung function, reduced risk of lung flare-ups, lower sweat chloride levels, increased weight gain, and improved quality of life. When combined with tezacaftor, even more improvements in lung function have been seen. It is important to note that this drug may cause an increase in liver transaminases (ALT, AST). Regular
How Kalydeco works in the bodyIvacaftor is a drug that helps people with cystic fibrosis. Cystic fibrosis is caused by mutations in a gene called CFTR, which is responsible for transporting ions across cell membranes. The most common mutation is called delta-F508 and it does not respond to ivacaftor, because it prevents the CFTR protein from being produced. However, the second most common mutation, G551D, does respond to ivacaftor. This mutation changes how the CFTR protein works, and ivacaftor binds to it and helps it to open and close properly. Ivacaftor helps improve symptoms of
When to interrupt dosage
The suggested dosage of Kalydeco is based on the determined condition, for instance, at least one variation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor, Cystic Fibrosis (CF) and ivacaftor-responsive CFTR mutation. The amount of dosage can be found in the table below, contingent upon the method of administration (e.g. Granule or Kit; Tablet, film coated - Oral).Condition
Dosage
Administration
Cystic Fibrosis (CF)
, 150.0 mg, 75.0 mg, 50.0 mg, 125.0 mg, 300.0 mg, 188.0 mg, 25.0 mg, 225.0 mg, 112.5 mg
, Oral, Tablet, Tablet - Oral, Granule, Granule - Oral, Tablet, film coated, Tablet, film coated - Oral
at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor
, 150.0 mg, 75.0 mg, 50.0 mg, 125.0 mg, 300.0 mg, 188.0 mg, 25.0 mg, 225.0 mg, 112.5 mg
, Oral, Tablet, Tablet - Oral, Granule, Granule - Oral, Tablet, film coated, Tablet, film coated - Oral
homozygous for F508del mutation in the CFTR gene
, 150.0 mg, 75.0 mg, 50.0 mg, 125.0 mg, 300.0 mg, 188.0 mg, 25.0 mg, 225.0 mg, 112.5 mg
, Oral, Tablet, Tablet - Oral, Granule, Granule - Oral, Tablet, film coated, Tablet, film coated - Oral
Warnings
Kalydeco ContraindicationsCondition
Risk Level
Notes
Severe Hypersensitivity Reactions
Do Not Combine
Ivacaftor may interact with Pulse Frequency
Common Kalydeco Drug Interactions
Drug Name
Risk Level
Description
Abemaciclib
Major
The serum concentration of Abemaciclib can be increased when it is combined with Ivacaftor.
Acalabrutinib
Major
The metabolism of Acalabrutinib can be decreased when combined with Ivacaftor.
Alectinib
Major
The metabolism of Alectinib can be decreased when combined with Ivacaftor.
Alpelisib
Major
The metabolism of Alpelisib can be decreased when combined with Ivacaftor.
Aminophylline
Major
The metabolism of Aminophylline can be decreased when combined with Ivacaftor.
Kalydeco Toxicity & Overdose RiskInformation on how much ivacaftor is dangerous is not known, but when combined with tezacaftor, the highest dose can cause diarrhea and dizziness. In the case of an overdose, supportive measures should be taken, as there is no antidote available.
Kalydeco Novel Uses: Which Conditions Have a Clinical Trial Featuring Kalydeco?
An active clinical trial is currently in progress to examine the potential of Kalydeco for homozygous F508del mutation in the CFTR gene and other ivacaftor-responsive CFTR mutations, as well as at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor.Condition
Clinical Trials
Trial Phases
Cystic Fibrosis (CF)
0 Actively Recruiting
at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor
0 Actively Recruiting
homozygous for F508del mutation in the CFTR gene
0 Actively Recruiting
Patient Q&A Section about kalydeco
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.How effective is Kalydeco?
"Patients who took Kalydeco had a 0.7% decrease in FEV in the US cohort, while those who didn't take the treatment had an 8.3% decrease. In the UK cohort, Kalydeco improved pulmonary function by 4.9%, while those who didn't take the treatment saw a 4.3% decrease."
Answered by AI
Who can take Kalydeco?
"i. KALYDECO is a medication that can be taken by people with cystic fibrosis who are at least 4 months old, and who have a mutation in their CF gene that makes them responsive to the medication.
ii. If you have cystic fibrosis and are at least 4 months old, you can take KALYDECO if at least one of your CF gene mutations makes you responsive to the medication."
Answered by AI
What does kalydeco treat?
"KALYDECO is a prescription medicine used to treat cystic fibrosis in patients who are responsive to the medication and are at least 4 months old."
Answered by AI
How quickly does Kalydeco work?
"The drug Kalydeco (ivacaftor) is most concentrated in your body four hours after taking it with a high-fat meal."
Answered by AI