The active ingredient in KALYDECO tablets is ivacaftor.
KALYDECO is classified as a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the G551D mutation.
Limitations of Use
KALYDECO is not effective in patients with CF who are homozygous for the F508del mutation in the CFTR gene and has not been studied in other populations of patients with CF.
Media Articles Related to Kalydeco (Ivacaftor)
Benefits of cystic fibrosis drug ivacaftor reported in pre-school children for the first time
Source: Cystic Fibrosis News From Medical News Today [2016.01.21]
The oral drug ivacaftor appears to be safe and could be beneficial to young children between the ages of 2 and 5 with a specific type of cystic fibrosis, according to new research published in The...
Published Studies Related to Kalydeco (Ivacaftor)
A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of
patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2
randomised controlled trial. 
CFTR potentiator that enhances chloride transport of CFTR on the cell surface... INTERPRETATION: We provide evidence that combination lumacaftor and ivacaftor
Clinical Trials Related to Kalydeco (Ivacaftor)
Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients [Recruiting]
This is a study of the short-term effects of ivacaftor on sweat chloride concentration and
lung function in cystic fibrosis (CF) patients who fall outside current FDA approval. This
new, first of its kind drug is approved for use only in CF patients with the G551D mutation
in whom it safely confers considerable benefits. However, it is highly likely that CF
patients with many other mutations can benefit similarly from this drug, some of whom can be
identified by phenotype or genotype.
We will enroll up to 30 CF subjects with clinical presentations in which there is one or
more signs of residual CF channel function. The signs of residual function include: normal
digestion, concentration of chloride in sweat between 55 and 85, or milder than expected CF
disease in a CF patient with severe gene mutations. The primary outcome measure will be the
difference in sweat chloride concentration measured in subjects on placebo and on ivacaftor.
Secondary outcome measured will be lung function.
Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation [Completed]
The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and
pharmacodynamics (PD), of ivacaftor in children with cystic fibrosis (CF) who are 2 through
5 years of age and have a CF Transmembrane Conductance Regulator (CFTR) gating mutation in
at least 1 allele.
Part A is designed to evaluate the safety and PK of multiple-dose administration of
ivacaftor in participants 2 through 5 years of age and to confirm the doses for Part B. Part
B is designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in participants 2
through 5 years of age.
Safety Study of Ivacaftor in Subjects With Cystic Fibrosis [Completed]
The purpose of this study was to evaluate the safety and tolerability of ivacaftor in
patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation
in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a
potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human
CFTR protein. Potentiators are pharmacological agents that increase the chloride ion
transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A
Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT) [Completed]
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects
with cystic fibrosis (CF) who have the R117H-CFTR mutation.
(Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil? [Recruiting]
Clinical studies of lumacaftor + ivacaftor (combo therapy) produced better FEV1 (forced
expiratory volume in 1 second) improvements than ivacaftor alone, without further
improvement in sweat chloride results.
To help understand why sweat chloride was unresponsive, the investigators will use a newly
developed sweat secretion test that provides accurate, in vivo readout of CFTR (cystic
fibrosis transmembrane conductance regulator) function in the sweat gland secretory coil.
The investigators devised a protocol to determine if short courses of ivacaftor (3. 5 days)
will produce significant increases in WT (Wild-Type, i. e. normal) CFTR open probability by
measuring CFTR-dependent sweating (C-sweat) in subjects with WT CFTR.
Reports of Suspected Kalydeco (Ivacaftor) Side Effects
Drug Ineffective (10),
Infective Pulmonary Exacerbation of Cystic Fibrosis (10),
Productive Cough (4),
Abdominal Pain Upper (4),
Chest Pain (4),
Dyspnoea (4), more >>
Page last updated: 2016-01-21