Trade name Kalydeco

Oral potentiator approved for patients with the G551D mutation of cystic fibrosis.

For the administration in cystic fibrosis patients having the G551D mutation.

G551D accounts for only 4-5% cases of cystic fibrosis.

Cystic fibrosis is caused by defects in the protein cystic fibrosis transmembrane conductance regulator, which regulates fluid flow within cells and affects the components of sweat, digestive fluids, and mucus.

G551D refers to a mutation in which the amino acid glycine (G) in position 551 is replaced with aspartic acid (D) and is characterized by a dysfunctional CFTR protein on the cell surface.

A CFTR potentiator that improves the transport of chloride through the ion channel.

Kaylydeco (ivacaftor) available for the treatment of cystic fibrosis, tripling the number of rare gene mutations that the drug is indicated to treat from 10 to 33.

Kalydeco is taken twice a day in either tablet or oral granule form with fat-containing food, and can be prescribed to patients 2 years and older with 1 of the mutations of cystic fibrosis that is responsive to treatment.

If a patient’s genotype is unknown, a mutation test can be used to determine the mutation of disease.

Adverse effects include headache, upper respiratory tract infection, including the common cold, sore throat, nasal or sinus congestion, abdominal pain, diarrhea, rash, nausea, and dizziness.

Kayldeco is also associated with increasing the risk for elevated transaminases and pediatric cataracts.

Not recommended to be taken with a strong CYP3A inducer as it has been shown to diminish the effectiveness of Kayldeco.

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