Cystic Fibrosis Medications

Cystic fibrosis (CF) is a major inherited disorder involving abnormalities of fluid and electrolyte transport in a number of different organs due to abnormal function of cystic fibrosis transmembrane conductance regulator (CFTR) protein.

More than 1,000 mutations in the CFTR gene have been identified in people with cystic fibrosis.
Most of these mutations change single amino acids in the CFTR protein or delete a small amount of DNA from the CFTR gene.
It mainly affects the lungs, and to a lesser extent the pancreas, liver, and intestine, causing the the build-up of thick, sticky mucus in these areas.
At the respiratory level, an alteration in bronchial secretions is generated, with increased viscosity and alteration of mucociliary clearance.

Vicious circle leading to the triad characteristic of the disease: Bronchial obstruction-infection-inflammation, leading to irreversible lung damage, with bronchiectasis and respiratory failure.

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Ivacaftor is one of the first drugs developed to treat the underlying cause of CF rather than the symptoms.
Pulmeran is an enhancer of the protein that regulates the transmembrane conductance of cystic fibrosis.
It is indicated in patients 6 years of age and older, weighing 25 kg or more, who have a mutation in the CFTR gene sensitive to the effect of Ivacaftor.
Pulmeran works by modifying the progression of the disease, providing significant and long-term benefits.

Pulmeran (Ivacaftor 150 mg):
For adults, adolescents, and children (6 years of age or older, weighing 25 kg or more), the recommended dose is 150 mg (1 Pulmeran Film-Coated Tablet) every 12 hours (total a daily dose of 300 mg) taken orally with fat-containing foods.

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Pulmeran L is a fixed-dose tablet (Ivacaftor 125mg/Lumacaftor 200mg), with a dual effect: a Corrector (lumacaftor) and Potentiator (ivacaftor) of the cystic fibrosis transmembrane conductance regulator (CFTR).
Lumacaftor improves the processing of F508del CFTR and its transport to the cell surface, while ivacaftor increases the channel’s open probability and transport of chloride.

Pulmeran L is indicated in patients (aged ≥12 years) homozygous for the most common CFTR mutation F508del of the gene that encodes the transmembrane conductance regulator protein of Cystic Fibrosis.
Pulmeran L significantly improves lung function, increases body mass index, and reduces exacerbations.
Benefits from Pulmeran L are maintained long-term (120 weeks)
It was observed in the Progress trial that the combination of Lumacaftor and Ivacaftor maintains efficacy and safety over time.

Pulmeran L (Ivacaftor 125 mg/Lumacaftor 200 mg):

Film-Coated Tablets is presented in packages containing 120 Film-Coated Tablets.
The mean recommended dosage for children aged 12 years and older and adults is 2 Film-Coated Tablets of Pulmeran L every 12 hours (total daily dose of 500 mg Ivacaftor and 800 mg Lumacaftor).

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