Lumacaftor/ivacaftor: Difference between revisions

Latest revision as of 16:24, 16 February 2025

Lumacaftor/Ivacaftor[edit]

Lumacaftor/ivacaftor is a combination medication used in the treatment of cystic fibrosis (CF) in patients who have specific genetic mutations. This medication is marketed under the brand name Orkambi and is produced by Vertex Pharmaceuticals.

Mechanism of Action[edit]

Lumacaftor/ivacaftor works by targeting the defective CFTR (cystic fibrosis transmembrane conductance regulator) protein, which is responsible for the symptoms of cystic fibrosis.

Lumacaftor acts as a chaperone that helps the CFTR protein fold correctly and reach the cell surface. It is classified as a CFTR corrector.
Ivacaftor is a CFTR potentiator that enhances the function of the CFTR protein once it is at the cell surface, improving chloride ion transport.

Together, these drugs improve the function of the CFTR protein in patients with the F508del mutation, the most common mutation causing cystic fibrosis.

Indications[edit]

Lumacaftor/ivacaftor is indicated for the treatment of cystic fibrosis in patients aged 2 years and older who are homozygous for the F508del mutation in the CFTR gene. This mutation is present in approximately 50% of cystic fibrosis patients.

Administration[edit]

The medication is administered orally in the form of tablets. The dosage is typically based on the patient's age and weight, and it is recommended to be taken with fat-containing food to enhance absorption.

Side Effects[edit]

Common side effects of lumacaftor/ivacaftor include:

Serious side effects can include liver enzyme elevations and respiratory events, particularly in patients with advanced lung disease.

Related Pages[edit]

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-'''Lumacaftor/ivacaftor''', sold under the brand name '''Orkambi''', is a combination medication used for the treatment of [[cystic fibrosis]] (CF) in individuals who have two copies of the F508del mutation in their [[CFTR gene]]. It is a significant advancement in the management of cystic fibrosis, targeting the underlying cause of the disease rather than just its symptoms.
+== Lumacaftor/Ivacaftor ==
-==Composition==
+[[File:Ivacaftor_and_lumacaftor.svg|thumb|right|Chemical structures of Ivacaftor and Lumacaftor]]
-Lumacaftor/ivacaftor is a combination of two drugs: [[lumacaftor]] and [[ivacaftor]], each targeting different aspects of the CFTR protein's function. Lumacaftor acts as a corrector, facilitating the proper folding and trafficking of the CFTR protein to the cell surface, while ivacaftor functions as a potentiator, enhancing the channel's function once it reaches the cell surface.
-==Indications==
+'''Lumacaftor/ivacaftor''' is a combination medication used in the treatment of [[cystic fibrosis]] (CF) in patients who have specific genetic mutations. This medication is marketed under the brand name Orkambi and is produced by [[Vertex Pharmaceuticals]].
-This medication is indicated for the treatment of cystic fibrosis in patients aged 2 years and older who have two copies of the F508del mutation in the CFTR gene. The presence of this specific genetic mutation must be confirmed through genetic testing before starting treatment.
-==Mechanism of Action==
+== Mechanism of Action ==
-Lumacaftor works by correcting the misfolded CFTR protein, allowing it to reach the cell surface, where it can function properly. Ivacaftor then enhances the activity of the protein, facilitating improved chloride transport across the cell membrane. This action helps to thin the thick mucus that is characteristic of cystic fibrosis, improving lung function and reducing the risk of infections.
-==Administration==
+Lumacaftor/ivacaftor works by targeting the defective [[CFTR]] (cystic fibrosis transmembrane conductance regulator) protein, which is responsible for the symptoms of cystic fibrosis.
-Lumacaftor/ivacaftor is administered orally, with the dosage and frequency depending on the patient's age and weight. It is important to take this medication with fatty foods to enhance its absorption.
-==Side Effects==
+* '''Lumacaftor''' acts as a [[chaperone]] that helps the CFTR protein fold correctly and reach the cell surface. It is classified as a CFTR corrector.
-Common side effects include shortness of breath, nausea, diarrhea, rash, and fatigue. Liver enzyme levels may increase, so regular monitoring of liver function is recommended during treatment. Patients should also be monitored for respiratory symptoms, as the medication can cause chest tightness and dyspnea in some individuals.
+* '''Ivacaftor''' is a CFTR potentiator that enhances the function of the CFTR protein once it is at the cell surface, improving chloride ion transport.
-==Approval and Regulation==
+Together, these drugs improve the function of the CFTR protein in patients with the F508del mutation, the most common mutation causing cystic fibrosis.
-The combination was approved by the [[Food and Drug Administration]] (FDA) in the United States in 2015 for use in patients with cystic fibrosis who have the F508del mutation. Its approval marked a significant milestone in the treatment of this condition, offering hope for improved quality of life and life expectancy for patients.
-==Impact==
+== Indications ==
-The introduction of lumacaftor/ivacaftor has been a breakthrough in cystic fibrosis care, offering patients the first treatment option that addresses the underlying cause of the disease for those with the F508del mutation. Clinical trials have shown improvements in lung function, reduced pulmonary exacerbations, and improved weight gain among patients treated with this combination therapy.
-==Conclusion==
+Lumacaftor/ivacaftor is indicated for the treatment of cystic fibrosis in patients aged 2 years and older who are homozygous for the F508del mutation in the CFTR gene. This mutation is present in approximately 50% of cystic fibrosis patients.
-Lumacaftor/ivacaftor represents a significant advancement in the treatment of cystic fibrosis, providing a novel approach to managing the disease by correcting the function of the defective CFTR protein. Its development and approval underscores the importance of targeted genetic therapies in treating hereditary diseases.
-[[Category:Pharmacology]]
+== Administration ==
-[[Category:Cystic Fibrosis]]
-{{pharmacology-stub}}
+The medication is administered orally in the form of tablets. The dosage is typically based on the patient's age and weight, and it is recommended to be taken with fat-containing food to enhance absorption.
+== Side Effects ==
+Common side effects of lumacaftor/ivacaftor include:
+* [[Respiratory tract infection]]
+* [[Nasal congestion]]
+* [[Diarrhea]]
+* [[Nausea]]
+* [[Rash]]
+Serious side effects can include liver enzyme elevations and respiratory events, particularly in patients with advanced lung disease.
+== Related Pages ==
+* [[Cystic fibrosis]]
+* [[CFTR]]
+* [[Vertex Pharmaceuticals]]
+* [[Ivacaftor]]
+{{Cystic fibrosis}}
+[[Category:Cystic fibrosis treatments]]
+[[Category:Combination drugs]]