Description
Lucaftor (lumacaftor/ivacaftor) is a prescription medication that belongs to the drug class of potentiator and corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It is used to treat cystic fibrosis (CF) in patients 2 years of age and older who have at least one F508del mutation in the CFTR gene.
Mechanism of Action
CF is a genetic disease that causes the CFTR protein to not work properly. The CFTR protein is responsible for transporting salt and water across cell membranes. When the CFTR protein does not work properly, it can lead to a buildup of mucus in the lungs and other organs.
Lucaftor works by correcting the folding of the CFTR protein and increasing its stability. Ivacaftor works by opening the CFTR protein once it has been corrected. Together, lumacaftor and ivacaftor help to improve the function of the CFTR protein and reduce the symptoms of CF.
Dosage and Administration
Lucaftor is taken twice daily by mouth, with food. The recommended dosage for adults is 200 mg of lumacaftor and 150 mg of ivacaftor per dose. The dosage may be adjusted depending on the patient’s individual needs and response to the medication.
Effectiveness
Lucaftor has been shown to be effective in improving lung function and reducing the symptoms of CF in clinical trials. For example, in one study, lumacaftor/ivacaftor improved lung function by an average of 4.6% in patients with CF who had at least one F508del mutation.
Side Effects
The most common side effects of lumacaftor/ivacaftor include:
- Cough
- Headache
- Diarrhea
- Nausea
- Upper respiratory tract infection
- Rash
- Fatigue
- Abdominal pain
- Vomiting
- Dizziness
- Shortness of breath
Lucaftor/ivacaftor can also cause serious side effects, such as:
- Liver problems
- Kidney problems
- Heart problems
- Pancreatitis
- Allergic reactions
Contraindications
Lucaftor/ivacaftor is contraindicated in patients with:
- Hypersensitivity to lumacaftor or ivacaftor
- Severe hepatic impairment
- Severe renal impairment
Lucaftor/ivacaftor may also interact with other medications you are taking. Tell your doctor about all the medications you are taking, including prescription drugs, over-the-counter drugs, vitamins, and herbal supplements.
Conclusion
Lucaftor is an effective treatment for CF in patients who have at least one F508del mutation in the CFTR gene. However, it is important to be aware of the potential side effects of lumacaftor/ivacaftor before starting treatment. Talk to your doctor about the risks and benefits of lumacaftor/ivacaftor to determine if it is right for you.
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