Kalydeco* (ivacaftor) vs Trikafta* (elexacaftor/tezacaftor/ivacaftor; ivacaftor)

Kalydeco (ivacaftor) is a CFTR potentiator designed to enhance the function of the CFTR protein once it reaches the cell surface, primarily effective in cystic fibrosis (CF) patients with specific genetic mutations. Trikafta, a combination of elexacaftor, tezacaftor, and ivacaftor, works by correcting the processing and function of the faulty CFTR protein, leading to an improvement in CFTR function at the cell surface; it is indicated for a broader range of CF mutations compared to Kalydeco. When deciding between Kalydeco and Trikafta, it is essential to consider the specific CFTR mutations a patient has, as Trikafta can treat individuals with at least one F508del mutation, which is the most common, while Kalydeco is for a select group of other mutations.

Difference between Kalydeco* and Trikafta*

Metric	Kalydeco* (ivacaftor)	Trikafta* (elexacaftor/tezacaftor/ivacaftor; ivacaftor)
Generic name	Ivacaftor	Elexacaftor/tezacaftor/ivacaftor
Indications	Cystic fibrosis in patients with specific mutations in the CFTR gene	Cystic fibrosis in patients aged 12 years and older with at least one F508del mutation in the CFTR gene
Mechanism of action	CFTR potentiator	CFTR modulator (combination of potentiator and correctors)
Brand names	Kalydeco	Trikafta
Administrative route	Oral	Oral
Side effects	Headache, upper respiratory tract infection, abdominal pain, diarrhea, rash, nausea, dizziness	Headache, upper respiratory tract infection, abdominal pain, diarrhea, rash, nausea, dizziness, increased liver enzymes
Contraindications	Use with certain drugs that are strong CYP3A inducers or inhibitors	Use with certain drugs that are strong CYP3A inducers or inhibitors
Drug class	CFTR potentiator	CFTR modulator
Manufacturer	Vertex Pharmaceuticals	Vertex Pharmaceuticals

Efficacy

Kalydeco (Ivacaftor) Efficacy in Cystic Fibrosis

Kalydeco (ivacaftor) is a medication approved for the treatment of cystic fibrosis (CF) in patients aged 6 months and older who have one of several specific mutations in the CFTR gene. The efficacy of Kalydeco in cystic fibrosis has been demonstrated in clinical trials where it has been shown to improve lung function, as measured by the percent predicted forced expiratory volume in one second (ppFEV1). Patients taking Kalydeco have experienced significant improvements in lung function compared to those on placebo.

Additionally, Kalydeco has been associated with other health benefits for CF patients, including a reduction in pulmonary exacerbations, which are periods when respiratory symptoms worsen significantly. It has also been linked to weight gain and improvements in body mass index (BMI), which are important indicators of overall health in CF patients, who often struggle with malnutrition and difficulty maintaining a healthy weight.

Trikafta (Elexacaftor/Tezacaftor/Ivacaftor) Efficacy in Cystic Fibrosis

Trikafta, a combination of elexacaftor, tezacaftor, and ivacaftor, represents a significant advancement in the treatment of cystic fibrosis. It is approved for use in patients aged 6 years and older who have at least one F508del mutation in the CFTR gene, which is the most common mutation causing CF. Clinical trials have shown that Trikafta substantially improves lung function, with patients experiencing an average increase in ppFEV1 that is clinically meaningful. This improvement in lung function is a strong indication of the drug's efficacy in treating the respiratory aspects of CF.

In addition to enhancing lung function, Trikafta has shown efficacy in improving other outcomes for CF patients. The medication has been associated with a decrease in the rate of pulmonary exacerbations and improvements in respiratory symptoms. Furthermore, patients on Trikafta have reported better quality of life scores, indicating that the drug not only addresses the physical aspects of the disease but also has a positive impact on patients' overall well-being.

Regulatory Agency Approvals

Kalydeco*

European Medical Agency (EMA), European Union
Food and Drug Administration (FDA), USA

Trikafta*

Food and Drug Administration (FDA), USA

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