Orkambi* (lumacaftor/ivacaftor) vs Kalydeco* (ivacaftor)

Orkambi (lumacaftor/ivacaftor) and Kalydeco (ivacaftor) are both medications used to treat cystic fibrosis, but they are indicated for different mutations of the CFTR gene. Orkambi is a combination therapy designed for patients who have two copies of the F508del mutation in the CFTR gene, targeting the protein folding defect characteristic of this mutation, while Kalydeco is suitable for patients with specific mutations that result in gating abnormalities of the CFTR protein, which can be addressed by ivacaftor alone. The choice between Orkambi and Kalydeco will depend on the patient's specific genetic mutation, and a healthcare provider will recommend the appropriate medication based on genetic testing.

Difference between Orkambi* and Kalydeco*

Metric	Orkambi* (lumacaftor/ivacaftor)	Kalydeco* (ivacaftor)
Generic name	Lumacaftor/Ivacaftor	Ivacaftor
Indications	Cystic fibrosis in patients who are homozygous for the F508del mutation in the CFTR gene	Cystic fibrosis in patients with specific mutations in the CFTR gene
Mechanism of action	Lumacaftor improves the conformational stability of F508del-CFTR, allowing it to reach the cell surface, where ivacaftor increases the probability of the chloride ion channel opening	Increases the activity of the CFTR protein once it reaches the cell surface
Brand names	Orkambi	Kalydeco
Administrative route	Oral	Oral
Side effects	Shortness of breath, upper respiratory tract infection, nausea, diarrhea, rash, fatigue, chest discomfort, increased blood creatine phosphokinase, etc.	Headache, upper respiratory tract infection, abdominal pain, diarrhea, rash, nausea, dizziness, etc.
Contraindications	Use with certain strong CYP3A inducers or inhibitors	Use with certain strong CYP3A inducers or inhibitors
Drug class	CFTR modulator	CFTR potentiator
Manufacturer	Vertex Pharmaceuticals	Vertex Pharmaceuticals

Efficacy

Orkambi (lumacaftor/ivacaftor) Efficacy in Cystic Fibrosis

Orkambi is a combination medication consisting of lumacaftor and ivacaftor, which is specifically designed for the treatment of cystic fibrosis (CF) in patients who have two copies of the F508del mutation in the CFTR gene. The efficacy of Orkambi in treating CF has been demonstrated in several clinical trials. These studies have shown that Orkambi can lead to improvements in lung function, as measured by the percent predicted forced expiratory volume in one second (ppFEV1). Patients taking Orkambi have also experienced fewer pulmonary exacerbations, which are episodes that can cause additional lung damage and are a significant concern for individuals with CF.

In addition to improvements in lung function and reductions in exacerbations, Orkambi has also been associated with gains in body weight, which is an important indicator of overall health in CF patients. This benefit is likely due to the role of the CFTR protein in the digestive system and the improvement in function that Orkambi provides. However, the response to Orkambi can vary among individuals, and not all patients may experience the same level of benefit.

Kalydeco (ivacaftor) Efficacy in Cystic Fibrosis

Kalydeco, which contains the active ingredient ivacaftor, is another medication used in the treatment of cystic fibrosis. Unlike Orkambi, Kalydeco is indicated for CF patients who have specific mutations in the CFTR gene that lead to gating abnormalities. The efficacy of Kalydeco has been established through clinical trials where it has been shown to significantly improve lung function. Patients treated with Kalydeco have demonstrated increases in ppFEV1, which is a critical measure of the ability to breathe effectively.

Furthermore, Kalydeco has been linked to a reduction in the rate of pulmonary exacerbations and improvements in other outcomes such as weight gain, quality of life, and a decrease in the concentration of sweat chloride—a diagnostic marker of CF. The medication works by enhancing the function of the defective CFTR protein, thereby improving the transport of chloride ions across cell membranes, which is a key problem in CF. As with Orkambi, the benefits of Kalydeco can vary from person to person, and it is most effective in individuals with specific CFTR mutations for which it is approved.

Regulatory Agency Approvals

Orkambi*

European Medical Agency (EMA), European Union
Food and Drug Administration (FDA), USA
Therapeutic Goods Administration (TGA), Australia

Kalydeco*

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

Access Orkambi* or Kalydeco* today

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