Orkambi* (lumacaftor/ivacaftor) vs Symkevi* (tezacaftor/ivacaftor)

Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) are both combination medications used to treat cystic fibrosis (CF) in patients with specific genetic mutations. Orkambi is indicated for patients who have two copies of the F508del mutation, while Symkevi is used for those with either two copies of the F508del mutation or one F508del mutation and one of several other mutations that respond to tezacaftor/ivacaftor. The choice between Orkambi and Symkevi should be made in consultation with a healthcare provider, considering factors such as the patient's specific genetic mutations, age, overall health, and potential side effects of each medication.

Difference between Orkambi* and Symkevi*

Metric	Orkambi* (lumacaftor/ivacaftor)	Symkevi* (tezacaftor/ivacaftor)
Generic name	Lumacaftor/Ivacaftor	Tezacaftor/Ivacaftor
Indications	Cystic fibrosis in patients age 2 years and older with two copies of the F508del mutation in the CFTR gene	Cystic fibrosis in patients age 6 years and older with two copies of the F508del mutation or one mutation in the CFTR gene that is responsive to tezacaftor/ivacaftor therapy
Mechanism of action	Lumacaftor improves the conformational stability of F508del-CFTR, allowing it to reach the cell surface, while ivacaftor enhances the channel activity of the CFTR protein at the cell surface	Tezacaftor facilitates the cellular processing and trafficking of normal and F508del-CFTR proteins to increase the amount of functional protein at the cell surface, ivacaftor then increases the probability that the channel is open, improving ion flow
Brand names	Orkambi	Symdeko (Symkevi in the EU)
Administrative route	Oral	Oral
Side effects	Shortness of breath, upper respiratory tract infection, nausea, diarrhea, rash, fatigue, abnormal chest sound, flu or flu-like symptoms, increased blood creatine phosphokinase	Headache, nasopharyngitis, sinus congestion, nausea, dizziness, rash, increased blood creatine phosphokinase, common cold, flu or flu-like symptoms, increased transaminases
Contraindications	Hypersensitivity to lumacaftor, ivacaftor or any of the excipients, severe hepatic impairment	Hypersensitivity to tezacaftor, ivacaftor or any of the excipients, moderate or severe hepatic impairment
Drug class	CFTR modulator	CFTR modulator
Manufacturer	Vertex Pharmaceuticals	Vertex Pharmaceuticals

Efficacy

Orkambi (lumacaftor/ivacaftor) in Cystic Fibrosis

Orkambi, a combination of lumacaftor and ivacaftor, is a medication specifically designed for the treatment of cystic fibrosis (CF) in patients who have two copies of the F508del mutation in the CFTR gene. This medication works by improving the function of the defective protein produced by this gene mutation, thereby enhancing the transport of chloride ions across cell membranes. Clinical trials have demonstrated that Orkambi can lead to modest improvements in lung function, as measured by the percent predicted forced expiratory volume in one second (ppFEV1). Patients on Orkambi have also experienced fewer pulmonary exacerbations, which are periods of worsening respiratory symptoms that often require antibiotics and hospitalization.

While the efficacy of Orkambi has been established, it is important to note that the degree of improvement in lung function varies among patients. Some individuals may experience more significant benefits than others. Additionally, Orkambi has been shown to have a positive effect on other aspects of CF, including body mass index (BMI), which is an important indicator of overall health in CF patients. However, the medication does not cure CF and is not effective for all CFTR mutations.

Symkevi (tezacaftor/ivacaftor) in Cystic Fibrosis

Symkevi, a combination of tezacaftor and ivacaftor, is another treatment option for cystic fibrosis, targeting the same F508del mutation as Orkambi, but also effective in patients with one F508del mutation and a second mutation that is responsive to tezacaftor/ivacaftor. Similar to Orkambi, Symkevi works to correct the function of the defective CFTR protein, leading to improved chloride ion flow across cell membranes. Clinical studies have shown that Symkevi can improve lung function, as indicated by increases in ppFEV1, and reduce the rate of pulmonary exacerbations in patients with the specified mutations.

The efficacy of Symkevi is generally comparable to that of Orkambi, with some studies suggesting that Symkevi may have a better side effect profile and potentially greater improvements in lung function for certain patients. As with Orkambi, the effects of Symkevi on lung function and other symptoms of CF are variable, and the treatment does not represent a cure for the disease. Symkevi's role in the management of CF continues to be evaluated in ongoing research and clinical practice, with particular attention to long-term outcomes and quality of life improvements for those affected by this condition.

Regulatory Agency Approvals

Orkambi*

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

Symkevi*

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

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