Understanding What Class of Drug Is Trikafta: The CFTR Modulator

October 10, 2025 •

2 min read

More than 90% of cystic fibrosis (CF) patients in the United States are eligible for treatment with Trikafta. So, what class of drug is Trikafta? It belongs to a revolutionary class of medications known as cystic fibrosis transmembrane conductance regulator (CFTR) modulators, specifically a triple-combination therapy, that directly targets the underlying genetic cause of the disease.

Quick Summary

Trikafta is a CFTR modulator that corrects and potentiates the function of the defective CFTR protein in cystic fibrosis patients, improving lung function and reducing symptoms.

Key Points

CFTR Modulator Class: Trikafta belongs to the class of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, which directly address the underlying cause of CF.
Triple-Combination Therapy: It is a combination of three active ingredients: elexacaftor and tezacaftor (correctors) and ivacaftor (a potentiator).
Mechanism of Action: The correctors help the CFTR protein fold correctly and reach the cell surface, while the potentiator helps the channel stay open, increasing the flow of salt and water.
High Efficacy: Clinical trials show Trikafta is highly effective, leading to significant improvements in lung function and quality of life for a broad range of CF patients.
Eligibility: It is indicated for patients with at least one F508del mutation or other responsive CFTR gene mutations.
Safety Considerations: Requires regular liver function monitoring due to the risk of serious liver injury.
Drug Interactions: Patients must be cautious of drug-drug interactions, particularly with CYP3A inducers and inhibitors, and should avoid grapefruit.

Trikafta's Drug Class: A CFTR Modulator

Trikafta is classified as a cystic fibrosis transmembrane conductance regulator (CFTR) modulator. This means it targets the genetic defect causing cystic fibrosis (CF), where a mutation in the CFTR gene results in a faulty protein that disrupts salt and water flow in organs like the lungs and pancreas, leading to thick mucus. Unlike older treatments addressing symptoms, CFTR modulators improve the protein's function at a cellular level. Trikafta is a triple-combination therapy with three drugs working together for maximum protein function.

The Triple-Combination Approach

Trikafta's three active ingredients, elexacaftor, tezacaftor, and ivacaftor, each contribute to correcting the CFTR protein's malfunction. Elexacaftor and tezacaftor are correctors that help the protein fold correctly and reach the cell surface, addressing issues like the misfolding seen in the F508del mutation. Ivacaftor acts as a potentiator at the cell surface, increasing the protein channel's opening probability, which boosts chloride ion and water flow, thinning mucus. This combination significantly improves CFTR activity and is more effective than earlier modulator therapies.

Impact and Effectiveness

Studies show Trikafta significantly benefits eligible CF patients, improving lung function (FEV1), quality of life, reducing pulmonary exacerbations, and decreasing sweat chloride levels. By targeting the disease's cause, it slows progression, making CF a more manageable condition for many.

Important Considerations and Side Effects

Patients taking Trikafta should discuss potential side effects and drug interactions with their doctor. Regular liver function monitoring is essential. Common side effects include headache, respiratory infections, stomach issues, and rash. Serious side effects like liver damage require careful monitoring. Trikafta interacts with drugs metabolized by the CYP3A enzyme system, so patients must inform their doctor of all medications and supplements. Grapefruit products should be avoided due to potential interference with metabolism and increased side effect risk.

Comparison of CFTR Modulator Therapies

Trikafta is considered the most effective CFTR modulator therapy available. For a comparison of different FDA-approved modulator regimens, including Kalydeco, Orkambi, Symdeko, and Trikafta, which details their drug components, mechanism, targeted mutations, and clinical efficacy, please refer to the Vertex Pharmaceuticals website {Link: Vertex Pharmaceuticals website https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-trikafta}.

Conclusion

Trikafta, a triple-combination CFTR modulator, represents a significant advance in cystic fibrosis treatment by addressing the root cause. This therapy combines two correctors and one potentiator to improve lung function and quality of life for a large population of CF patients. While not a cure, it substantially restores CFTR protein function. Monitoring for side effects and drug interactions is crucial for patient safety. For further information on CFTR modulators and cystic fibrosis, consult the Cystic Fibrosis Foundation.

Frequently Asked Questions

Older CF treatments focused on managing symptoms caused by thick mucus, such as antibiotics for infections or mucolytics to thin mucus. In contrast, Trikafta and other CFTR modulators are disease-modifying, targeting the root cause by fixing the defective CFTR protein at the cellular level.

Trikafta contains elexacaftor and tezacaftor, which are 'correctors' that help the CFTR protein reach the cell surface. The third component, ivacaftor, is a 'potentiator' that helps open the channel once the protein is at the cell surface.

A CFTR modulator is a drug that improves the production, intracellular processing, and/or function of the defective CFTR protein. This is the malfunctioning protein that causes cystic fibrosis.

Trikafta is approved for patients with at least one F508del mutation or another CFTR gene mutation that has been shown to be responsive to the medication based on clinical or in vitro data.

Yes, Trikafta can cause serious liver damage, including liver failure. Regular liver function tests are necessary, especially during the initial phase of treatment. Other serious side effects can include severe allergic reactions and cataracts in children.

Yes, Trikafta should be taken with fat-containing food to ensure proper absorption. Examples include meals prepared with butter or oil, eggs, cheese, or nuts.

Patients should avoid grapefruit and products containing grapefruit, as it can interact with Trikafta and increase the risk of side effects. Strong CYP3A inducers, such as rifampin and St. John's wort, are also not recommended as they can reduce Trikafta's effectiveness.