Trikafta: A Life-Changing Treatment for Many Cystic Fibrosis Patients

What is cystic fibrosis?

Cystic fibrosis (CF) is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR gene is responsible for telling our body how to make the CFTR protein, which has the important function of regulating the movement of salt and water in many different areas of our bodies.

There are over 1,700 mutations in the CFTR gene that can cause the CFTR protein to not function correctly. When one of these mutations occurs, not enough water is transported into the mucus that is present in different organs in our bodies, causing the mucus becomes thicker than it should be since it is not being thinned out by water. Two of the main organs that are most often affected are the lungs and pancreas. In the lungs, the thickened mucus can block airways and trap bacteria, leading to breathing difficulties and infections. In the pancreas, the mucus can block the pancreas's release of digestive enzymes that the body needs to properly absorb food, resulting in malnutrition.

A few decades ago, most people with CF were not expected to live beyond their teenage years. However, due to the effectiveness of new treatments such as Trikafta, the median life expectancy of people with CF born between 2020 and 2024 is predicted to be 65 years!

What is Trikafta?

Trikafta is an oral medication that was approved in 2019. It is made up of three different drugs: elexacaftor, tezacaftor, and ivacaftor. These drugs work in different ways to help restore the function of the broken CFTR proteins in patients with certain CFTR mutations.

Who can take Trikafta?

Trikafta is approved for patients with CF who are 2 years old or older and who have at least one copy of one of 272 of the mutations that can cause CF. Trikafta cannot be used for all CF patients because some CFTR mutations cause the CFTR protein to malfunction in ways that the drugs in Trikafta do not fix. However, it does work for the most commonly found CFTR mutation, so a majority of patients qualify for this medication.

How is Trikafta taken?

Trikafta comes in the form of oral tablets that are taken twice daily. There are two tablets taken together in the morning and one tablet taken in the evening. The doses should be approximately 12 hours apart and taken with a fat-containing food to help the body absorb the medication better.

For children between the ages of 2 and 5 years old, there is also the option of taking Trikafta in the form of oral granules if the child cannot swallow tablets yet. The granules come in a packet and are mixed with a soft food or liquid before administration. There is one packet that is taken in the morning and one packet that is taken in the evening. Just like with the oral tablets, the doses should be approximately 12 hours apart and taken with a fat-containing food.

How well does Trikafta work?

When Trikafta was studied in comparison with placebo, after patients had taken it for 24 weeks it was found to increase lung function by 14.3%, decrease pulmonary exacerbations by 63%, improve Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain scores (a score that ranges from 0 to 100 and measures respiratory symptoms such as wheezing, mucus production, cough, and difficulty breathing) by 20.2 points, and increase body mass index (BMI) by 1 kg/m2.

Although those results on their own are impressive, it was also important to compare Trikafta to other current treatments for CF. When Trikafta was studied in comparison to Symdeko, another medication that is used to treat CF, after patients had taken it for 4 weeks it was found to increase lung function by 10% and improve CFQ-R Respiratory Domain scores by 17.4 points in comparison with Symdeko.

Is Trikafta safe?

Trikafta is well-tolerated by most patients. However, two of the main potential safety issues with Trikafta are serious liver damage in people of any age and cataracts in children and adolescents. Because of this, when a patient starts Trikafta, their healthcare provider will do blood tests to monitor their liver before starting the medication and while on the medication. These blood tests will be more frequent during the first year and a half of taking Trikafta, but then after that they only need to be done yearly. If the patient is under 18 years old, their healthcare provider will recommend that they get eye examinations at least yearly to check for cataracts.

References:

Cystic Fibrosis Foundation. (n.d.). About cystic fibrosis. https://www.cff.org/intro-cf/about-cystic-fibrosis

Cystic Fibrosis Foundation. (n.d.). CFTR modulator therapies. https://www.cff.org/managing-cf/cftr-modulator-therapies

Cystic Fibrosis Foundation. (n.d.). Dispelling misconceptions about cystic fibrosis. https://www.cff.org/intro-cf/dispelling-misconceptions-about-cystic-fibrosis

Cystic Fibrosis Foundation. (n.d.). Types of CFTR mutations. https://www.cff.org/research-clinical-trials/types-cftr-mutations

Cystic Fibrosis Foundation. (n.d.). Understanding changes in life expectancy. https://www.cff.org/managing-cf/understanding-changes-life-expectancy

FDA. (n.d). Orphan drug designations and approvals: Trikafta. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=647618

National Jewish Health. (2019). Cystic fibrosis: life expectancy. https://www.nationaljewish.org/conditions/cf/life-expectancy

Trikafta. (n.d.). How to take Trikafta tablets. https://www.trikafta.com/how-to-take-trikafta-tablets

Trikafta. (n.d.). How to take Trikafta granules. https://www.trikafta.com/how-to-take-trikafta-granules

Trikafta. (n.d.). How Trikafta works. https://www.trikafta.com/how-trikafta-works

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