Trikafta Approval: Frequently Asked Questions

Trikafta®, a highly effective modulator therapy initially approved by the FDA on October 21, 2019, treats the underlying cause of CF. The following are commonly asked questions about Trikafta.

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Summary
  • Trikafta® is a highly effective therapy for the underlying cause of cystic fibrosis.
  • It is approved for use in people with CF ages 6 and older who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data.
  • It is not a cure for CF.

Last updated: June 9, 2021

About Trikafta®

What is Trikafta?

Trikafta is a "triple-combination therapy" consisting of three different modulators -- tezacaftor/ivacaftor (which make up Symdeko®) combined with elexacaftor. Modulators work by helping to fix defective CFTR protein. Trikafta has the potential to be significantly more effective than current modulators.

Who is eligible to take Trikafta?

The U.S. Food and Drug Administration (FDA) approved Trikafta for people with CF ages 6 years and older who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on laboratory data. (Please see the current list of eligible mutations.)

What are the potential benefits of taking Trikafta?
  • Clinical trial data of Trikafta have shown dramatic improvements in key measures of the disease, including lung function.
  • People ages 12 and older with two copies of the F508del mutation had a 10 percent increase in lung function compared to treatment with the modulator tezacaftor/ivacaftor (Symdeko®), and people with one copy of F508del had more than a 14 percent increase in lung function compared to placebo.
  • Participants also saw a significant increase in the assessment used to measure individuals' quality of life and a decrease in sweat chloride.
  • Research suggests that Trikafta may slow or prevent the progression of CF. When started at an early age, this therapy (and other highly effective modulator therapy) has potential to stave off the most common complications of CF before they start. For example, studies have shown that pancreatic function was restored in some young children taking Kalydeco® (ivacaftor).
  • However, advanced damage that has already occurred in the lungs and other organs (such as the pancreas) generally cannot be reversed.
When will Trikafta be expanded to younger age groups?

A Phase 3 trial of Trikafta in children with CF ages 2 to 5 is underway. Vertex Pharmaceuticals, the drug's manufacturer, has not announced when it will release results of the trial or when it plans to apply to the FDA for approval for this age group.

Safety Profile and Interactions

Are there known risks or side effects? Is it safe?
  • In clinical trials, Trikafta was shown to be safe and effective with potentially fewer negative side effects than some previous modulators. Trikafta comes with warnings related to elevated liver function tests, drug-drug interactions with products that are inducers or inhibitors of a certain liver enzyme, and the possible risk of cataracts in younger age groups.
  • There is no current data to suggest any widespread safety issues that are not already on the label. However, potential side effects should be taken seriously. If you or someone you know is experiencing a symptom that may be related to medication, speak with your care team immediately. Also, be sure to report your symptoms to the MedWatch reporting program to ensure the FDA can continue to monitor the safety of this important therapy.
  • In order to help ensure clinicians and people with CF know as much about this important therapy as possible, a clinical study called PROMISE is evaluating the short- and long-term effects of triple-combination therapy on the overall health of people with CF, including infections, inflammation, mucus clearance, Gl health, blood sugar, growth, and liver function. In addition, the Foundation is planning a study to better understand the potential effects of highly effective modulators, including Trikafta, on various aspects of the mental health of people with CF.
Are there drug-drug interactions?
  • Certain drugs may interact with Trikafta, including some antifungal medicines and some antibiotics.
  • You should not take Trikafta if you are on certain antibiotics (rifampin or rifabutin), specific seizure medications, and St. John's wort.
  • Talk to your doctor about all your current medications and supplements before taking Trikafta.
Will I be able to stop my other medications and treatments?
  • People with CF who take the drug will likely still need other daily treatments to manage the symptoms of the disease. This should be discussed with your care team before making any changes to your medications or treatments.
  • We recognize that it is a burden to take daily medications. The Foundation is funding a study called SIMPLIFY that will look at whether stopping inhaled hypertonic saline or dornase alfa (Pulmozyme®) for a few weeks impacts lung function in people with CF who are taking Trikafta.
  • Another study called HERO2 will be using real world data from people with CF to advance our understanding of treatment care routines while on Trikafta. This study, conducted by Indiana University, will ask people with CF to track their symptoms and treatments for 12 months using an internet-based app powered by Folia Health.
  • For more information about these and other CF studies, please check out the Clinical Trial Finder.

Access and Coverage

How do I get Trikafta?

If you think you may qualify, speak to your care team about being evaluated to see whether your mutations qualify and if you are a good candidate for the medicine.

Will my insurance cover Trikafta?
  • Your coverage will depend on your insurance provider(s), and we expect policies to vary. Vertex offers copay assistance for eligible individuals with commercial/private insurance. For more information on Vertex's GPS program, including eligibility requirements for assistance, contact Vertex at 1-877-752-5933 or visit www.VertexGPS.com.
  • Compass, a personalized service provided by the CF Foundation, can help you determine if your plan covers Trikafta and what to expect to pay in out-of-pocket costs. Call 844-COMPASS (844-266-7277) Monday through Friday, 9 a.m. until 7 p.m. ET, or email compass@cff.org.
What is the Foundation doing to help people obtain coverage for this medication?
  • The Foundation has contacted major public and private insurers, including Medicaid programs and large national plans, to ensure they understand cystic fibrosis as well as the significance of Trikafta, and its potentially critical role in improving the lives of people with CF.
  • We are closely monitoring coverage policies for Trikafta and will work strategically with the community -- clinicians, patients, and families -- to urge payers to cover the drug as soon as possible to avoid delays and restrictions.
Can I take Trikafta if I have received a lung transplant?
  • Similar to other CFTR modulators, Trikafta is not expected to improve lung function or pulmonary symptoms following a lung transplant.
  • Immunosuppressive drugs are required following transplantation. If Trikafta is considered, potential drug-drug interactions must be anticipated. Before making any changes in your treatment regimen, discuss it with your transplant team.
What financial assistance is available for patients who cannot afford the out-of-pocket cost of Trikafta?
  • Compass is available to help people with CF navigate their insurance, coordinate and maximize benefits, and look for and connect with national and local nonprofit assistance foundations that offer funds that might free up financial resources in other parts of life such as housing or rent. Compass can also connect you with Vertex Pharmaceuticals, the manufacturer of Trikafta, to understand all copay and financial assistance opportunities available.
  • Vertex offers copay assistance for eligible individuals with commercial/private insurance. For more information on Vertex's GPS program, including eligibility requirements for assistance, contact Vertex at 1-877-752-5933 or visit www.VertexGPS.com.
  • National and local nonprofit patient assistance foundations offer out-of-pocket cost assistance for Trikafta as well as other CF-specific medications. Because each program has specific enrollment criteria, it is important to contact them individually to determine your eligibility.
What financial assistance is available for patients on Medicare or Medicaid?
  • Compass is available to help people with CF navigate their insurance, coordinate and maximize benefits, and look for and connect with national and local nonprofit assistance foundations that offer funds that might free up financial resources in other parts of life such as housing or rent. Compass can also help identify programs to understand all copay and financial assistance opportunities available.
  • Although manufacturer programs, including Vertex GPS, do not provide copay assistance to people with public insurance, there are national and local nonprofit foundations that offer out-of-pocket cost assistance for Trikafta as well as other CF-specific medications. Because each program has specific enrollment criteria, it is important to contact them individually to determine your eligibility.

Additional Information

What is the Foundation doing for people who will not benefit from Trikafta?
  • We recognize that not all people with CF will benefit from the triple-combination therapy, and we are steadfast in our commitment to finding treatments for the underlying cause of the disease for all people with CF, including those with two nonsense or other rare mutations. Learn more about the potential treatments the Foundation is exploring for people with nonsense and other rare mutations.
  • We are also committed to developing better treatments for CF beyond fixing the broken CFTR protein, including better antibiotics, mucus medicines, enzymes, and anti-inflammatories.

 

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CFTR Modulators
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Trikafta Approved Mutations, January 2021 Download (PDF)
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