TRIKAFTA Approved for Pediatric Patients with F508del-Mutated Cystic Fibrosis

In a news release from June 9, 2021, biotechnology company Vertex Pharmaceuticals, Inc. (“Vertex”) shared that its therapy TRIKAFTA (elexacaftor/texacaftor/ivacaftor and ivacaftor) was approved for expanded use. Now, the treatment is available for pediatric patients (ages 6-11) with cystic fibrosis (CF) who have at least one F508del mutation – or another mutation that responds to TRIKAFTA – within the CFTR gene. Altogether, this opens the door to around 1,500 pediatric patients who can now be treated in a more targeted manner for the first time.


Prior to this most recent expanded use approval, TRIKAFTA was approved for patients with CF aged 12+. According to Cystic Fibrosis News, TRIKAFTA is:

a CFTR modulator that helps defective CFTR proteins work more effectively. Both elexacaftor and texacaftor work as correctors [and] bind to the faulty CFTR protein and help it fold correctly, [while] ivacaftor is a potentiator: it binds to the CFTR protein and holds the channel open so that more salt can pass through it.

The expanded use approval hinged on data from a Phase 3 clinical trial. Altogether, 66 pediatric patients (aged 6-11) with CF enrolled. Patients either had two F508del mutations or one F508del mutation with another minimal function mutation. During the trial, researchers hoped to evaluate the safety, efficacy, tolerability, and pharmacokinetics of TRIKAFTA. Researchers determined that TRIKAFTA was relatively safe and well-tolerated.

With this new approval, Vertex hopes that TRIKAFTA can improve patients’ quality of life. Additionally, the therapy is now available in more dosages to offer better treatment options to patients. In the future, Vertex hopes to also gain expanded use within the European Union (EU), as well as Switzerland, Israel, and Australia. Want to learn more about TRIKAFTA? Check here.

TRIKAFTA Safety Data

While taking TRIKAFTA, patients should avoid using St. John’s wort, seizure medication, or certain antibiotics. Additionally, patients should avoid grapefruit juice or any food/drinks containing grapefruit while using TRIKAFTA. Although TRIKAFTA is relatively safe, some adverse reactions may occur. These include:

  • Abdominal pain
  • Headache
  • Diarrhea
  • Upper respiratory tract infections
  • Rash
  • Cataracts
  • Dizziness
  • Increases in: creatine phosphokinase, liver enzymes, blood bilirubin

However, if you or someone you is taking TRIKAFTA and experiences any of the following symptoms, please contact your doctor immediately:

  • Jaundice (yellowing of the skin and eyes)
  • Appetite loss
  • Nausea and vomiting
  • Abdominal pain in the upper-right corner
  • Dark or amber urine

Cystic Fibrosis (CF)

A variety of CFTR gene mutations cause cystic fibrosis (CF), a progressive genetic condition causing respiratory and digestive system damage. These mutations cause CFTR protein malfunction. As a result, salt movement is interrupted throughout the body. Ultimately, this causes the formation of thick, sticky mucus (as opposed to healthy, slippery mucus) to accumulate throughout various organs. Because CF is inherited in an autosomal recessive pattern, it means patients must inherit one defective gene from each parent to have CF.

As the mucus builds up within the body, it causes a variety of health problems. For example, in the digestive system, mucus accumulation prevents the body from releasing digestive enzymes. As a result, patients cannot absorb nutrients as well. In the airways, the mucus causes breathing problems, frequent infections, and lung damage. In some patients, CF can even cause respiratory failure.

An estimated 1 in every 2500-3500 Caucasian newborns within America have CF. Typically, CF is rarer in other ethnic backgrounds. Most patients are diagnosed before 2 years old. Symptoms of CF include:

  • Salty-tasting skin
  • Greasy or foul-smelling stool
  • Persistent coughing and wheezing
    • Note: One complication of CF also includes a cough which produces blood.
  • Exercise intolerance
  • Recurrent stuffy nose
  • Shortness of breath and/or difficulty breathing
  • Frequent lung infections
  • Male infertility
  • Osteoporosis
  • Meconium ileus, an intestinal blockage that occurs in newborns
  • Poor weight gain
  • Constipation
  • Nasal polyps
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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