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.
TRIKAFTA
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
