Trikafta (elexacaftor, tezacaftor, and ivacaftor tablets; ivacaftor tablets): For Cystic Fibrosis

Introduction

Trikafta is a groundbreaking medication that received initial approval from the U.S. Food and Drug Administration (FDA) in 2019. This innovative drug offers hope to individuals with cystic fibrosis (CF) aged six years and older who harbor specific genetic mutations. In this comprehensive overview, we will delve into its FDA approval, indications, mechanism of action, proper dosage and administration, any contraindications and warnings, potential side effects, and recent research developments, and conclude with the significance of this drug.

FDA Approval

Trikafta achieved initial FDA approval in 2019, marking a significant milestone in the treatment of cystic fibrosis.

Indications

Trikafta is prescribed for the management of cystic fibrosis in individuals aged six years and above, provided they have either one F508del mutation in the CFTR gene or a mutation in the CFTR gene that demonstrates reactivity based on in vitro data.

Mechanism of Action

This groundbreaking medication combines three active ingredients: elexacaftor, tezacaftor, ivacaftor, each playing a pivotal role in managing CF. Ivacaftor is a CFTR potentiator, while tezacaftor and elexacaftor work together to enhance CFTR protein function. This combination addresses the underlying genetic defects associated with CF. It improves chloride ion transport, ultimately aiding in the reduction of thickened mucus production in the airways.

Dosage and Administration

The recommended dosage varies according to age and weight:

• For patients aged 6 to less than 12 years weighing less than 30 kgs, the morning dose includes two tablets of elexacaftor 50mg, tezacaftor 25mg, and ivacaftor 37.5mg, with ivacaftor tablet of  75mg in the evening.
• For patients aged 6 to less than 12 years weighing 30 kgs or more, the morning dose involves two tablets of elexacaftor 100mg, tezacaftor 50mg, and ivacaftor 75mg, with one tablet of ivacaftor 150mg in the evening.
• Patients aged 12 years and older are prescribed two tablets of elexacaftor 100mg, tezacaftor 50mg, and ivacaftor 75mg in the morning, along with one tablet of ivacaftor 150mg in the evening. It is crucial to take Trikafta with a fat-containing meal to optimize absorption.

Contraindications and Warnings

Trikafta does not have any specified contraindications, but it is essential to be aware of certain warnings and precautions:

• Elevated liver function tests: Regularly monitoring ALT, AST, and bilirubin levels is recommended. Treatment should be interrupted if ALT or AST exceeds specific thresholds.
• Use with CYP3A inducers: Co-administration with strong CYP3A inducers is not advised due to potential reductions in efficacy.
• Ophthalmic evaluations are recommended in pediatric patients before initiating Trikafta.

Side Effects

Common adverse reactions include:

• Headache
• Upper respiratory tract infections
• Abdominal pain
• Diarrhea
• Rash
• Increased liver enzymes (alanine aminotransferase and aspartate aminotransferase)
• Nasal congestion
• Elevated blood creatine phosphokinase
• Rhinorrhea
• Rhinitis
• Influenza
• Sinusitis
• Elevated blood bilirubin

Recent Research

A study published in the New England Journal of Medicine found that Trikafta can provide additional benefit for CF patients who have genetic mutations that made them eligible for previously approved CFTR modulators.

Ongoing research in the field of CF continues to explore the long-term safety and efficacy of Trikafta. Recent studies focus on the medication’s impact on disease progression, patient outcomes, and potential applications in younger age groups.

Conclusion

Trikafta represents a remarkable advancement in the treatment of cystic fibrosis. With its innovative mechanism of action, well-defined dosage guidelines, and ongoing research, it offers real hope to those affected by this challenging genetic condition. The FDA’s approval of Trikafta in 2019 marked a significant step forward in improving the quality of life for patients with CF, and the continued research in the field promises even more progress in the years to come. Trikafta price in India depends on the product requirement. Request more details by contacting our Patient Support Team at ‎(+91) 9315705373 or help@sansfro.com.  

Reference

1. https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis
2. https://www.cff.org/news/2023-04/trikafta-approval-ages-2-5-mutations
3. https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/212273s004lbl.pdf