Top Performing Drug – Trikafta/Kaftrio (May Edition)

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Top Performing Drug – Trikafta/Kaftrio (May Edition)


  • In continuation of our previous series on the top-performing drug of the month, based on 2021 revenue, this month we have covered Trikafta/Kaftrio as the top-performing drug of the month
  • Trikafta is a combination of ivacaftor, tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis in pediatric patients who have at least one F508del mutation in the CFTR gene
  • PharmaShots presents an informative take on the key features of Trikafta/Kaftrio with a detailed analysis of its revenue, clinical trials, alternatives, and approvals. The report is concluded with an engaging SWOT analysis and edifying KOL reviews

Active Ingredient: (elexacaftor, tezacaftor, and ivacaftor tablets, ivacaftor tablets)

Dosage Forms & Strengths: Tablets: fixed-dose combination containing elexacaftor 100 mg, tezacaftor 50 mg, and ivacaftor 75 mg.

Mechanism of Action: CFTR stimulants

Originator: Vertex Pharmaceuticals

First Approvals: Below mentioned are the first approvals of Trikafta from different regulatory agencies.

*The brand name of the drug is Trikafta in the US and Kaftrio in Europe.

Revenue Analysis1

Trikafta/Kaftrio is developed and commercialized by Vertex Pharmaceuticals. Trikafta is now approved and accessible in more than 30 countries outside the U.S., including the United Kingdom, Australia, and Canada. In 2022, Vertex Pharma’s net product revenue spiked by 18%, vs. 2021, mainly due to influx of Trikafta in multiple countries internationally and the growing performance of Trikafta in the U.S.

The analysis of Trikafta/Kaftrio’s past 4 years (as approved in 2019) revenue is showcased below through a graphical representation.

Trikafta/Kaftrio Approved Indications2

Trikafta/Kaftrio is a combination drug indicated for the treatment of cystic fibrosis in patients as young as 2 years old with at least one F508del mutation in the CFTR gene.

Clinical Trials Analysis3

Trikafta/Kaftrio has currently 27 ongoing trials, incl. 10 Interventional trials and 17 Observational trials.

PharmaShots' analysis of Trikafta/Kaftrio’s clinical trials is depicted below: (Trials were taken on 9th May 2023).

Trikafta/Kaftrio’s Trials Representation (Country-wise)4

The stacked column chart below is a visual representation of the clinical trials conducted with Trikafta/Kaftrio so far. We have showcased active clinical trials of the top 10 countries worldwide. (The chart depicts data till 10th May 2023)  

Product Dashboard

PharmaShots has created a product dashboard that presents key metrics and data on Trikafta/Kaftrio in a visual format.

Trikafta/Kaftrio Alternative Drugs5

In response to Trikafta/Kaftrio, several other drugs are available in the market that are used for the treatment of Cystic Fibrosis. Some of these are mentioned below:

Trikafta/Kaftrio SWOT Analysis6


  • High Efficacy: Trikafta has shown significant efficacy in treating cystic fibrosis (CF). Clinical trials have shown considerable improvements in lung function, reduction in exacerbations, and improved quality of life for patients.
  • Expanded Patient Population: Trikafta is approved for use in patients aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This wider patient population allows more individuals with CF to benefit from the treatment.
  • Combination Therapy: Trikafta combines three drugs—elexacaftor, tezacaftor, and ivacaftor—to address the underlying CFTR defects. This triple combination therapy targets multiple mutations, leading to improved clinical outcomes for a broader range of patients.
  • Disease Modification: Trikafta has shown the potential to modify the underlying disease progression of cystic fibrosis. By addressing the defective CFTR protein, it can slow down the decline in lung function and improve long-term outcomes for patients.
  • Growing Potential: Trikafta is now approved in more than 30 countries outside the U.S. including Europe, the UK, Australia, and Canada thus increasing its global recognition and acceptance as a highly beneficial treatment option.


  • Cost and Accessibility: Trikafta is a high-cost medication, which can pose challenges in terms of affordability and accessibility for some patients. The cost may limit its availability in certain healthcare systems and regions, creating disparities in access.
  • Limited Coverage: Insurance coverage and reimbursement policies may vary, leading to potential barriers for some patients in accessing Trikafta. This limitation could hinder the widespread adoption and use of the medication.
  • Adverse Effects: Trikafta is associated with certain side effects, including respiratory symptoms, elevated liver enzymes, and drug-drug interactions. Monitoring and management of these potential adverse effects are necessary to ensure patients’ safety.


  • Global Market Expansion: Trikafta has been approved in several countries, but there is still potential for further global market expansion. Access to Trikafta in diverse regions could benefit more CF patients worldwide.
  • Combination Therapies: There is an ongoing research and development program in the field of CFTR modulators, including the exploration of novel combination therapies. Trikafta's success and experience could potentially lead to the development of more effective and targeted treatments for cystic fibrosis.
  • Expanded Indications: Although, Trikafta is approved for Cystic Fibrosis, its expansion in different indications or for the different genetic mutations for Cystic Fibrosis in the future can amplify its growth.


  • Growing competition: Other pharmaceutical companies are developing CFTR modulators and treatments for cystic fibrosis. Increased competition in the market may impact Trikafta's market share and adoption.
  • Unknown Long-term Effects: As Trikafta is a relatively new medication, the long-term effects and potential side effects are still being studied. Continued research is needed to better understand its safety profile and any potential long-term risks.
  • Regulatory Challenges: Obtaining regulatory approvals for new treatments and expanding indications for Trikafta in different countries can be a time-consuming and complex process. Regulatory requirements and policies may differ, potentially delaying access to Trikafta in certain regions.

Patient Stories7

Patients' stories have been instrumental in sharing their perspectives on individual experiences in facing health challenges. These are a few key resources to understand healthcare services and policies. Some of the patients’ stories for Trikafta/Kaftrio are mentioned below:

SARAH’S STORY: Sarah had a serious chest infection and pneumonia when she was 15. She underwent multiple procedures to prevent chest infections after, which her consultant Professor Martin Walshaw offered her the drug Kaftrio and found it miraculous. She says, I felt the impact in just six hours. I started coughing up loads of mucus; usually, it is so sticky and thick it doesn’t come out, but this was like water and kept coming. It was incredible, very exciting but also overwhelming to get my head round it, after always thinking I would die before my mum and accepting it

KATE’S STORY: Kate, a 7-year-old girl, became the first young child to be offered Kaftrio on the NHS. She felt better and can now breathe without coughing. Kate says, “I am really happy because it is a life-changing drug, and it is going to help me a lot when I am older”

JAMES’S STORY: James suffered from lung puncture several times after which it was expected that he had to undergo a lung transplant. He was administered Kaftrio and felt an instant effect. He says, “Kaftrio has completely changed my life. Before I was just surviving, now I am living my life to the full; my life has been given back to me and I can’t thank the hospital team enough for what they have done, researching and pushing for me to get the drug”

SAMANTHA’S STORY: Samantha was diagnosed with CF when she was 9 and it got worse when she turned 23. Her health deteriorated and expected to undergo a lung transplant. She was offered Trikafta and in 3 hours of the first dose, there was a noticeable change. Samantha says, “Trikafta has saved my life, has given my kids a mom, and my husband an equal partner. Trikafta has given me a future that I couldn’t see anymore. Trikafta has given me the ability to do the simplest of tasks without effort, like sleeping, walking, and breathing”

KOL* Reviews8

KOL reviews are valuable resources for any drug to increase its reach and reliability. Generally, these reviews are helpful when consumers research the product and read multiple reviews before buying it. Below are some of the KOL reviews for Trikafta/Kaftrio.

1. In Oct 2019, Trikafta was approved by the US FDA for patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ned Sharpless, M.D, FDA Commissioner: “At the FDA, we’re consistently looking for ways to help speed the development of new therapies for complex diseases, while maintaining our high standards of review. Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to additional effective therapy.”

2. Jennifer Goralski, M.D., Assistant Professor of Medicine and Pediatrics, Co-Director, Adult Cystic Fibrosis Center, University of North Carolina School of Medicine, said: "Early intervention with CFTR modulator therapies like TRIKAFTA can offer the potential to improve the trajectory of CF lung disease. With this approval, we now have the ability to treat young children with TRIKAFTA and can proactively address the underlying cause of their disease.”

3. Terri Laguna M.D., M.S.C.S., Associate Director of the Cystic Fibrosis Center and Division Head, Pulmonary and Sleep Medicine, Ann & Robert H. Lurie Children’s Hospital of Chicago, said: “Clinical experience with TRIKAFTA in patients 12 and older over the past 20 months has demonstrated this medicine has a meaningful and unprecedented clinical benefit for patients. I look forward to now being able to treat younger patients with this breakthrough medicine, including those who have not presented major signals of disease progression.”

4. After the approval of Trikafta/Kaftrio for children ages 6 to 11 years with cystic fibrosis in Jun 2021, Mandana Emamzadeh, Ph.D., Healthcare Analyst at GlobalData said “Trikafta/Kaftrio has greatly expanded the number of CF-related mutations that are eligible for CFTR modulator treatment across age groups. The drug is reported to decrease the use of symptomatic drug classes such as antibiotics, mucolytics, and anti-inflammatory drugs due to overall improvements to lung health in patients. As a result, the overall survival of CF patients is expected to increase remarkably over the next decades.”

5. In Apr 2023, Vertex announced the U.S. FDA Approval for Trikafta in Children with Cystic Fibrosis Ages 2 Through 5 With Certain Mutations, Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex said: “Since its initial approval, Trikafta has had a significant impact on the CF community, transforming the lives of thousands of people living with cystic fibrosis.”

* Key Opinion Leaders (KOLs) are crucial when it comes to the launch and assessment of pharmaceutical products. At Octavus, we recognize the importance of KOLs in the industry, which is why our proficient team dedicatedly tracks their activities and provides valuable insights to the pharma fraternity. 

We understand that KOL tracking and selection can be overwhelming and time-consuming. That's why we offer extensive KOL tracking services to help our clients stay ahead of the curve. Our team of experts can provide you with the latest information on KOL activities, including their opinions, publications, and affiliations.   

Interested in learning more about our KOL tracking services? Don't hesitate to reach out to us at bd@octavusconsulting.com or connect@pharmashots.com. We would be more than happy to provide you with more information and discuss how our services may benefit your business.   

Octavus is a dedicated consulting company that offers a one-stop market solution to life science enterprises, biopharma, MedTech, diagnostic centres, digital health companies, animal healthcare, and start-ups.


  1. Vertex Pharmaceuticals 10-K
  2. Trikafta Prescribing Information
  3. Clinical Trial Analysis
  4. Trikafta Trials Representation (Country-wise)
  5. Trikafta Alternative Drugs
    1. accessdata.fda.gov
    2. ema.europa.eu
    3. drugbank.com
    4. rxlist.com
  6. Trikafta’s SWOT Analysis
    1. accessdata.fda.gov
    2. pubmed.ncbi.nlm.nih.gov
    3. trikafta.com
  7. Patient Stories
    1. lhch.nhs.uk
    2. independent.co.uk
    3. cysticfibrosis.ca
  8. KOL Reviews
    1. Vertex Pharmaceutical Press Releases
    2. fda.gov/news
    3. globaldata.com
    4. businesswire.com

Related Post: Top Performing Drug – Dupixent (April Edition)

Senior Editor

Senior Editor at PharmaShots. She is curious and very passionate about recent updates and developments in the life sciences industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots.

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