Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026

Abstract
“Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026” Report Highlights:

• Global CFTR Modulators Market Opportunity: > US$ 20 Billion
• Global CFTR Modulators Market Growth: 42% CAGR (2017 -2020)
• Global CFTR Modulators Market Growth In 2020: 55%
• Global CFTR Modulators Pipeline: >30 Drug
• Commercially Available CFTR Modulators: 4 Drugs
• Trikafta Market Share 2020: > 50%

“Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026” provides comprehensive insight on clinical and non-clinical factors that are driving the global CFTR Modulators market and its impact on the global pharmaceutical market landscape. The report has been prepared in a view to deeply profile the current market trends along with the sales insight on 4 commercially available CFTR Modulators with their, market share and the clinical profile.

The advent of CFTR modulators in the management of cystic fibrosis has greatly transformed the overall market. Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies are targeted drugs which have been developed by the researchers to correct the malfunctioning protein made by the CFTR gene. Because different mutations cause different defects in the protein, the medications that have been developed so far are effective only in people with specific mutations.

Currently, four CFTR Modulators have been approved for the management of cystic fibrosis. Among them, Trikafta developed by Vertex Pharmaceuticals is the first triple combination therapy available to treat patients who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population. This drug has shown high clinical response and has significantly enhanced the median survival rates in patients.

Trikafta was recently granted approval in late 2019 and within short span of time, the drug has dominated the overall CFTR modulator drug market. As of 2020, the novel drug Trikafta have sales of more than US$ 3 Billion which is expected to rise at high rates to reach about US$ 5 Billion by 2026. The high market size of the drug is mainly attributed to the increase in prevalence of the disease and the unmet need of combinational therapy in management of them. Moreover, the high adoption rate of this drug is due to its ability to enhance the median survival rates in patients.

The elexacaftor and tezacaftor contained in TRIKAFTA bind to the CFTR protein and facilitate the cellular processing of F508del-CFTR. The combination helps in increasing the amount of CFTR protein delivered to the cell surface, while ivacaftor aids in the gating of the CFTR protein at the cell surface. The combined effect of the three drugs boosts the amount and function of F508del-CFTR at the cell surface. The targeted nature of drug in the management of cystic fibrosis also aids in boosting the growth of CFTR modulator drugs market.

US is currently dominating the market and is expected to do so for next few years. This is mainly due to the rising Caucasian population in the region which has maximum risk of developing the disease. In addition to this, the high adoption rates of novel therapy as well as rising initiative by government to initiate research and development activities will also drive the future of the market. In addition to this, Europe and Asia are also competing to occupy a considerable position in the market by investing a huge amount in research and development.

As per “Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026” report findings, it is expected that the drug Trikafta will dominate the market for next few years which is due to its triple combination which have better ability to tackle the complexity of cystic fibrosis disease. The overall CFTR modulator market will also witness high growth rates and rapid approval of several other modulators which are currently under clinical trials. It is expected that CFTR modulator drugs will emerge as a potential drugs in the management of cystic fibrosis in coming years.

Content
1. Introduction to CFTR Modulators
1.1 Overview
1.2 Evolution of CFTR Modulators

2. CFTR Modulators Mechanism of Action

3. Global CFTR Modulator Market Overview
3.1 Current Market Scenario
3.2 Future Market Potential of CFTR Modulators

4. US CFTR Modulator Market Overview
4.1 Current Market Scenario
4.2 Future Market Potential of CFTR Modulators

5. ROW CFTR Modulator Market Overview
5.1 Current Market Scenario
5.2 Future Market Potential of CFTR Modulators

6. Ivacaftor (Kalydeco) - Clinical & Commercial Insight
6.1 Overview
6.2 Patents & Assignees
6.3 Price & Dosage Insight
6.4 Sales Analysis

7. Ivacaftor/Lumacaftor (Orkambi) - Clinical & Commercial Insight
7.1 Overview
7.2 Patents & Assignees
7.3 Price & Dosage Insight
7.4 Sales Analysis

8. Tezacaftor/Ivacaftor (Symdeko) - Clinical & Commercial Insight
8.1 Overview
8.2 Patent & Assignees
8.3 Price & Dosage Insight
8.4 Sales

9. Elexacaftor/Ivacaftor/Tezacaftor (Trikafta) - Clinical & Commercial Insight
9.1 Overview
9.2 Patent & Assignees
9.3 Price & Dosage Insight
9.4 Global Sales

10. Global CFTR Modulators Vaccine Pipeline Overview
10.1 By Country
10.2 By Company
10.3 By Patient Segment
10.4 By Phase

11. Global CFTR Modulators Clinical Trials Insight
11.1 Research
11.2 Preclinical
11.3 Phase-I
11.4 Phase-I/II
11.5 Phase-II
11.6 Phase-III

12. Marketed CFTR Modulators Clinical Insights

13. CFTR Modulator Drugs Market Dynamics
13.1 Favorable Parameters
13.2 Market Restraints

14. CFTR Modulator Market Future Perspective

15. Competitive Landscape
15.1 Vertex Pharmaceuticals
15.2 Eloxx Pharma
15.3 ReCode Therapeutics
15.4 Arcturus Therapeutics
15.5 AbbVie
15.6 Spirovant Sciences
15.7 Translate Bio
15.8 ReAlta Life Sciences
15.9 Southern Research Institute
15.10 4D Molecular Therapeutics

List of Figures
Figure 1-1: Different Approaches for CFTR Modulators
Figure 1-2: Classes of CFTR Protein Mutations
Figure 1-3: History of CFTR Modulators

Figure 2-1: CFTR Modulators – Specific Target for Different Approaches

Figure 3-1: Global – CFTR Modulator Drugs Market Size (US$ Million), 2017-2020
Figure 3-2: Global – CFTR Modulator Drugs Market Size by Product (US$ Million), 2019 & 2020
Figure 3-3: Global – CFTR Modulator Drugs Market Size by Product (%), 2020
Figure 3-4: Global – CFTR Modulator Drugs Market Size by Product (%), 2019
Figure 3-5: Global – CFTR Modulator Drugs Market (US$ Billion), 2021-2026
Figure 3-6: Global – Cystic Fibrosis Incidence, 2020-2026

Figure 4-1: US – Incidence of Cystic Fibrosis, 2010-2020
Figure 4-2: Global – CFTR Modulator Market Size by Region (US$ Billion), 2020
Figure 4-3: Global – CFTR Modulator Market Size by Region (%), 2020
Figure 4-4: US – Expected CFTR Modulator Drugs Market Size (US$ Billion), 2021-2026
Figure 4-5: US – Cystic Fibrosis Incidence, 2021-2026

Figure 5-1: Global – CFTR Modulator Market Size by Region (US$ Billion), 2020
Figure 5-2: Global – CFTR Modulator Market Size by Region (%), 2020
Figure 5-3: ROW – Expected CFTR Modulator Drugs Market Size (US$ Billion), 2021-2026
Figure 5-4: ROW – Cystic Fibrosis Incidence, 2020-2026

Figure 6-1: US – Kalydeco FDA Approval History
Figure 6-2: Europe – Kalydeco FDA Approval History
Figure 6-3: US- Kalydeco FDA Approval & Patent Expiration Year
Figure 6-4: Europe - Kalydeco FDA Approval & Patent Expiration Year
Figure 6-5: US – Kalydeco Patent Approval & Expiration Year
Figure 6-6: US – Cost of Supply of 56 Oral Granule & Per Unit Cost of Kalydeco Oral Granule (US$), April’2021

Figure 6-7: US – Cost of Supply of 56 Oral Tablet & Per Unit Cost of 150mg Kalydeco Oral Tablet (US$), April’2021
Figure 6-8: US – Cost of Supply of 60 Oral Tablet & Per Unit Cost of 150mg Kalydeco Oral Tablet (US$), April’2021
Figure 6-9: Europe – Cost of Supply of 28 Oral Tablet & Per Unit Cost of 150mg Kalydeco Oral Tablet (Eur/US$), April’2021
Figure 6-10: Europe – Cost of Supply of 56 Oral Tablet & Per Unit Cost of 50mg, 75mg & 150mg Kalydeco Oral Tablet (Eur/US$), April’2021
Figure 6-11: UK – Cost of Supply of 56 Oral Granules & Per Unit Cost of 25mg, 50mg & 75mg Kalydeco Oral Granules (Eur/US$), April’2021
Figure 6-12: UK – Cost of Supply of 56 Oral Tablet & Per Unit Cost of 150mg Kalydeco Oral Tablet (Eur/US$), April’2021
Figure 6-13: UK – Cost of Supply of 28 Oral Tablet & Per Unit Cost of 150mg Kalydeco Oral Tablet (Eur/US$), April’2021
Figure 6-14: Kalydeco – Recommended Pediatric Dose of Oral Granules for Cystic Fibrosis by Weight (mg/12hrs)
Figure 6-15: Kalydeco – Maximum Recommended Pediatric Dose for Cystic Fibrosis by Type (mg/12hrs)
Figure 6-16: Global – Kalydeco Annual Sales (US$ Million), 2017-2020
Figure 6-17: Global – Kalydeco Quarterly Sales (US$ Million), 2020
Figure 6-18: Global – Kalydeco Quarterly Sales (US$ Million), 2019

Figure 7-1: US – Orkambi FDA Approval History by Age
Figure 7-2: Europe – Orkambi FDA Approval History by Age
Figure 7-3: US- Orkambi FDA Approval & Patent Expiration Year
Figure 7-4: Europe - Orkambi FDA Approval & Patent Expiration Year
Figure 7-5: US – Orkambi Patent Approval & Expiration Year
Figure 7-6: US - Cost of Supply of 56 Oral Granule & Per Unit Cost of Orkambi Oral Granule (US$), April'2021
Figure 7-7: US - Cost of Supply of 112 Oral Tablet & Per Unit Cost of Orkambi Oral Tablet (US$), April'2021
Figure 7-8: Europe - Cost of Supply of 112 Oral Tablet & Per Unit Cost of Orkambi Oral Tablet (Euro/US$), April'2021
Figure 7-9: UK- Cost of Supply of 112 Oral Tablet & Per Unit Cost of Orkambi Oral Tablet (Euro/US$), April'2021


Figure 7-10: UK- Cost of Supply of 56 Oral Granules & Per Unit Cost of Orkambi Oral Granule (Euro/US$), April'2021
Figure 7-11: Orkambi - Recommended Dose by Age (mg/day)
Figure 7-12: Global - Orkambi Annual Sales (US$ Million), 2017-2020
Figure 7-13: Global - Orkambi Quarterly Sales (US$ Million), 2020
Figure 7-14: Global - Orkambi Quarterly Sales (US$ Million), 2019

Figure 8-1: US - Symdeko FDA Approval History by Age
Figure 8-2: Europe - Symdevi EU Approval History by Age
Figure 8-3: Europe - Symdevi Approval & Patent Expiration Year
Figure 8-4: US - Symdeko Approval & Patent Expiration Year
Figure 8-5: US - Symdeko Patent Approval & Expiration Year
Figure 8-6: US - Cost of Supply of 56 Oral Tablet & Per Unit Cost of Symdeko Oral Tablet (US$), April'2021
Figure 8-7: Europe - Cost of Supply of 56 Oral Tablet & Per Unit Cost of Symdeko Oral Tablet (Euro/US$), April'2021
Figure 8-8: Europe - Cost of Supply of 28 Oral Tablet & Per Unit Cost of Symkevi Oral Tablet (Euro/US$), April'2021
Figure 8-9: Global - Symdeko/Symkevi Annual Sales (US$ Million), 2018-2020
Figure 8-10: Global - Symdeko/Symkevi Quarterly Sales (US$ Million), 2020
Figure 8-11: Global - Symdeko/Symkevi Quarterly Sales (US$ Million), 2019

Figure 9-1: US - Trikafta FDA Approval History by Mutation
Figure 9-2: Trikafta/Kaftrio - Initial Approval Year by Region
Figure 9-3: Europe - Kaftrio Approval & Patent Expiration Year
Figure 9-4: US - Trikafta Approval & Patent Expiration Year
Figure 9-5: US - Trikafta Patent Approval & Expiration Year
Figure 9-6: US - Cost of Supply of 84 Oral Tablet & Per Unit Cost of Trikafta Oral Tablet (US$), April'2021
Figure 9-7: Europe - Cost of Supply of 56 Oral Tablet & Per Unit Cost of Kaftrio Oral Tablet (Euro/US$), April'2021
Figure 9-8: UK - Cost of Supply of 56 Oral Tablet & Per Unit Cost of Kaftrio Oral Tablet (Euro/US$), April'2021
Figure 9-9: Trikafta/Kaftrio - Recommended Initial & Reduced Dose (mg/day)
Figure 9-10: Global - Trikafta/Kaftrio Annual Sales (US$ Million), 2019 & 2020
Figure 9-11: Global - Trikafta/Kaftrio Quarterly Sales (US$ Million), 2020

Figure 10-1: Global - CFTR Modulators in Clinical Pipeline by Country, 2021 till 2026
Figure 10-2: Global - CFTR Modulators in Clinical Pipeline by Company, 2021 till 2026
Figure 10-3: Global – CFTR Modulators in Clinical Pipeline by Patient Segment, 2021 till 2026
Figure 10-4: Global - CFTR Modulators in Clinical Pipeline by Phase, 2021 till 2026
Figure 13-1: CFTR Modulator Market Favorable Parameters
Figure 13-2: Global - CFTR Modulator Annual Sales by Product (US$ Million), 2020
Figure 13-3: US - CFTR Modulator Annual WAC by Product (US$)
Figure 13-4: Stages of Drug Development