Global CFTR Modulators Market Opportunity, Drug Sales & Clinical Trials Insight 2028

Abstract
Global CFTR Modulators Market Opportunity, Drug Sales & Clinical Trials Insight 2028 Report Insight:

• Global CFTR Modulators Market Opportunity: > USD 25 Billion
• Global CFTR Modulators Market Insights: Current, Regional and Future Outlook 2028
• Annual, Quarterly and Regional Sales Insights On CFTR Modulators
• CFTR Modulators Availability By Dosage, Patent, and Price Analysis
• Clinical Insight On More Than 30 CFTR Modulators In Trials
• CFTR Modulators Clinical Trials Insight By Country, Company & Phase: > 30 Drugs

The approval of first CF transmembrane conductance regulator (CFTR) modulator has changed the face of cystic fibrosis in the era of precision medicine. Cystic fibrosis is a rare, life-shortening genetic disease caused by mutations of the cystic fibrosis transmembrane conductance regulator – or CFTR – gene. These modulators are designed to correct the malfunctioning protein made by the CFTR gene. This drug class has grown considerably from 2012, up to four agents available in the global market. Kalydeco was the first approved CFTR modulatory drug which has shown robust response in the market by significantly increasing the median survival rates in patients.

The complexity of the disease has surged the development of several combinational therapies which have the ability to better tackle the complex nature of the disease. To overcome this, Orkambi, Symdeko, and Trikafta have been approved which belong to the class of CFTR combinational drugs. Currently, the market is dominated by combinational drugs owing to their enhanced efficacy in the management of disease and ability to overcome limitations of monotherapy.

The encouraging response of CFTR modulators in the market has led to further research and development activities in this domain. Several promising compounds are currently being evaluated which can be proven to be alternate to VX-770 to overcome gating mutations. Vertex pharmaceutical is conducting clinical trial on VX-561 (also called deutivacaftor) which an analogue of VX-770 in which one of the tert-butyl groups was replaced by a per-deuterated one. The preclinical data has demonstrated that the drug has similar activity as VX-770; however it has enhanced stability suggesting it could be prescribed once daily only, contrary to VX-770, which must be administered every 12 hours. The company is also developing another corrector, VX-121, which has reached clinical phase 3. It is being assessed in combination with VX-661, VX-770, and/or VX-56.

Although CFTR modulators have shown to dominate the cystic fibrosis treatment market, but there are still several challenges which need to be overcome. The currently available CFTR modulators drugs are target the underlying cause of cystic fibrosis. However, some patients with rare CFTR mutations are still ineligible to receive currently marketed therapies. Therefore, the opportunity remains for developers to investigate CFTR modulators for less common mutations. In addition, resistance against these drugs is another challenge which needs to be overcome.

As per our analysis, the global CFTR modulator market is expected to surpass US$ 25 Billion by 2028. The increasing prevalence and increasing funding from several nonprofit organizations including, Cystic Fibrosis Foundation, Cystic Fibrosis Worldwide, South African Cystic Fibrosis Trust, and South African Cystic Fibrosis Association, will drive the market growth but also encourages the research and development activities in cystic fibrosis. These organizations are investing huge amount of funds to find various CFTR modulators for cystic fibrosis and to support the research and developmental activities by prominent players. Further, robust clinical pipeline of drugs which will gain approval during forecast period will also boost the growth of market.

Our report “Global CFTR Modulators Market Opportunity, Drug Sales & Clinical Trials Insight 2028” enables the key stakeholders about the major trends, drivers, investments, and government initiatives toward the disease treatment. The report also provides comprehensive details of the existing pure-play companies and new players entering the market with key drugs in research and development. Moreover, the report provides insights about the major challenges that are going to hamper the market growth during the forecast period. Additionally, report provides information on the globally approved CFTR modulators along with their patent, price, dosage, and sales analysis till Q1 2022.

Content
Table of Contents

1. Introduction to CFTR Modulators
1.1 Overview
1.2 Evolution of CFTR Modulators
1.3 CFTR Modulators Mechanism of Action

2. Global CFTR Modulator Market Overview
2.1 Current Market Scenario
2.2 Future Market Potential of CFTR Modulators

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

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

5. Ivacaftor (Kalydeco) - Clinical & Commercial Insight
5.1 Overview
5.2 Patents & Assignees
5.3 Price & Dosage Insight
5.4 Sales Analysis

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

7. Tezacaftor/Ivacaftor (Symdeko) - Clinical & Commercial Insight
7.1 Overview
7.2 Patent & Assignees
7.3 Price & Dosage Insight
7.4 Sales

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

9. Global CFTR Modulators Clinical Pipeline Overview
9.1 By Country
9.2 By Company
9.3 By Patient Segment
9.4 By Phase

10. Global CFTR Modulators Clinical Trials Insight
10.1 Research
10.2 Preclinical
10.3 Phase-I
10.4 Phase-I/II
10.5 Phase-II
10.6 Phase-III

11. Marketed CFTR Modulators Clinical Insights

12. CFTR Modulator Drugs Market Dynamics
12.1 Favorable Parameters
12.2 Market Restraints

13. CFTR Modulator Market Future Perspective

14. Competitive Landscape
14.1 Vertex Pharmaceuticals
14.2 Eloxx Pharma
14.3 ReCode Therapeutics
14.4 Arcturus Therapeutics
14.5 AbbVie
14.6 Spirovant Sciences
14.7 Translate Bio
14.8 ReAlta Life Sciences
14.9 Southern Research Institute
14.10 4D Molecular Therapeutics


List of Figure
Figure 1-1: Different Approaches for CFTR Modulators
Figure 1-2: Classes of CFTR Protein Mutations
Figure 1-3: History of CFTR Modulators
Figure 1-4: CFTR Modulators – Specific Target for Different Approaches

Figure 2-1: Global – CFTR Modulator Drugs Market Size (US$ Million), 2017-2021
Figure 2-2: Global – CFTR Modulator Drugs Market Size by Product (US$ Million),
2020 & 2021
Figure 2-3: Global – CFTR Modulator Drugs Market Size by Product (%), 2021
Figure 2-4: Global – CFTR Modulator Drugs Market Size by Product (%), 2020
Figure 2-5: Global – CFTR Modulator Drugs Market (US$ Billion), 2022-2028
Figure 2-6: Global – Cystic Fibrosis Incidence, 2020-2026

Figure 3-1: US – Incidence of Cystic Fibrosis, 2010-2020
Figure 3-2: Global – CFTR Modulator Market Size by Region (US$ Billion), 2021
Figure 3-3: Global – CFTR Modulator Market Size by Region (%), 2021
Figure 3-4: US – Expected CFTR Modulator Drugs Market Size (US$ Billion), 2022 –
2028
Figure 3-5: US – Cystic Fibrosis Incidence, 2021-2026

Figure 4-1: Global – CFTR Modulator Market Size by Region (US$ Billion), 2021
Figure 4-2: Global – CFTR Modulator Market Size by Region (%), 2021
Figure 4-3: ROW – Expected CFTR Modulator Drugs Market Size (US$ Billion), 2020 –
2028
Figure 4-4: ROW – Cystic Fibrosis Incidence, 2020-2026

Figure 5-1: US – Kalydeco FDA Approval History
Figure 5-2: Europe – Kalydeco FDA Approval History
Figure 5-3: US- Kalydeco FDA Approval & Patent Expiration Year
Figure 5-4: Europe - Kalydeco FDA Approval & Patent Expiration Year
Figure 5-5: US – Kalydeco Patent Approval & Expiration Year
Figure 5-6: US – Cost of Supply of 56 Oral Granule & Per Unit Cost of Kalydeco Oral
Granule (US$), May’2022
Figure 5-7: US – Cost of Supply of 56 Oral Tablet & Per Unit Cost of 150mg Kalydeco
Oral Tablet (US$), May’2022
Figure 5-8: Europe – Cost of Supply of 28 Oral Tablet & Per Unit Cost of 150mg
Kalydeco Oral Tablet (Eur/US$), May’2022
Figure 5-9: Europe – Cost of Supply of 56 Oral Tablet & Per Unit Cost of 50mg, 75mg
& 150mg Kalydeco Oral Tablet (Eur/US$), May’2022
Figure 5-10: UK – Cost of Supply of 56 Oral Granules & Per Unit Cost of 25mg, 50mg
& 75mg Kalydeco Oral Granules (GBP/US$), May’2022
Figure 5-11: UK – Cost of Supply of 56 Oral Tablet & Per Unit Cost of 150mg
Kalydeco Oral Tablet (GBP/US$), May’2022
Figure 5-12: UK – Cost of Supply of 28 Oral Tablet & Per Unit Cost of 150mg
Kalydeco Oral Tablet (GBP/US$), May’2022
Figure 5-13: Kalydeco – Recommended Pediatric Dose of Oral Granules for Cystic
Fibrosis by Weight (mg/12hrs)
Figure 5-14: Kalydeco – Maximum Recommended Pediatric Dose for Cystic Fibrosis
by Type (mg/12hrs)
Figure 5-15: Global - Kalydeco Sales (US$ Million), Q1’2021 & Q1’2022
Figure 5-16: Global – Kalydeco Annual Sales (US$ Million), 2017-2021
Figure 5-17: Global – Kalydeco Quarterly Sales (US$ Million), 2021
Figure 5-18: Global – Kalydeco Quarterly Sales (US$ Million), 2020
Figure 5-19: Global – Kalydeco Quarterly Sales (US$ Million), 2019

Figure 6-1: US – Orkambi FDA Approval History by Age
Figure 6-2: Europe – Orkambi FDA Approval History by Age
Figure 6-3: US- Orkambi FDA Approval & Patent Expiration Year
Figure 6-4: Europe - Orkambi FDA Approval & Patent Expiration Year
Figure 6-5: US – Orkambi Patent Approval & Expiration Year
Figure 6-6: US - Cost of Supply of 56 Oral Granule & Per Unit Cost of Orkambi Oral
Granule (US$), May’2022
Figure 6-7: US - Cost of Supply of 112 Oral Tablet & Per Unit Cost of Orkambi Oral
Tablet (US$), May’2022
Figure 6-8: Europe - Cost of Supply of 112 Oral Tablet & Per Unit Cost of Orkambi
Oral Tablet (Euro/US$), May’2022
Figure 6-9: UK- Cost of Supply of 112 Oral Tablet & Per Unit Cost of Orkambi Oral
Tablet (GBP/US$), May’2022
Figure 6-10: UK- Cost of Supply of 56 Oral Granules & Per Unit Cost of Orkambi Oral
Granule (GBP/US$), May’2022

Figure 6-11: Orkambi - Recommended Dose by Age (mg/day)
Figure 6-12: Global - Orkambi Sales (US$ Million), Q1’2021 & Q1’2022
Figure 6-13: Global - Orkambi Annual Sales (US$ Million), 2017-2021
Figure 6-14: Global - Orkambi Quarterly Sales (US$ Million), 2021
Figure 6-15: Global - Orkambi Quarterly Sales (US$ Million), 2020
Figure 6-16: Global - Orkambi Quarterly Sales (US$ Million), 2019

Figure 7-1: US - Symdeko FDA Approval History by Age
Figure 7-2: Europe - Symdevi EU Approval History by Age
Figure 7-3: Europe - Symdevi Approval & Patent Expiration Year
Figure 7-4: US - Symdeko Approval & Patent Expiration Year
Figure 7-5: US - Symdeko Patent Approval & Expiration Year
Figure 7-6: US - Cost of Supply of 56 Oral Tablet & Per Unit Cost of Symdeko Oral
Tablet (US$), May’2022
Figure 7-7: Europe - Cost of Supply of 56 Oral Tablet & Per Unit Cost of Symdeko
Oral Tablet (Euro/US$), May’2022
Figure 7-8: Europe - Cost of Supply of 28 Oral Tablet & Per Unit Cost of Symkevi
Oral Tablet (GBP/US$), May’2022
Figure 7-9: Global - Symdeko/Symkevi Sales (US$ Million), Q1’2021 & Q1’2022
Figure 7-10: Global - Symdeko/Symkevi Annual Sales (US$ Million), 2018-2021
Figure 7-11: Global - Symdeko/Symkevi Quarterly Sales (US$ Million), 2021
Figure 7-12: Global - Symdeko/Symkevi Quarterly Sales (US$ Million), 2020
Figure 7-13: Global - Symdeko/Symkevi Quarterly Sales (US$ Million), 2019

Figure 8-1: US - Trikafta FDA Approval History by Mutation
Figure 8-2: Trikafta/Kaftrio - Initial Approval Year by Region
Figure 8-3: Europe - Kaftrio Approval & Patent Expiration Year
Figure 8-4: US - Trikafta Approval & Patent Expiration Year
Figure 8-5: US - Trikafta Patent Approval & Expiration Year
Figure 8-6: US - Cost of Supply of 84 Oral Tablet & Per Unit Cost of Trikafta Oral
Tablet (US$), May’2022
Figure 8-7: Europe - Cost of Supply of 56 Oral Tablet & Per Unit Cost of Kaftrio Oral
Tablet (Euro/US$), May’2022
Figure 8-8: UK - Cost of Supply of 56 Oral Tablet & Per Unit Cost of Kaftrio Oral
Tablet (GBP/US$), May’2022
Figure 8-9: Trikafta/Kaftrio - Recommended Initial & Reduced Dose (mg/day)
Figure 8-10: Global - Trikafta/Kaftrio Sales (US$ Million), Q1’2021 & Q1’2022
Figure 8-11: Global - Trikafta/Kaftrio Annual Sales (US$ Million), 2019 - 2021
Figure 8-12: Global - Trikafta/Kaftrio Quarterly Sales (US$ Million), 2021
Figure 8-13: Global - Trikafta/Kaftrio Quarterly Sales (US$ Million), 2020

Figure 9-1: Global - CFTR Modulators in Clinical Pipeline by Country, 2022 till 2028
Figure 9-2: Global - CFTR Modulators in Clinical Pipeline by Company, 2022 till 2028
Figure 9-3: Global – CFTR Modulators in Clinical Pipeline by Patient Segment, 2022
till 2028
Figure 9-4: Global - CFTR Modulators in Clinical Pipeline by Phase, 2022 till 2028
Figure 12-1: CFTR Modulator Market Favorable Parameters
Figure 12-2: Global - CFTR Modulator Annual Sales by Product (US$ Million), 2020
Figure 12-3: US - CFTR Modulator Annual WAC by Product (US$)
Figure 12-4: Stages of Drug Development