World CFTR Modulators Market Insights: Current, Regional and Future Outlook 2028: > $25 Billion Market Opportunity, >30 Pipeline Drugs

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DUBLIN, June 27, 2022 /PRNewswire/ -- The "Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2028" report has been added to's offering.

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The report 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 approval of the 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, with 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 can 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 the 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 alternatives to VX-770 to overcome gating mutations. Vertex pharmaceutical is conducting a clinical trial on VX-561 (also called deutivacaftor) an analog of VX-770 in which one of the tert-butyl groups was replaced by a per-deuterated one. The preclinical data have 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 been shown to dominate the cystic fibrosis treatment market, there are still several challenges that need to be overcome. The currently available CFTR modulators drugs 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 to these drugs is another challenge that needs to be overcome.

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 amounts of funds to find various CFTR modulators for cystic fibrosis and to support the research and developmental activities of prominent players. Further, the robust clinical pipeline of drugs that will gain approval during the forecast period will also boost the growth of the market.

The report 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 into the major challenges that are going to hamper the market growth during the forecast period. Additionally, the report provides information on the globally approved CFTR modulators along with their patent, price, dosage, and sales analysis till Q1 2022.

Key Report Highlights:

  • 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, and Phase: > 30 Drugs

Key Topics Covered:

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

2. Global CFTR Modulators Mechanism of Action

3. US CFTR Modulator Market Overview

4. ROW CFTR Modulator Market Overview

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

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

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

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

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

10. 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

11. Marketed CFTR Modulators Clinical Insights

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

13. CFTR Modulator Market Future Perspective

14. Competitive Landscape

  • Vertex Pharmaceuticals

  • Eloxx Pharma

  • ReCode Therapeutics

  • Arcturus Therapeutics

  • AbbVie

  • Spirovant Sciences

  • Translate Bio

  • ReAlta Life Sciences

  • Southern Research Institute

  • 4D Molecular Therapeutics

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