Harbinger Times

“The Cystic Fibrosis Market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period (2023-2034). Furthermore, launching various multiple- Cystic Fibrosis pipeline products will significantly revolutionize the Cystic Fibrosis market dynamics”

 

The Cystic Fibrosis market report provides current treatment practices, Cystic Fibrosis drugs, market share of individual therapies, and current and forecasted 7MM Cystic Fibrosis market size from 2020 to 2034. The report also covers current Cystic Fibrosis treatment market algorithms and unmet medical needs to curate the best opportunities and assess the market’s underlying potential.

 

Key Takeaways from the Cystic Fibrosis Market Research Report

• The increase in Cystic Fibrosis Market Size is a direct consequence of the expected launch of potential therapies, along with increasing awareness and development of novel therapies.
• The estimates show that in 2021, the Cystic Fibrosis diagnosed prevalence in the United States was 49% of all the cases in the 7MM.
• As per DelveInsight’s analysis, a higher percentage of diagnosed prevalent cases were males, except Japan, where females occupy a larger patient pool than males.
• The leading Cystic Fibrosis Companies working in the market include Chiesi Farmaceutici S.p.A., Medidata Solutions, Bayer, Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Vertex Pharmaceuticals, Santhera Pharmaceuticals, Laurent Pharmaceuticals Inc., and others.
• Promising Cystic Fibrosis Pipeline Therapies in the various stages of development include Ciprofloxacin (Cipro Inhale), BAYQ3939, P-1037, Hypertonic Saline, VX-371 + HS, inhaled mannitol, and others.
• January 2024: 4D Molecular Therapeutics announced a study of Phase 1 & 2 clinical trials for 4D-710. This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with CF who are ineligible for or unable to tolerate CFTR modulator therapy.
• December 2023: Vertex Pharmaceuticals announced a study of Phase 3 clinical trials for VX-121/TEZ/D-IVA. The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
• December 2023: Arcturus Therapeutics Inc. announced a study of Phase 1 clinical trials for ARCT-032.  Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-032 in healthy adult subjects (Phase 1) and of two doses in Adults with Cystic Fibrosis (Phase 1b).

 

Discover more about therapies set to grab major Cystic Fibrosis Market Share @ Cystic Fibrosis Market Size

 

Cystic Fibrosis Overview

Cystic fibrosis (CF) is an autosomal recessive condition known as brought on by mutations in the Cystic Fibrosis transmembrane conductance regulator (CFTR) gene. F508del is the most prevalent variation reported globally, but there are over 2000 others, although not all of these are disease-causing.

 

Cystic Fibrosis Epidemiology Segmentation in the 7MM

• Cystic Fibrosis Diagnosed Prevalent Cases
• Cystic Fibrosis Age-specific Diagnosed Prevalent Cases
• Cystic Fibrosis Gender-specific Diagnosed Prevalent Cases
• Cystic Fibrosis Mutation-specific Diagnosed Prevalent Cases

 

Download the report to understand which factors are driving Cystic Fibrosis Epidemiology Trends @ Cystic Fibrosis Epidemiological Insights

 

Cystic Fibrosis Drugs Market

The current drugs for CF focus on symptomatic improvement, complication prevention as well as more recently, protein rectifiers to correct underlying structural and functional abnormalities. The paradigm includes inhaled antibiotics for treatment of chronic lung infection, mucolytic for reducing the viscosity of pulmonary mucus, pancreatic enzyme replacement therapy (PERT) for the treatment of CF-associated exocrine pancreatic insufficiency(PPI), and CFTR modulators for enhancing CFTR function, targeting the underlying cause of the disease.

 

Cystic Fibrosis Market Insights

In condition such as Cystic Fibrosis (CF), close collaboration among all stakeholders is important because collaborative studies and systematic data collection will help further development in the disease management. As a next step, a global prospective study will help all the research fraternity in better understanding of biomarkers and diagnostic tests which will be relevant for future therapeutic clinical trials. In the present scenario a number of agents are being investigated. These molecules are in early, middle and late Phase of their clinical investigation.

 

To know more about Cystic Fibrosis Treatment options, visit @ Cystic Fibrosis Drugs

 

Cystic Fibrosis Companies and Therapies

• Vertex Pharmaceuticals: VX-659
• Proteostasis Therapeutics Inc.: PTI-808
• Galapagos NV: GLPG2222 50 mg
• InterMune: Interferon Gamma-1b
• Verona Pharma PLC: RPL554
• PTC Therapeutics: Ataluren

 

Cystic Fibrosis Drugs Uptake

ELX-02 (NB-124) is a synthetic aminoglycoside that acts as a ribosome protein modulator. It is a eukaryotic ribosomal selective glycoside (ERSG) that has designed to increase the read-through activity in patients with nonsense mutations which enables the production of sufficient amounts of full-length functional protein to restore activity. The US FDA granted FTD for ELX-02, Eloxx Pharmaceuticals, Inc., drug candidate intended to treat cystic fibrosis patients with nonsense mutations. In addition, the FDA and European Commission granted ELX-02, ODD for the Cystic Fibrosis treatment.

Lonodelestat (previously known as POL6014), is a highly potent and selective peptide inhibitor of human neutrophil elastase (hNE), in development for the treatment of cystic fibrosis and other neutrophilic pulmonary diseases such as non-cystic fibrosis bronchiectasis (NCFB), alpha1 antitrypsin deficiency (AATD) and primary ciliary dyskinesia (PCD). Lonodelestat has ODD for the treatment of CF as well as for AATD and PCD in both EU and US.

LAU-7b (fenretinide) is a novel oral form of fenretinide, an atypical retinoid that acts on certain cell membrane lipids involved in both viral replication and immune-inflammatory responses. Results from a Phase Ib clinical trial in adult patients with CF have shown LAU-7b to have good safety and pharmacokinetic profiles, coupled with promising pharmacodynamics on specific markers of inflammation. Currently, Laurent Pharma has completed two Phase II clinical studies with LAU-7b, a first-in-class pro-resolving drug candidate, in cystic fibrosis and COVID-19.

 

Learn more about the Cystic Fibrosis Pipeline Therapies in clinical trials @ Cystic Fibrosis Market Landscape

 

Scope of the Cystic Fibrosis Market Research Report

• Coverage- 7MM
• Cystic Fibrosis Companies- Chiesi Farmaceutici S.p.A., Medidata Solutions, Bayer, Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Vertex Pharmaceuticals, Santhera Pharmaceuticals, Laurent Pharmaceuticals Inc., and others.
• Cystic Fibrosis Pipeline Therapies- Ciprofloxacin (Cipro Inhale), BAYQ3939, P-1037, Hypertonic Saline, VX-371 + HS, inhaled mannitol, and others.
• Cystic Fibrosis Market Dynamics: Cystic Fibrosis Market Drivers and Barriers
• Cystic Fibrosis Market Access and Reimbursement, Unmet Needs, and Future Perspectives

 

Discover more about Cystic Fibrosis Drugs in development @ Cystic Fibrosis Ongoing Clinical Trials Analysis

 

Table of Content

1. Key Insights

2. Cystic Fibrosis Report Introduction

3. Cystic Fibrosis Market Overview at a Glance

4. Cystic Fibrosis Market: Future Perspective

5. Cystic Fibrosis Executive Summary

6. Key Events

7. Cystic Fibrosis Disease Background and Overview

8. Cystic Fibrosis Epidemiology and Patient Population

9. Patient Journey

10. Cystic Fibrosis Marketed Drugs

11. Cystic Fibrosis Emerging Drugs

12. Cystic Fibrosis: Seven Major Market Analysis

13. Key Opinion Leaders’ View

14. Cystic Fibrosis Market Drivers

15. Cystic Fibrosis Market Barriers

16. Cystic Fibrosis SWOT Analysis

17. Cystic Fibrosis Unmet Needs

18. Reimbursement and Cystic Fibrosis Market Access

19. Appendix

20. DelveInsight Capabilities

21. Disclaimer

22. About DelveInsight

 

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