Cystic Fibrosis Market

Cystic Fibrosis Market

LAS VEGAS, NEVADA, UNITED STATES, March 21, 2024 /EINPresswire.com/ -- DelveInsight’s ‘Cystic Fibrosis Market Insights, Epidemiology, and Market Forecast–2034’ report deliver an in-depth understanding of the Cystic Fibrosis, historical and forecasted epidemiology as well as the Cystic Fibrosis market trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

Key Takeaways from the Cystic Fibrosis Market Research Report
• The increase in Cystic Fibrosis Market Size is a direct consequence of expected launch of potential therapies, along with increasing awareness and development of novel therapies.
• 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.
• March 2024: Cystetic Medicines Inc. announced a study to Phase 1 clinical trials for ABCI. This is a 3-part, single-ascending dose Phase 1a randomized, double-blind, placebo-controlled study in healthy volunteers (Part A) and multiple-ascending dose Phase 1a randomized, double-blind, placebo-controlled study in healthy volunteers (Part B), and a Phase 1b open-label study in subjects with CF (Part C) to assess the safety, tolerability, PK, and preliminary efficacy of ABCI.
• March 2024: Vertex Pharmaceuticals Incorporated announced a study of Phase 1 & 2 clinical trials for VX-522 mRNA therapy. The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.
• 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.

Discover which therapies are expected to grab the Cystic Fibrosis market share @ Cystic Fibrosis Market Outlook

Cystic Fibrosis Overview
Cystic fibrosis (CF) is an autosomal recessive condition known as brought on by mutations in the CF 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. CF is usually diagnosed in childhood, however adults with no symptoms (or mild symptoms) during their youth can still be found to have the disease. The signs and symptoms of CF generally begin to occur around 6-8 months after birth, though this can differ significantly from person to person. Symptoms tend to differ depending on age and can affect various areas of the body.

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.

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.

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

Yash Bhardwaj
DelveInsight
+91 9650213330
email us here

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