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Cystic Fibrosis Market is Projected to Boost by 2034, Predicts DelveInsight | Key Companies - Verona Pharmaceuticals, Vertex Pharmaceuticals, Krystal Biotech, Aridis Pharmaceuticals, BiomX, Inc., Boehringer Ingelheim

According to DelveInsight’s analysis, the market for cystic fibrosis is anticipated to increase during the forecast period (2024–2034), owing to the launch of emerging therapies and healthcare spending.

New York, USA, July 11, 2024 (GLOBE NEWSWIRE) -- Cystic Fibrosis Market is Projected to Boost by 2034, Predicts DelveInsight | Key Companies - Verona Pharmaceuticals, Vertex Pharmaceuticals, Krystal Biotech, Aridis Pharmaceuticals, BiomX, Inc., Boehringer Ingelheim

According to DelveInsight’s analysis, the market for cystic fibrosis is anticipated to increase during the forecast period (2024–2034), owing to the launch of emerging therapies and healthcare spending.

DelveInsight’s Cystic Fibrosis Market Insights report includes a comprehensive understanding of current treatment practices, cystic fibrosis emerging drugs, market share of individual therapies, and current and forecasted cystic fibrosis market size from 2020 to 2034, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Key Takeaways from the Cystic Fibrosis Market Report

  • According to DelveInsight’s analysis, the market size of cystic fibrosis in the US was found to be around USD 7 billion in 2023.
  • According to DelveInsight’s analysis, the total diagnosed prevalent cases of cystic fibrosis in the US are expected to increase with a significant CAGR by 2034, from around 33K cases in 2023 in the US. 
  • Prominent companies working in the domain of cystic fibrosis, including Verona Pharmaceuticals, Laurent Pharmaceuticals Inc., Vertex Pharmaceuticals, SpliSense Ltd., Krystal Biotech, Inc., Aridis Pharmaceuticals, Inc., 4D Molecular Therapeutics, Sound Pharmaceuticals, Incorporated, Clarametyx Biosciences, Inc., BiomX, Inc., Boehringer Ingelheim, Respirion Pharmaceuticals Pty Ltd, Anagram Therapeutics, Inc., and others, are actively working on innovative drugs for cystic fibrosis. These novel cystic fibrosis therapies are anticipated to enter the cystic fibrosis market in the forecast period and are expected to change the market.
  • Some of the key therapies for cystic fibrosis treatment include Ensifentrine, LAU-7b, Vanzacaftor/tezacaftor/deutivacaftor, SPL84, KB407, AR-501, 4D-710, SPI-1005, CMTX-101, BX004-A, BI 1291583, RSP-1502, ANG003, and others. 

Discover which therapies are expected to grab the cystic fibrosis market share @ Cystic Fibrosis Market Report

Cystic Fibrosis Overview

Cystic fibrosis is a genetic disorder that primarily affects the respiratory and digestive systems. It is caused by mutations in the CFTR gene, which encodes a protein responsible for regulating the movement of salt and water in and out of cells. The most common mutation is ΔF508, although over 2,000 different mutations can cause the disease. These mutations lead to the production of thick, sticky mucus that can clog the airways and trap bacteria, leading to recurrent lung infections, inflammation, and respiratory failure. Additionally, the mucus obstructs the pancreatic ducts, hindering digestive enzyme secretion and impairing nutrient absorption.

Symptoms of cystic fibrosis vary but commonly include chronic cough, wheezing, frequent lung infections, sinusitis, nasal polyps, and difficulty breathing. Digestive symptoms often include poor growth, greasy and bulky stools, and abdominal pain. Cystic fibrosis can also cause male infertility and complications such as diabetes and liver disease.

Diagnosis of cystic fibrosis typically involves a combination of clinical evaluations and diagnostic tests. A sweat chloride test, which measures the concentration of chloride in sweat, is the standard diagnostic test; elevated levels indicate cystic fibrosis. Genetic testing can confirm the presence of CFTR mutations, and newborn screening programs often include tests for cystic fibrosis to enable early detection and intervention. Pulmonary function tests, chest X-rays, and stool fat analysis are also used to assess the extent of lung and digestive involvement, respectively. Early diagnosis and comprehensive care can improve the quality of life and outcomes for individuals with cystic fibrosis.


Cystic Fibrosis Epidemiology Segmentation

The cystic fibrosis epidemiology section provides insights into the historical and current cystic fibrosis patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.

The cystic fibrosis market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

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

Download the report to understand which factors are driving cystic fibrosis epidemiology trends @ Cystic Fibrosis Epidemiological Insights

Cystic Fibrosis Treatment Market 

The treatment landscape for cystic fibrosis in the United States is characterized by a complex interaction among various pharmaceutical companies, defining a wide-ranging market paradigm. Over the past ten years, this field has seen significant transformation, marked by advancements that have greatly improved both the quality of life and the median survival age of those affected. This progress is driven by a comprehensive approach to management, addressing different aspects of the condition such as pancreatic insufficiency, malnutrition, and lung disease.

To address the gastrointestinal symptoms of cystic fibrosis, treatment typically involves enzyme replacement and nutritional supplementation to alleviate pancreatic insufficiency and malnutrition. On the other hand, managing lung disease is a key focus, with strategies aimed at optimizing airway clearance and aggressively treating endobronchial bacterial infections and inflammation. The use of bronchodilators like albuterol and mucolytics such as dornase alfa is notable for relieving symptoms and improving respiratory function.

The foundation of cystic fibrosis treatment is antibiotic therapy, which is crucial for fighting the bacterial infections commonly associated with the condition. Aerosolized antibiotics are particularly important as they help prevent infections and alleviate symptoms by decreasing the number of bacteria in the airways. Furthermore, improvements in diagnostic techniques and the use of palliative care have significantly extended the median life expectancy, highlighting the profound effect of these advancing treatment methods.

The significant market for cystic fibrosis in the US is driven by several approved CFTR modulators, including KALYDECO (ivacaftor), ORKAMBI (lumacaftor/ivacaftor), SYMDEKO (tezacaftor/ivacaftor and ivacaftor), and TRIKAFTA (elexacaftor, tezacaftor, ivacaftor). Additionally, the US FDA has approved PULMOZYME (dornase alfa), BRONCHITOL, and TOBI PODHALER for treating cystic fibrosis patients.

Learn more about the FDA-approved drugs for cystic fibrosis @ Drugs for Cystic Fibrosis Treatment 

Cystic Fibrosis Emerging Drugs and Companies

Some of the drugs in the pipeline include Ensifentrine (Verona Pharmaceuticals), LAU-7b (Laurent Pharmaceuticals Inc.), vanzacaftor/tezacaftor/deutivacaftor (Vertex Pharmaceuticals), and others.

Ensifentrine is a pioneering inhaled medication that simultaneously inhibits the phosphodiesterase 3 (PDE3) and phosphodiesterase 4 (PDE4) enzymes. It activates the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), which helps decrease mucus thickness and enhance mucociliary clearance, making it a promising treatment option for cystic fibrosis. Currently, the drug is in Phase II of clinical trials for this condition.

LAU-7b (fenretinide) is an innovative oral version of fenretinide, a unique type of retinoid that influences specific lipids in cell membranes, crucial for viral replication and immune-inflammatory reactions. Current evidence suggests that LAU-7b offers a therapeutic strategy that complements CFTR modulators. Its pro-resolving properties can potentially rectify the imbalance between DHA and AA, managing inflammation without disrupting natural defense mechanisms. Additionally, LAU-7b has been observed to restore certain long-chain sphingolipids (ceramides) related to CFTR protein regulation, particularly during inflammatory conditions. The leading drug candidate of the company, LAU-7b, is a distinct oral formulation of fenretinide protected by patents, ideally suited for a once-daily low-dose treatment approach.

The other therapies in the pipeline for cystic fibrosis treatment include

  • SPL84: SpliSense Ltd.
  • KB407: Krystal Biotech, Inc.
  • AR-501: Aridis Pharmaceuticals, Inc.
  • 4D-710: 4D Molecular Therapeutics
  • SPI-1005: Sound Pharmaceuticals, Incorporated
  • CMTX-101: Clarametyx Biosciences, Inc.
  • BX004-A: BiomX, Inc.
  • BI 1291583: Boehringer Ingelheim
  • RSP-1502: Respirion Pharmaceuticals Pty Ltd
  • ANG003: Anagram Therapeutics, Inc.

The anticipated launch of these emerging therapies are poised to transform the cystic fibrosis market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the cystic fibrosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.

To know more about cystic fibrosis clinical trials, visit @ Cystic Fibrosis Treatment Drugs 

Cystic Fibrosis Market Dynamics

The cystic fibrosis market dynamics are anticipated to change in the coming years. The cystic fibrosis market is driven by several key factors, including advancements in genetic research, the development of novel therapies, and the increasing prevalence of the disease. Enhanced diagnostic techniques have led to earlier and more accurate detection, improving patient outcomes and expanding the market. Additionally, significant investments by pharmaceutical companies in the development of innovative treatments, such as CFTR modulators, have revolutionized the management of cystic fibrosis, offering improved quality of life and extended life expectancy for patients. 

Government support and funding for cystic fibrosis research, along with rising awareness about the disease, further contribute to market growth. The increasing number of clinical trials and a robust pipeline of promising drugs continue to fuel the cystic fibrosis market's expansion, offering hope for better therapeutic options shortly.

Furthermore, many potential therapies are being investigated for the treatment of cystic fibrosis, and it is safe to predict that the treatment space will significantly impact the cystic fibrosis market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the cystic fibrosis market in the 7MM.

However, several factors may impede the growth of the cystic fibrosis market. High costs of advanced therapies, such as CFTR modulators, limit accessibility for many patients, particularly in low- and middle-income countries. Additionally, the need for early and accurate genetic screening and diagnosis, which is not universally available, poses another challenge. Regulatory hurdles and the extensive clinical trial processes for new treatments also delay the introduction of innovative therapies. 

Furthermore, the chronic nature of cystic fibrosis necessitates lifelong treatment, adding to the financial burden on healthcare systems and patients. Addressing these barriers requires coordinated efforts to improve diagnostic infrastructure, reduce costs, and expedite regulatory approvals.

Moreover, cystic fibrosis treatment poses a significant economic burden and disrupts patients’ overall well-being and QOL. Furthermore, the cystic fibrosis market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the cystic fibrosis market growth.

Cystic Fibrosis Report Metrics Details
Study Period 2020–2034
Cystic Fibrosis Report Coverage 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]
Cystic Fibrosis Market Size in 2023 USD 7 Billion (United States)
Key Cystic Fibrosis Companies Verona Pharmaceuticals, Laurent Pharmaceuticals Inc., Vertex Pharmaceuticals, SpliSense Ltd., Krystal Biotech, Inc., Aridis Pharmaceuticals, Inc., 4D Molecular Therapeutics, Sound Pharmaceuticals, Incorporated, Clarametyx Biosciences, Inc., BiomX, Inc., Boehringer Ingelheim, Respirion Pharmaceuticals Pty Ltd, Anagram Therapeutics, Inc., and others
Key Cystic Fibrosis Therapies Ensifentrine, LAU-7b, Vanzacaftor/tezacaftor/deutivacaftor, SPL84, KB407, AR-501, 4D-710, SPI-1005, CMTX-101, BX004-A, BI 1291583, RSP-1502, ANG003, and others

Scope of the Cystic Fibrosis Market Report

  • Cystic Fibrosis Therapeutic Assessment: Cystic Fibrosis current marketed and emerging therapies
  • Cystic Fibrosis Market Dynamics: Attribute Analysis of Emerging Cystic Fibrosis Drugs
  • Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
  • Unmet Needs, KOL’s views, Analyst’s views, Cystic Fibrosis Market Access and Reimbursement

Discover more about cystic fibrosis drugs in development @ Cystic Fibrosis Clinical Trials

Table of Contents

1. Cystic Fibrosis Market Key Insights
2. Cystic Fibrosis Market Report Introduction
3. Cystic Fibrosis Market Overview at a Glance
4. Cystic Fibrosis Market Executive Summary
5. Disease Background and Overview
6. Cystic Fibrosis Treatment and Management
7. Cystic Fibrosis Epidemiology and Patient Population
8. Patient Journey
9. Cystic Fibrosis Marketed Drugs
10. Cystic Fibrosis Emerging Drugs
11. Seven Major Cystic Fibrosis Market Analysis
12. Cystic Fibrosis Market Outlook
13. Potential of Current and Emerging Therapies
14. KOL Views
15. Unmet Needs
16. SWOT Analysis
17. Appendix
18. DelveInsight Capabilities
19. Disclaimer
20. About DelveInsight

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.

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