Cystic Fibrosis Market Size in the 7MM was ~USD 18,800 Million in 2023, estimated DelveInsight

Cystic Fibrosis Market

DELHI, DELHI, INDIA, July 8, 2024 /EINPresswire.com/ -- DelveInsight’s report titled “Cystic Fibrosis Therapeutics Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of burns, historical and forecasted epidemiology, as well as the burns market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. The report examines current treatment market methodologies and algorithms for cystic fibrosis, assessing the overall market potential, identifying business prospects, and addressing pertinent unmet medical requirements.

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Key Takeaways from the Cystic Fibrosis Market
• July 2024:- Vertex Pharmaceuticals Incorporated- A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age. 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.
• June 2024:- Boehringer Ingelheim- A Randomized, Double-blind, Placebo-controlled, Parallel Group Trial Evaluating Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of BI 1291583 One Tablet Once Daily Over 12 Weeks Versus Placebo in Adult Patients With Cystic Fibrosis Bronchiectasis (Clairafly™)
• As per DeveInsight analysis, in 2023, the estimated total diagnosed prevalent cases of cystic fibrosis in the 7MM were approximately 67,897 cases in 2023. This expanded genetic testing will facilitate the identification of more individuals with cystic fibrosis, contributing to the overall increase in reported cases.
• In 2023, among the 7MM, the US accounted for the highest diagnosed prevalent cases of cystic fibrosis with approximately 33,391 cases. These cases are expected to increase during the forecast period (2024-2034).
• Among the EU4 and the UK, the UK had the highest diagnosed prevalent cases of cystic fibrosis with nearly 11,275 cases, followed by France, and Germany with approximately 7,506, and 7,043 diagnosed prevalent cases respectively.
• According to DeveInsight analysis, males accounted for more diagnosed prevalent cases of cystic fibrosis than females. In 2023, there were approximately 17,363 males and 16,027 females diagnosed with prevalent cases of cystic fibrosis in the US.
• The leading Cystic Fibrosis Companies such as Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Vertex Pharmaceuticals, Santhera Pharmaceuticals, Laurent Pharmaceuticals Inc., and others
• Promising Cystic Fibrosis Therapies such as ELX/TEZ/IVA, IVA, 4D-710, BI 1291583, Cefiderocol, CMTX-101, Brensocatib 10 mg, and others.

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Cystic Fibrosis Epidemiology Segmentation in the 7MM
• Total Cystic Fibrosis diagnosed prevalent cases
• Cystic Fibrosis gender-specific diagnosed prevalent cases
• Cystic Fibrosis age-specific diagnosed prevalent cases
• Cystic Fibrosis type-specific diagnosed prevalent cases

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Cystic Fibrosis Marketed Drugs

• TRIKAFTA: Vertex Pharmaceuticals
TRIKAFTA is a combination of ivacaftor—a CFTR potentiator—tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the CFTR gene. If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of at least one F508del mutation. Elexacaftor and tezacaftor bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of F508del-CFTR to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Ivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface. Furthermore, the combined effect of elexacaftor, tezacaftor, and ivacaftor increases the quantity and function of F508del-CFTR at the cell surface, resulting in increased CFTR activity as measured by CFTR-mediated chloride transport.

• SYMDEKO: Vertex Pharmaceuticals
SYMDEKO (Symkevi) is a combination of tezacaftor and ivacaftor, indicated for the treatment of patients with cystic fibrosis ages 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the CFTR gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence. If the patient’s genotype is unknown, an FDA-cleared cystic fibrosis mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing.

• KALYDECO: Vertex Pharmaceuticals
KALYDECO (ivacaftor; VX-770) is a CFTR potentiator indicated for the treatment of cystic fibrosis in patients aged 6 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use. Ivacaftor is a potentiator of the CFTR protein. The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. Ivacaftor facilitates increased chloride transport by potentiating the channel open probability (or gating) of CFTR protein located at the cell surface. The overall level of ivacaftor-mediated CFTR chloride transport is dependent on the amount of CFTR protein at the cell surface and how responsive a particular mutant CFTR protein is to ivacaftor potentiation.

Cystic Fibrosis Emerging Drugs

• VX-121/TEZ/ VX-561: Vertex Pharmaceuticals
VX-121 is an orally administered small molecule, CFTR corrector that helps fix and restore the function of defective CFTR protein. In people with certain types of mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the CFTR protein is not processed and cannot move through the cell normally. The triple combination of vanzacaftor/tezacaftor/deutivacaftor is being developed as an investigational once-daily treatment for people with CF with certain mutations in the CFTR gene.

• Ensifentrine: Verona Pharmaceuticals
Ensifentrine (RPL554) is a first-in-class, inhaled, dual inhibitor of the phosphodiesterase 3 (PDE3) and phosphodiesterase 4 (PDE4) enzymes, and Verona Pharma’s lead pipeline asset. This dual inhibition enables it to combine bronchodilator and anti-inflammatory properties in one compound, differentiating it from existing drug classes used to treat COPD, including corticosteroids, beta2-agonists, and anti-muscarinic. The drug candidate has demonstrated positive Phase II results, ensifentrine has been shown to activate the cystic fibrosis transmembrane conductance regulator, which is beneficial in reducing mucous viscosity and improving mucociliary clearance. This suggests ensifentrine has potential as a therapy for cystic fibrosis. Additionally, ensifentrine is being investigated in non-cystic fibrosis bronchiectasis, asthma, and other respiratory diseases.

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Cystic Fibrosis Drug Market
Cystic Fibrosis treatment encompasses various pharmacological options aimed at addressing the underlying genetic defect and managing associated symptoms. The use of cystic fibrosis transmembrane conductance regulator (CFTR) protein modulators is common. Potentiators, like ivacaftor (Kalydeco), enhance the function of defective CFTR protein that has reached the cell surface, thus improving chloride ion transport. Correctors, such as lumacaftor/ivacaftor (Orkambi) and tezacaftor/ivacaftor (Symdeko/Symkevi), aid in the processing and trafficking of CFTR protein to the cell surface, thereby increasing the amount of functional CFTR protein available.

Cystic Fibrosis Market Outlook
Cystic Fibrosis treatment typically involves a multidisciplinary approach aimed at managing symptoms, preventing complications, and improving quality of life. People with cystic fibrosis have difficulty breathing and suffer from bronchospasms. Hence bronchodilators are administered to them to relax the smooth muscles of airways and aid in respiration. Albuterol which is marketed as Ventolin, is the most commonly used broncho-dilating agent that provides selective agonistic action on beta2-adrenoceptors. These treatments include airway clearance techniques, mucus-thinning medications, bronchodilators, antibiotics for respiratory infections, nutritional support, lung transplantation in severe cases, and emerging gene-based therapies such as CFTR modulators.

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Scope of the Cystic Fibrosis Market Report
• Coverage- 7MM
• Cystic Fibrosis Companies- Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Vertex Pharmaceuticals, Santhera Pharmaceuticals, Laurent Pharmaceuticals Inc., and others
• Cystic Fibrosis Therapies- ELX/TEZ/IVA, IVA, 4D-710, BI 1291583, Cefiderocol, CMTX-101, Brensocatib 10 mg, and others.
• Cystic Fibrosis Market Dynamics: Cystic Fibrosis Market drivers and Cystic Fibrosis Market Barriers
• Cystic Fibrosis Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Cystic Fibrosis Unmet Needs, KOL's views, Analyst's views, Cystic Fibrosis Market Access and Reimbursement

Uncover actionable insights into the Cystic Fibrosis Market dynamics with DelveInsight. From detailed Market Size assessments to precise Market Forecast projections, navigate the complexities of the Drugs Market and Treatment Market with confidence @ Cystic Fibrosis Treatment Market Size- https://www.delveinsight.com/sample-request/cystic-fibrosis-market?utm_source=einpresswire&utm_medium=pressrelease&utm_campaign=ypr

Table of Content
1 Key Insights
2 Report Introduction
3 Cystic Fibrosis (CF) Market Overview at a Glance
4 Methodology of Cystic Fibrosis Epidemiology and Market
5 Executive Summary of Cystic Fibrosis
6 Key Events
7 Disease Background and Overview: Cystic Fibrosis
8 Epidemiology and Patient Population
9 Patient Journey
10 Marketed Drugs
11 Emerging Drugs
12 Cystic Fibrosis: Seven Major Market Analysis
13 Key Opinion Leaders’ Views
14 SWOT Analysis
15 Unmet Needs
16 Market Access and Reimbursement
17 Appendix
18 DelveInsight Capabilities
19 Disclaimer
20 About DelveInsight

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