Ultra Market Research | United States Cystic Fibrosis Market
United States Cystic Fibrosis Market
Report ID : 987
Category : United-States(US)
No Of Pages : 90
Published on: February 2025
Status: Published
Format :
Key Question Answer
Global Market Outlook
In-depth analysis of global and regional trends
Analyze and identify the major players in the market, their market share, key developments, etc.
To understand the capability of the major players based on products offered, financials, and strategies.
Identify disrupting products, companies, and trends.
To identify opportunities in the market.
Analyze the regional penetration of players, products, and services in the market.
Comparison of major players financial performance.
Evaluate strategies adopted by major players.
Recommendations
United States Cystic Fibrosis Market
Introduction
Cystic fibrosis (CF) is a genetic disease affecting a patient's respiration, digestion, and reproductive systems, causing severe lung infection and other complications in the United States, the CF market includes some therapeutic approaches such as CFTR modulators, mucolytics, and antibiotics to handle and treat the disease. Recent trends have shown a huge uptake of CFTR modulators, mainly because of the development of drugs and personalized medicines of 2023. The U.S. cystic fibrosis market was valued at around USD 7 billion in 2023, and it is expected to grow further with ongoing research and development.
Segmentation
By Therapy Type
CFTR Modulators
Potentiators
Ivacaftor
Others
Correctors
Lumacaftor
Tezacaftor
Elexacaftor
Others
Amplifiers
Nesolicaftor
Others
Others
Mucolytics
Inhaled Mucolytics
Dornase Alfa
Hypertonic Saline
Others
Oral Mucolytics
N-acetylcysteine
Others
Antibiotics
Inhaled Antibiotics
Tobramycin
Aztreonam
Others
Oral Antibiotics
Ciprofloxacin
Others
Intravenous Antibiotics
Ceftazidime
Meropenem
Others
Others
By Route of Administration
Oral
Tablets
Capsules
Others
Inhalation
Nebulizers
Dry Powder Inhalers
Metered Dose Inhalers
Others
Intravenous
Infusions
Injections
Others
Others
By Age Group
Pediatric
0-5 Years
6-12 Years
13-17 Years
Others
Adult
18-30 Years
31-50 Years
Above 50 Years
Others
By Distribution Channel
Hospital Pharmacies
Retail Pharmacies
Online Pharmacies
Others
List of Market Players
Vertex Pharmaceuticals (USA)
AbbVie Inc. (USA)
Gilead Sciences, Inc. (USA)
Novartis AG (Switzerland)
F. Hoffmann-La Roche AG (Switzerland)
Pfizer Inc. (USA)
GlaxoSmithKline plc (UK)
Sanofi S.A. (France)
Teva Pharmaceutical Industries Ltd. (Israel)
Mylan N.V. (USA)
Chiesi Farmaceutici S.p.A. (Italy)
Pharmaxis Ltd. (Australia)
Alcresta Therapeutics, Inc. (USA)
Proteostasis Therapeutics, Inc. (USA)
Galapagos NV (Belgium)
Drivers
The United States cystic fibrosis market is primarily driven by the increasing prevalence of CF and the subsequent rise in demand for effective treatments. Advancements in genetic research have led to the development of CFTR modulators, which target the underlying cause of the disease, thereby improving patient outcomes. Additionally, heightened awareness and early diagnosis have contributed to a larger patient pool receiving timely interventions. Supportive government programs and funding that support CF research also enhance growth in the marketplace, allowing newer therapies to reach the market. The focus towards personalized medicine-tailored by individual genetic signatures-has also fostered the expansion of novel drugs in the treatment of CF market.
Restraints
Despite the positive growth trajectory, the cystic fibrosis market faces several challenges. The high cost of CFTR modulators and other advanced therapies is a significant barrier to access, especially for uninsured or underinsured patients. Furthermore, the complexity of CF as a multi-system disorder requires comprehensive treatment regimens that can be burdensome for patients and healthcare providers alike. Limited awareness in some populations will result in delayed diagnosis, reducing the effectiveness of treatments available. In addition, adverse effects related to long-term use of CF medications may hinder patient adherence and, therefore, impact overall treatment efficacy.
Opportunities
The cystic fibrosis market is evolving, and there are several opportunities for growth. Research into gene therapies is ongoing and promises curative treatments that could change the standard of care for CF patients. Expansion into underserved regions and populations can increase market reach and improve patient outcomes. Collaborations between pharmaceutical companies and research institutions can expedite the development of innovative therapies. In addition, advances in diagnostic technologies are now allowing for early detection of CF, thus making it timely to intervene and managing the disease effectively. Conjoining digital health solutions, such as telemedicine and remote monitoring, provides opportunities for improved care for patients and enhanced adherence to treatment protocols.
Trends
Recent trends in the United States cystic fibrosis market include combination therapies that focus on the various aspects of the disease simultaneously. The recent approvals of triple-combination CFTR modulators, such as Vertex Pharmaceuticals' Trikafta, set a new standard for treatment efficacy. There is also an increased focus on the use of personalized treatment plans based on genetic profiling to optimize the therapeutic outcome. The use of real-world evidence and patient registries is increasingly gaining importance in treatment effects over long-term periods as well as informing clinical decision-making. In this regard, more patient advocacy groups are being integrated into the financing of research studies and policy decisions, thereby guiding the course of future therapies. The market also sees an investment in inhaled antibiotic formulations aimed at enhancing delivery and minimizing systemic side effects of drugs.
As of 2023, the market is valued at approximately USD 7 billion, with strong growth potential due to advancements in CF treatments.
Major drivers include the rising prevalence of CF, advancements in CFTR modulators, increased research funding, and growing adoption of personalized medicine.
Leading companies include Vertex Pharmaceuticals, AbbVie, Gilead Sciences, Novartis, Pfizer, and GlaxoSmithKline, among others.
High treatment costs, limited accessibility to advanced therapies, potential side effects, and the complexity of disease management are significant restraints.
The market is expected to grow due to continued research, the development of gene therapies, improved diagnostic techniques, and increased government support for CF treatments.
Table of Contents
Introduction 1.1 Report Description 1.2 Research Methodology 1.2.1 Data Mining 1.2.2 Market Modeling and Forecasting 1.2.3 Data Validation 1.2.4 Industry Analysis
Market Dashboard
Market Overview 3.1 Market Definition and Scope 3.2 Market Segmentation 3.2.1 By Treatment Types 3.2.2 By Patient Demographics 3.2.3 By Distribution Channels 3.2.4 By Therapeutic Applications 3.3 Market Dynamics 3.3.1 Drivers 3.3.2 Restraints 3.3.3 Opportunities 3.3.4 Challenges
United States Cystic Fibrosis Market by Treatment Types 4.1 Introduction 4.2 Market Size and Growth Rate by Treatment Types (2024-2030) 4.2.1 Mucolytics 4.2.2 Bronchodilators 4.2.3 CFTR Modulators 4.2.4 Antibiotics 4.2.5 Pancreatic Enzyme Replacements 4.2.6 Anti-Inflammatory Drugs 4.2.7 Others
United States Cystic Fibrosis Market by Patient Demographics 5.1 Introduction 5.2 Market Size and Growth Rate by Patient Demographics (2024-2030) 5.2.1 Children 5.2.1.1 Infants 5.2.1.2 Toddlers and Young Children 5.2.2 Adults 5.2.2.1 Young Adults 5.2.2.2 Seniors
United States Cystic Fibrosis Market by Distribution Channels 6.1 Introduction 6.2 Market Size and Growth Rate by Distribution Channels (2024-2030) 6.2.1 Hospital Pharmacies 6.2.1.1 Tertiary Care Centers 6.2.1.2 Cystic Fibrosis Specialty Clinics 6.2.2 Retail Pharmacies 6.2.2.1 Local Pharmacies 6.2.2.2 Chain Pharmacies 6.2.3 Online Pharmacies
United States Cystic Fibrosis Market by Therapeutic Applications 7.1 Introduction 7.2 Market Size and Growth Rate by Therapeutic Applications (2024-2030) 7.2.1 Maintenance Therapy 7.2.2 Acute Exacerbation Management 7.2.3 Emerging Experimental Therapies
Competitive Landscape 8.1 Company Profiles 8.1.1 Amgen Inc. (United States) * Business Overview * Product Portfolio * Strategic Developments * Financial Overview 8.1.2 Novartis AG (Switzerland) * Business Overview * Product Portfolio * Strategic Developments * Financial Overview 8.1.3 Pfizer Inc. (United States) * Business Overview * Product Portfolio * Strategic Developments * Financial Overview 8.1.4 Vertex Pharmaceuticals (United States) * Business Overview * Product Portfolio * Strategic Developments * Financial Overview 8.1.5 Gilead Sciences (United States) * Business Overview * Product Portfolio * Strategic Developments * Financial Overview 8.1.6 AbbVie Inc. (United States) * Business Overview * Product Portfolio * Strategic Developments * Financial Overview 8.1.7 Sanofi (France) * Business Overview * Product Portfolio * Strategic Developments * Financial Overview 8.1.8 Eli Lilly and Company (United States) * Business Overview * Product Portfolio * Strategic Developments * Financial Overview
Conclusion and Recommendations 10.1 List of Tables
Table 1: United States Cystic Fibrosis Market, by Treatment Types (2024-2030)
Table 2: United States Cystic Fibrosis Market, by Patient Demographics (2024-2030)
Table 3: United States Cystic Fibrosis Market, by Distribution Channels (2024-2030)
Table 4: United States Cystic Fibrosis Market, by Therapeutic Applications (2024-2030)
Table 5: Competitive Landscape Analysis
10.2 List of Figures * Figure 1: Market Segmentation Overview * Figure 2: Market Dynamics – Drivers, Restraints, Opportunities, and Challenges * Figure 3: Growth Trends in Cystic Fibrosis Treatments * Figure 4: Competitive Landscape – Key Players Market Share * Figure 5: Regional Analysis of Cystic Fibrosis Market in the United States
Research Methodology
The process of market research at Ultra Market Research is an iterative in application and usually follows following path. Information from secondary used to build data models, then results from data models are validated from primary participants. Then cycle repeats where, according to inputs from primary participants, additional secondary research is done, and new Information is again incorporated into data model. The process continues till desired level of Information is not generated
To calculate the market size, the report considers the revenue generated from the sales of Ultra Market Research providers. The revenue generated from the sales of Ultra Market Research has been calculated through primary and secondary research. The report also presents the key players operating in the Ultra Market Research market across the globe identified through secondary research and a corresponding detailed analysis of the top vendors in the market. The market size calculation also includes distribution channel segmentation determined using secondary sources and verified through primary sources.
Secondary Research
The secondary research source that are typically referred to include, but are not limited to:
Company websites, annual reports, financial reports, broker reports, investor presentations and SEC filings
Internal and external proprietary databases, relevant patent and regulatory databases
National government documents, statistical databases and market reports
News articles, press releases and web-casts specific to the companies operating in the market
The source for secondary research includes but is not limited to: Factiva, Hoovers and Statista
Inner Circle Represents – Stage of Research Process
Middle Circle Represents – Source of Information
Outer Circle Represents – Information Derived from that source
Primary Research We conduct primary interviews on an ongoing basis with industry participants and commentators in order to validate data and analysis. A typical research interview fulfills the following functions:
It provides first-hand Information on the market size, market trends, growth trends, competitive landscape, future outlook etc.
Helps in validating and strengthening the secondary research findings
Further develops the analysis team’s expertise and market understanding
Primary research involves E-mail interactions, telephonic interviews as well as face-to-face interviews for each market, category, segment and sub-segment across geographies
The participants who typically take part in such a process include, but are not limited to:
Industry participants: CEOs, VPs, marketing/ distribution channel managers, market intelligence managers and national sales managers
Purchasing managers, technical personnel, distributors and resellers
Outside experts: Investment bankers, valuation experts, research analysts specializing in specific markets
Key opinion leaders specializing in different areas corresponding to different industry distribution channels
Models Where no hard data is available, we use modeling and estimates in order to produce comprehensive data sets. A rigorous methodology is adopted in which the available hard data is cross referenced with the following data distribution channels to produce estimates:
Demographic data: Population split by segment
Macro-economic indicators: GDP, etc.
Industry indicators: Expenditure, distribution channel stage & infrastructure, sector growth and facilities.
Data is then cross checked by the expert panel.
2..1 Company Share Analysis Model
Company share analysis is used to derive the size of United Kingdom market. As well as study of revenues of companies for last three to five years also provide the base for forecasting the market size and its growth rate. This model is built in following steps: 2..2 Revenue Based Modeling
Revenue based models can be built in two ways – Top-Down or Bottom-Up irrespective of industry. Market size estimated from company share analysis acts as a validation point for bottom-up approach where as it acts as starting point for top-down approach.
2.1 Research Limitations Inflation is not a part of pricing in this report. Prices of United States Cystic Fibrosis Market and its derivatives vary in each region and hence similar revenue ratio does not follow for each individual region. The same price for each distribution channel has been taken into account while estimating and forecasting market revenue Globally. Regional average price has been considered while breaking down this market by application in each region. This report provides market size of United States Cystic Fibrosis Market for the past year and forecasts for the next six years. United States Cystic Fibrosis Market size is given in terms of revenue. Market revenue is defined in USD Million. Market numbers are given on the basis of different United States Cystic Fibrosis Market categories. Market size and forecasts for each major application is provided in the context of Global market. The numbers provided in this report are derived on the basis of demand for United States Cystic Fibrosis Market from different application industries in different regions.
