DUBLIN–(BUSINESS WIRE)–The “United States Cystic Fibrosis Therapeutics Market to See Strong Growth Through 2033 – Driven by Gene Therapy and CFTR Innovation” report has been added to ResearchAndMarkets.com’s offering.
The United States Cystic Fibrosis Therapeutics Market is expected to reach US$ 2.32 billion in 2024 and further reach US$ 4.72 billion by the end of 2033 at a compound annual growth rate of 8.22% during the forecast period of 2025-2033. The growth is triggered by innovation in gene therapy, expanded patient access to CFTR modulators, enhanced awareness, and robust research efforts.
In the United States, therapeutic growth for cystic fibrosis has been strong due to huge investment in research and development, well-established healthcare infrastructure, and early embracement of new therapies. The United States has a large population of diagnosed patients and organizations such as the Cystic Fibrosis Foundation, which aggressively encourage awareness, clinical trials, and patient support initiatives. As there are ongoing developments and greater exposure to precision medicine, popularity and performance of cystic fibrosis drugs continue to expand nationwide.
Growth Drivers of the United States Cystic Fibrosis Therapeutics Market
CFTR Modulator Therapies Advances
Approval and development of CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulators have revolutionized the treatment of cystic fibrosis. These medications, like Trikafta (elexacaftor/tezacaftor/ivacaftor), attack the faulty protein that causes the disease at its root. They have dramatically enhanced life expectancy and quality of life. More FDA approvals, investments by major pharmaceutical companies, and continuous clinical trials for wider patient use are driving growth.
As an increasing number of patients benefit from these high-impact treatments, the market continues to grow. December 2024, The FDA approved Vertex Pharmaceuticals’ Alyftrek for cystic fibrosis patients aged 6 and older with at least one F508del mutation or another treatment-responsive CFTR gene mutation. This approval expands treatment options and offers a more convenient dosing regimen.
Strong Support from Advocacy Organizations
Nonprofit organizations such as the Cystic Fibrosis Foundation are key drivers of awareness, research funding, and drug development acceleration. These organizations provide patient access to clinical trials and enable government and private sector partnerships. Their actions not only enhanced patient outcomes but also brought new entrants and investors into the market. This ecosystem fosters innovation, accelerates regulatory processes, and maintains long-term U.S. market growth.
High Prevalence and Early Diagnosis
One of the highest prevalence rates of cystic fibrosis is found in the United States, with newborn screening programs available in all 50 states, guaranteeing early diagnosis. Early diagnosis supports early therapeutic intervention, enhancing outcome and driving demand for effective therapies. Improved genetic testing, public health infrastructure, and increasing awareness have driven the increase in diagnosed cases, extending the therapeutic market. This wide patient base still sustains a strong demand for new and current treatments. There are roughly 40,000 individuals with cystic fibrosis in the United States and roughly 100,000 globally. Roughly 1 in 30 individuals in the United States is a carrier.
Challenges in United States Cystic Fibrosis Therapeutics Market
High Cost of Treatment
Cystic fibrosis treatments, especially CFTR modulators, are very costly – typically more than $300,000 per patient per year. This creates a tremendous economic burden for health systems, payers, and patients, reducing access. Even with insurance coverage and support programs, cost continues to be a deterrent for most. The pricing pressure also attracts regulatory attention and criticism, with the potential to impact market growth and innovation.
Limited Access in Rural Areas
While urban centers often have specialized CF care facilities, rural areas in the U.S. may lack access to expert care and advanced therapies. Transportation barriers, specialist shortages, and limited health education contribute to care disparities. Patients in these regions often experience delayed diagnosis or suboptimal treatment. Bridging this gap requires investment in telemedicine, outreach programs, and rural healthcare infrastructure – challenges that remain persistent.
Cystic Fibrosis Therapeutics Products Launches in USA
- May 2024: Arcturus Therapeutics Holdings Inc. said it will report Phase 1 results in healthy volunteers and interim Phase 1b data in CF patients for its inhaled mRNA therapeutic ARCT-032.
- Sept. 2024: 4D Molecular Therapeutics will present at the 2024 North American Cystic Fibrosis Conference in Boston.
Key Players Analysis (Overviews, Key Persons, Recent Developments, SWOT Analysis, Revenue Analysis)
- Vertex Pharmaceuticals
- Abbvie Inc.
- Novartis AG
- Gilead Sciences
- Viatris INC
- Pfizer
- AstraZeneca
- F. Hoffmann-La Roche Ltd
Top 10 States
- California
- Texas
- New York
- Florida
- Illinois
- Pennsylvania
- Ohio
- Georgia
- Washington
- New Jersey
- Rest of United States
Key Attributes:
| Report Attribute | Details |
| No. of Pages | 200 |
| Forecast Period | 2024 – 2033 |
| Estimated Market Value (USD) in 2024 | $2.32 Billion |
| Forecasted Market Value (USD) by 2033 | $4.72 Billion |
| Compound Annual Growth Rate | 8.2% |
| Regions Covered | United States |
Key Topics Covered:
1. Introduction
2. Research & Methodology
2.1 Data Source
2.2 Research Approach
2.3 Forecast Projection Methodology
3. Executive Summary
4. Market Dynamics
4.1 Growth Drivers
4.2 Challenges
5. United States Cystic Fibrosis Therapeutics Market
5.1 Historical Market Trends
5.2 Market Forecast
6. Market Share Analysis
6.1 By Drugs Type
6.2 By Route of Administration
6.3 By Distribution Channel
6.4 By States
7. Drug Class
7.1 Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)
7.2 Bronchodilators
7.3 Mucolytic
7.4 Pancreatic Enzyme Supplement
7.5 Others
8. Route of Administration
8.1 Oral Drugs
8.2 Inhaled Drugs
9. Distribution Channel
9.1 Hospitals
9.2 Retail Pharmacies
9.3 Others
10. Top 10 States
10.1 California
10.2 Texas
10.3 New York
10.4 Florida
10.5 Illinois
10.6 Pennsylvania
10.7 Ohio
10.8 Georgia
10.9 Washington
10.10 New Jersey
10.11 Rest of United States
11. Value Chain Analysis
12. Porter’s Five Forces Analysis
12.1 Bargaining Power of Buyers
12.2 Bargaining Power of Suppliers
12.3 Degree of Competition
12.4 Threat of New Entrants
12.5 Threat of Substitutes
13. SWOT Analysis
13.1 Strength
13.2 Weakness
13.3 Opportunity
13.4 Threats
14. Cystic Fibrosis Drugs Insights
14.1 Marketed Drugs
14.1.1 TRIKAFTA: Vertex Pharmaceuticals
14.1.1.1 Product Description
14.1.1.2 Clinical Trial & Development
14.1.1.3 Safety and Efficacy
14.1.2 SYMDEKO: Vertex Pharmaceuticals
14.1.3 ORKAMBI: Vertex Pharmaceuticals
14.1.4 KALYDECO: Vertex Pharmaceuticals
14.2 Emerging Drugs
14.2.1 Ensifentrine: Verona Pharmaceuticals
14.2.2 ELX-02: Eloxx Pharmaceuticals
14.2.3 VX-121/TEZ/VX-561: Vertex Pharmaceuticals
14.2.4 POL6014 (Lonodelestat): Santhera Pharmaceuticals
14.2.5 LAU-7b: Laurent Pharmaceuticals
15. Pricing Benchmark Analysis
15.1 Vertex Pharmaceuticals
15.2 Abbvie Inc.
15.3 Novartis AG
15.4 Gilead Sciences
15.5 Viatris Inc
15.6 Pfizer
15.7 AstraZeneca
15.8 F. Hoffmann-La Roche Ltd
16. Key Players Analysis
For more information about this report visit https://www.researchandmarkets.com/r/psisx7
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