Cystic Fibrosis Therapeutics Market - By Type, By Age Group, - Global Forecast, 2025 – 2034

Cystic Fibrosis Therapeutics Market Size

The global cystic fibrosis therapeutics market size was valued at USD 15.7 billion in 2024. The market is expected to grow from USD 18.1 billion in 2025 to USD 68.4 billion in 2034 at a CAGR of 15.9% from 2025 to 2034. The rising prevalence of cystic fibrosis (CF) and growing emphasis on effective treatment has driven the market growth. The high prevalence of these CF cases results in the need for early detection and improved diagnostic solutions which in turn stimulated the adoption of effective therapeutics in the market.
 

According to a study reported in the Journal of Cystic Fibrosis, nearly 65% of CF cases were diagnosed with nearly 105,352 cases across 94 countries in 2022. This growing CF incidence emphasizes the need for effective treatment to enhance the management of the condition.
 

Increased research and development funding accelerates the research activities developing advancement of potential treatment. The emergence of CF transmembrane conductance regulator (CFTR) modulator and the extending pipeline studies with expanding number of clinical trials to develop potential treatment therapy addressing the growing CF concern expanded opportunities in the market.
 

For instance, in September 2024, an investigational Phase 1b study was initiated by ReCode Therapeutics. This study aimed to evaluate tolerability and safety of RCT2100, an inhaled mRNA dosing in CF patient who are unable to respond to CFTR modulators and RCT1100 for the treatment of primary ciliary dyskinesia. This study initiation and expected positive results are likely to offer significant opportunities for a potential treatment line in CF patients. Also, an increase in awareness involving advanced therapies fuels the adoption of the therapeutics, thus greatly stimulating growth in the market.
 

Cystic fibrosis therapeutics refers to the treatment developed to address and improve the symptoms caused by CF. This genetic disorder primarily affects the digestive system, respiratory system and reproductive system. The treatment methods comprehend medication including CFTR modulators, mucolytic agents, bronchodilators, anti-infective agents, and other drugs classes while non-medication management include therapy interventions to manage the CF symptoms.
 

Cystic Fibrosis Therapeutics Market Trends

• Improvements in the CFTR modulator therapy coupled with emerging advanced therapy to address the concern of CF patients fuels the growth of the market. Development of combination therapies or other CFTR modulators such as Trikafta, Symdeko, Orkambi significantly improved the lung functioning and reduced the hospitalization of the CF patients.
   
• These therapies primarily target the causative of CF patients, managing pulmonary obliteration and enhancing the functionality of the lungs and life expectancy. For example, in December 2020 Vertex Pharmaceuticals received Food and Drug Administration (FDA) approval for TRIKAFTA in CF patients of 2 years of age and older having one F508del mutation or other responsive mutations.
   
• This approval signified treatment with access to an estimated additional 300 patients. This achievement highlighted the ongoing determination to improve care delivery for patients suffering from CF.
   
• Also, the growing advancement in CF treatment with the focus on developing potential breakthroughs offering long-term solutions for CF patients has further fueled market growth. A treatment in Western Australia on Cystic Fibrosis patients is undergoing clinical trials testing in the U.S. and Australia. The Kids Research Institute Australia and Perth Children's Hospital stated the treatment RSP-1502, combines the use of an inhaled antibiotics booster for the treatment of respiratory infections in CF patients.
   
• However, an early WA-based trial utilizing this inhaled antibiotic booster on chronically infected CF patients showcased a staggering 800-fold reduction in bacterial load and a lung function improvement of 16% on average versus the 5% improvement noted with standard treatment. This new combination is into an advancement, improving anti-infective outcomes and lung function in CF patients.
   
• Moreover, certain restraining factors such as limited accessibility of advanced therapies in developing areas, stringent regulatory requirements, potential side effects of the current therapies are impeding the market growth.
   

Cystic Fibrosis Therapeutics Market Analysis

Based on type, the global market is categorized into medication and non-medication. The medication segment valued for USD 11.9 billion 2023, estimated to generate revenue of USD 14 billion in 2024 witnessing its dominance throughout the forecast period at a CAGR of 16.1%.
 

• The medication segment is further categorized into CFTR modulators, mucolytic agents, bronchodilators, anti-infective agents, pancreatic enzyme supplements, and other drug classes.
   
• The growing advancement in the drug therapies, including CFTR modulators and other symptomatic treatments has significantly prompted the demand for medication, thereby witnessing segment prominence in the market. These drugs tend to improve the patient outcome responses by targeting the symptoms for CF and genetic mutations.
   
• The increased frequency of CF cases bolstered research and development fundings and activities enabling potential treatment availability, thereby strengthening the segment position in the market.
   
• Furthermore, advancements in personalized medicine and expanding reimbursement support emphasizing its central role in improving the health of CF patients. This is anticipated to expand market growth in the coming years.
   

Based on the age group, the global cystic fibrosis therapeutics market is categorized into Below 18 years, 18 - 40 years, and 41 and above. The below 18 years segment accounted for the highest market share of 76.8% in 2024.
 

• The segment prominence for below 18 age group was asserted to be a high occurrence of diseases such as CF in children and adolescents. Most children with CF are diagnosed by the time they're 2 years old. However, few of them with a mild form may not be diagnosed until they're a teen.
   
• Such diseases prompt early diagnosis and treatment, thereby surging the need for focused therapies and individualized care in this population.
   
• Increased attention from caregivers, better diagnostics, and access to new treatment options also play a role in the prominence of this segment. Further, the emphasis on pediatric research and regulatory approvals sustains dominance for the market throughout the forecast period.
   

In 2024, the U.S. held a prominent position in the North America cystic fibrosis therapeutics market accounting for USD 10.5 billion. This is an increase from USD 8.9 billion in 2023, demonstrating growth at a CAGR of 15.9%.
 

• The high prevalence of CF in the country elevated the need for effective CF treatment coupled with rigorous research and development activities catering to manage the disease burden has strengthened U.S. leadership in the market.
   
• According to the American Lung Association, nearly 40,000 children and adults are living with CF which is up from 38,804 prevalent cases from 2020 in U.S. Such rising cases surge the demand for effective treatment solution and subsequently boosts market growth in U.S.
   
• In addition, the country gains higher traction in the market through extensive government and private funding contributions in developing innovative therapies and advanced drugs like CFTR modulators to enhance the management of CF symptoms.
   
• Similarly, the expansion of innovative therapies like gene therapy is expected to expand treatment options.
   
• The presence of organizations such as Cystic Fibrosis Foundation and their generous reimbursement policies also contributes to the high adoption of treatments.
   
• Furthermore, the presence of top pharmaceutical companies along with significant investment in research and development continues to strengthen the position of the country in the region.
   

Germany is poised to grow at a substantial growth rate in the Europe cystic fibrosis therapeutics market.
 

• The market growth potential in the country was asserted due to the rising CF incidence along with the emerging awareness with enhanced improvement in CF management.
   
• The increased focus and efforts towards early detection through newborn screening programs offered in the country stimulates the demand for effective treatment options. For instance, every year, the NBS center Heidelberg screens newborns for about 140,000 children from Southwest Germany. Almost all parents opting for NBS for their child also consented to partake in CF-NBS (99.8%).
   
• This initiative tends to enhance the early interventions improving the patient outcomes, urging the research and development investments, and expansion of treatment accessibility in CF patients.
   
• Also, the substantial investments in research and development and collaboration between research and academic institutes and the pharmaceutical companies promote the development of innovative therapies. For example, Mukoviszidose e.V. provide fundings for CF-related research projects and clinical studies entailing a clinical application in CF care and awareness. This supportive funding aims to optimize research and development activities corresponding to the disease burden and improve patients' outcome with an emerging opportunity.
   
• Further, the supportive reimbursement policies and public aid for rare disease awareness and treatment further strengthened Germany’s position within the European market.
   

Japan held the prominent position in the Asia Pacific cystic fibrosis therapeutics market.
 

• The country's market was driven by the increasing focus on rare disease treatment coupled with favorable regulatory support like the Orphan Drug Designation system, thereby fostering innovation in CF drug development in the country
   
• The growing awareness and treatment accessibility was improved by the expansion of early diagnosis programs and the advancement of genetic screening that has further strengthened Japan's position in the Asia Pacific market.
   
• Moreover, regulatory support such as the Orphan Drug Designation system helps foster innovation in CF drug development. Therefore, these factors collectively witnessed to maintain Japan’s position expanding CF treatment availability in the region and improve market growth.
   

Brazil's cystic fibrosis therapeutics market is projected to witness growth in Latin America market.
 

• The Brazilian market is set for substantial growth due to significant CF patient population emphasizing the need for potential CF treatment with improved efficacy.
   
• For instance, according to the Brazilian cystic fibrosis Patient Registry, Brazil was having an incidence rate of about 1 in 7, 576 live births with cystic fibrosis in 2017. Also, newborn screening for CF is mandatory, aiding early diagnosis and intervention.
   
• Therefore, the rising CF cases with expanding screening access would likely spur the demand for potential disease management solutions, thereby propelling the country level market growth.
   
• Furthermore, rising awareness, advancements in healthcare infrastructure, improved access to advanced treatments and rising investments in rare disease research further sited Brazil as a key market for cystic fibrosis therapeutics in Latin America region.
   

Cystic Fibrosis Therapeutics Market Share

The top 4 players in the global market hold a share of ~50%. The key players in the market like Vertex Pharmaceuticals, F. Hoffmann-La Roche, Gilead, Novartis AG hold a substantial share of the industry. These companies are actively focusing on developing potential treatment options, targeting the disease's underlying genetic causes, addressing CF management. In addition, to address the needs, companies are actively engaging in market consolidation activities, including collaborations with research institutions and healthcare organizations to promote innovation and expedite drug discovery.
 

Furthermore, the extended pipeline for CF therapeutics including novel drugs undergoing clinical trials, highlighted the growing focus to addressing unmet needs. Also, increasing number of approvals and the growing focus on personalized medicine intensify the competition in the market. Companies are heavily investing to expand their geographic presence, particularly in emerging markets, to capitalize on growing demand and improve accessibility to cutting-edge treatments and penetration in the market.
 

Cystic Fibrosis Therapeutics Market Companies

Prominent players operating in the cystic fibrosis therapeutics industry are as mentioned below:

• Alcresta Therapeutics
• Baxter 
• Chiesi Farmaceutici S.p.A.
• F. Hoffmann-La Roche
• Gilead
• Koninklijke Philips N.V.
• Lupin
• Monaghan Medical 
• Novartis AG
• Savara
• Sionna Therapeutics
• Teva Pharmaceutical Industries
• Vertex Pharmaceuticals 
   

Vertex Pharmaceuticals is one of the key leaders in the market, with a focus on providing the CFTR modulator. The company has a range of CF therapeutics and possesses a strong pipeline with a goal to improve patient outcomes and enhance life expectancy of CF patients. For example, the company is investigating VX-522, a CFTR mRNA that can be delivered to the lung by advanced drug delivery system like lipid nanoparticles. The aim of this investigational study is to address the underlying cause of lung disease affected due to CF. The development of this drug would signify an innovative therapy alternative to CFTR modulator or the patient lacking to respond to the CFTR modulator.
 

Gilead is one of the leading providers of CF-specific antibacterial and antifungal therapies that aim to provide better infection control, improving patient safety and outcome.
 

F. Hoffmann-La Roche focuses on providing disease modifying therapies to cater to the needs of CF patients, leveraging expertise in precision medicine and diagnostics. For instance, Roche acquired the phase-1 pan-genotype CF program for Enterprise Therapeutics, providing a set of potentiator compounds TMEM16A designed to restore lung function in CF patients. This acquisition aimed to expand the company’s research capabilities, accelerating the innovation in CF industry.
 

Cystic Fibrosis Therapeutics Industry News

• In December 2024, Vertex Pharmaceuticals received the FDA granted for ALYFTREK, a once-daily triple combination CFTR modulator, for CF patients aged 6 and older with at least one F508del mutation or another responsive CFTR mutation. This approval marked a significant advancement in CF treatment options, enhancing patient care and outcomes.
   
• In October 2024, Researchers at the University of Liverpool initiated a strategy to improve lung health management in the UK by introducing a Cystic Fibrosis Innovation Hub. The hub was funded by LifeArc and Cystic Fibrosis Trust to enhance tests and treatments efficacy for lung infections in CF patients.
• In January 2024, Lupin received a U.S. FDA tentative approval to market its generic Ivacaftor oral granules for CF treatment. This approval marked a significant milestone for Lupin to penetrate in the market and offer effective therapies catering to the CF patient needs.
   

The cystic fibrosis therapeutics market research report includes in-depth coverage of the industry with estimates and forecasts in terms of revenue in USD Million from 2021 – 2034 for the following segments:

Click here to Buy Section of this Report

Market, By Type

• Medication
  • Drug class
    • CFTR modulators
    • Mucolytic agents
    • Bronchodilators
    • Anti-infective agents
    • Pancreatic enzyme supplements
    • Other drug classes
  • Route of administration     
    • Oral
    • Inhalation
  • Distribution channel
    • Hospital pharmacies
    • Retail pharmacies
    • Online pharmacies
• Non-medication

Market, By Age Group

• Below 18 years
• 18 - 40 years
• 41 and above

The above information is provided for the following regions and countries:

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • UK
  • Ireland
• Asia Pacific
  • India
  • Japan
  • Australia 
• Latin America
  • Brazil
  • Mexico
  • Argentina
• Middle East and Africa
  • South Africa
  • Saudi Arabia
  • UAE