Report

Global cystic fibrosis therapeutics market is projected to witness a CAGR of 14.70% during the forecast period 2025-2032, growing from USD 13.61 billion in 2024 to USD 40.76 billion in 2032. The market's growth is supported by the increasing burden of cystic fibrosis in different regions, rising approvals from different regulatory bodies, and growing awareness about the condition among the general population. According to the estimates of the Cystic Fibrosis Foundation, about 1,000 new cases of cystic fibrosis are diagnosed each year in the United States, with over 75% of the cases being diagnosed by the time the patient is 2 years old.
Additionally, the rapid introduction of innovative therapies along with the growing number of clinical trials to expand the patient base are further providing lucrative growth opportunities for the market. Cystic fibrosis impairs the electrolyte transport system, because of which cells start retaining water and sodium, exacerbating issues related to mucus-producing glands and sweat. Thus, patients suffering from cystic fibrosis are highly susceptible to chronic coughs, lung collapses, hemoptysis, and respiratory infections, among others. Therefore, leading healthcare organizations and pharmaceutical companies are increasingly focusing on developing therapeutic solutions to combat the growing threat of the condition. The pharmaceutical companies are investing heavily in research and development activities to introduce targeted therapies, combination treatments, and gene editing technologies, among others.
Supportive healthcare policies, including orphan drug designations and reimbursement frameworks are enhancing accessibility to advanced therapies. Meanwhile, increasing awareness campaigns by nonprofit organizations coupled with the rising government initiatives are improving early diagnosis rates through expanded newborn screening programs, bolstering the market's demand.
Rising Regulatory Approvals Support Market Expansion
Increasing regulatory approvals are significantly driving the growth of the cystic fibrosis therapeutics market by facilitating the availability of innovative treatments and therapeutic solutions. In March 2025, ReCode Therapeutics, Inc. received orphan drug designation for RCT2100 from the United States Food and Drug Administration (US FDA). The investigational mRNA therapy is being developed for treating cystic fibrosis. RCT2100 is designed for delivering cystic fibrosis transmembrane conductance regulator mRNA directly to target cells in the lungs and is formulated by leveraging the company's selective organ targeting lipid nanoparticle platform.
Similarly, in February 2025, porosome reconstitution therapy for cystic fibrosis was granted orphan drug designation by the FDA. The therapy displayed increased efficacy in secretion of mucus during an analysis in which the plasma membrane of the cystic fibrosis transmembrane conductance regulator mutant cells were subjected to functional porosomes.
Additionally, the growing pipeline of clinical trials, supported by favorable regulatory environment and policies, continues to offer new opportunities for growth for the key players in the industry. Moreover, regulatory approvals often result in better reimbursement policies, boosting the accessibility of treatments more for the patient population, further bolstering the market's expansion.
Increasing Investments in Development of New Therapies Drive Market Growth
These initiatives are driving the growth of the market by fostering innovation and expanding the number of treatment options available. Investments are also accelerating research into biomarker development, personalized medicine, and gene editing technologies enabling early intervention and more effective treatments.
For instance, in January 2025, Glox Therapeutics Ltd. was awarded a grant of approximately USD 642,000 (�500,000) to advance the discovery and development of precision antibiotics that target antimicrobial-resistant lung infections in patients suffering from cystic fibrosis. Cystic fibrosis often causes thick mucus buildup in the lungs, leading to infections that become resistant to antibiotics over a period of time. Thus, addressing the urgent requirements for effective therapeutics and improving the lives of the patient population.
CFTR Modulators Account for Significant Share of the Market
The segment's growth can be attributed to the ability of these drugs to target the underlying genetic defect caused by mutations in the genes, providing a disease-modifying approach rather than addressing symptoms. This precision medicine strategy has significantly improved patient outcomes, due to improved lung functioning and increased life expectancy. Moreover, the convenience of oral administration also contributes to the popularity of CFTR modulators, allowing them to hold a significant share of the revenue.
Additionally, the rising research activities to evaluate the safety and efficacy of CFTR modulators on patients of different age groups is also supporting the segment's expansion. For instance, the La Societe Francaise de la Mucoviscidose is conducting an observational study to understand the response of CFTR modulators in patients with cystic fibrosis that are under 18. The study is estimated to conclude in July 2025.
North America Holds Major Market Share
The market's expansion in the region can be attributed to the high prevalence of cystic fibrosis in the region, increasing spendings in the healthcare sector, and strong presence of leading pharmaceutical companies. As per the estimates of the American Lung Association, approximately 40,000 people in the United States have cystic fibrosis and about one in thirty people in the country is a carrier. Moreover, research and development activities are heavily funded in North America, with major pharmaceutical corporations and research institutions collaborating to develop novel therapeutic solutions. Additionally, favorable reimbursement policies and government initiatives enhance patient access to these therapies.
Meanwhile, the Asia-Pacific is expected to witness significant growth over the forecast period due to rising investments towards the healthcare and pharmaceutical industries, improved access to innovative therapies, increasing awareness among the patient population, and growing number of research initiatives and clinical trials.
Future Market Scenario (2025 - 2032F)
The market is expected to witness significant growth in the coming years due to ongoing advancements in CFTR modulator therapies, growing awareness about the condition in developing regions, advent of various genetic editing solutions, and rising prevalence of cystic fibrosis. Additionally, increasing collaborations between biotech companies and research institutions are accelerating the development and availability of innovative solutions. Furthermore, gene therapies targeting permanent correction of cystic fibrosis transmembrane conductance regulator mutations are anticipated to enter the clinical stage over the coming years, potentially shifting the market from chronic disease management to curative approaches. Strategic partnerships, licensing deals, and public-private collaborations are also anticipated to play a crucial role in the market's expansion across the globe.
Key Players Landscape and Outlook
The key players of the market are focusing on launching new products and receiving approvals from the different regulatory bodies. For instance, in March 2025, Vertex Pharmaceuticals Incorporated's Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) for use in patients that are six or older. Alyftrek combines enhances cystic fibrosis transmembrane regulator protein function, improving salt and water transport across cell membranes. This mechanism aids in reducing thick mucus buildup, a hallmark of cystic fibrosis, and improves lung functioning, slowing the progression of the disease. Such approvals are allowing the key players of the market to increase their revenues and improve their position in the market.