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August 2024
The global cystic fibrosis therapeutics market size is calculated at USD 10.34 billion in 2024, grew to USD 11.81 billion in 2025, and is projected to reach around USD 39.15 billion by 2034. The market is expanding at a CAGR of 14.24% between 2025 and 2034. The rising incidences of cystic fibrosis, growing research and development, and technological advancements drive the market.
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Cystic fibrosis is a condition that damages the lungs, digestive system, and other organs of the body. The goals of cystic fibrosis treatment include: preventing and controlling infections in the lungs, removing and loosening mucus from the lungs, treating and preventing intestinal blockage, and getting enough nutrition. However, there is no cure for cystic fibrosis, but therapeutics can ease symptoms and reduce complications. The different treatment regimens available include medicines, airway-clearing techniques, and surgery. Antibiotics, anti-inflammatory drugs, bronchodilators, mucus thinners, and CFTR modulators are commonly prescribed to patients. Apart from these, additional support is given to patients with severe conditions such as oxygen therapy, pulmonary rehabilitation, ventilator support, and extracorporeal membrane oxygenation (ECMO).
The rising incidences and prevalence of cystic fibrosis and its complications necessitate its effective treatment. Approximately 100,000 people are estimated to have cystic fibrosis globally. The latest advancements in diagnostics for the early detection of cystic fibrosis allow healthcare professionals to make clinical decisions and prescribe potential therapeutics. The growing research and development activities lead to an increasing number of clinical trials for developing novel treatment regimens, boosting the market. Additionally, technological advancements drive the latest innovations in cystic fibrosis treatment.
Artificial intelligence (AI) and machine learning (ML) algorithms revolutionize the therapeutic regimen of cystic fibrosis. AI and ML helps in diagnosing the disease by analyzing cystic fibrosis mutations in individual patients. AI-driven diagnosis enables healthcare professionals to provide advanced treatment care to patients. AI and ML can also be used to develop novel, personalized treatment regimens for cystic fibrosis. They also suggest effective treatment plans to patients based on their genetic profile, enhancing efficiency and reducing manual errors. Additionally, they can predict treatment outcomes by analyzing how patients will react to already available medications. AI and ML can also predict the pharmacokinetic and pharmacodynamic profile of novel drugs. They can also detect the toxicity of these drugs before administering to patients, enabling development of drugs with reduced side effects.
Increasing Number of Clinical Trials
The major growth factor of the cystic fibrosis therapeutics market is the increasing number of clinical trials. This is a result of growing research and development activities. Since there is no cure for the disease, scientists are investigating novel molecules to treat the root cause of the disease. Novel molecules are designed with the aim to improve additional important clinical improvements such as body mass index, reported well-being, and pulmonary exacerbation rates. Major pharmaceutical companies are developing cystic fibrosis drugs to expand their drug pipeline which helps them to strengthen their market position. Several regulatory agencies necessitate preclinical and clinical trial data which require companies and institutions to conduct clinical trials for their novel therapeutics. There are a total of 1,647 clinical studies reported on clinicaltrials.gov related to cystic fibrosis.
High Cost
The major challenge of the market is the high treatment cost for cystic fibrosis. The average treatment cost for cystic fibrosis range from $451 to $160,000 per person annually. This limits the affordability of several low- and middle-income groups to afford the treatment, restricting market growth.
Personalized Treatment
The development of personalized treatment for cystic fibrosis patients presents future cystic fibrosis therapeutics market growth opportunities. Personalized treatments are developed based on individual’s genetic profile. The advent of genetic testing enable the association of specific mutations to disease severity, leading to the development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Additionally, identifying emerging biomarkers such as inflammatory markers and pulmonary function indicators, aid in early detection and monitoring progression. These allow healthcare professionals to provide customized treatment to their patients. The increasing investments and favorable government policies support the development of personalized treatment, augmenting market growth. The Personalized Medicine Coalition organization works with the U.S. government and the Centers for Medicare and Medicaid Services to advance strategies documenting the value of personalized medicine, promising better outcomes and more efficient health systems.
By drug class, the CFTR modulators segment held a dominant presence in the cystic fibrosis therapeutics market in 2024. CFTR modulators improve production, intracellular processing, and function of the defective CFTR protein. The different CFTR modulators used are triple combinations elexacaftor-tezacaftor-ivacaftor, ivacaftor monotherapy, and dual combinations tezacaftor-ivacaftor and lumacaftor-ivacaftor. These are recently approved cystic fibrosis treatment drugs that treats the root cause of the disease. Hence, they are widely prescribed as other drugs can only relieve symptoms. The recent advancements in genomics and combination therapies as well as favorable regulatory frameworks augment the segment’s growth.
By drug class, the pancreatic enzyme supplements segment is anticipated to grow with the highest CAGR in the market during the studied years. Pancreatic enzyme supplements are essential for almost all patients with cystic fibrosis (80-90%) to prevent malnutrition. They are generally available in tablets or capsules and are administered before every meal. This allows patients to digest nutrients to keep their body healthy. The rising incidences of cystic fibrosis and significant benefits of supplements to improve health boost the segment’s growth.
By route of administration, the oral segment held the largest share of the cystic fibrosis therapeutics market in 2024. The oral drugs are highly preferred due to their cost-effectiveness and ease of administration. They eliminate the need for trained professionals to administer medications. Oral drugs enable sustained release to delay drug absorption at a desired time. They also increase patient medication adherence and reduce manufacturing and transportation cost.
By route of administration, the inhaled segment is projected to expand rapidly in the market in the coming years. Inhaled route enables patients for targeted release directly into the lungs and immediate effect due to targeted release. Bronchodilators or corticosteroids are usually administered through inhalation using metered dose inhalers. The latest innovations in drug delivery systems and growing research and development to invent novel therapeutics fuel the segment’s growth. Technological advancements favor the development of novel devices.
By distribution channel, the hospital pharmacies segment led the global cystic fibrosis therapeutics market in 2024. The segmental growth is attributed to favorable infrastructure, suitable capital investments, and the presence of trained professionals. Hospitals are part of several clinical trials allowing patients to take benefit of novel treatment regimens before their marketed approval. The favorable reimbursement policies also potentiate the segment’s growth. The rising severity of disease require surgery, increasing the demand for hospital pharmacies.
By distribution channel, the online pharmacies segment is expected to grow at the fastest rate in the market during the forecast period. The rising adoption of smartphones and the burgeoning e-commerce sector propel the segment’s growth. Online pharmacies offer 24x7 facilities and same-day home delivery which potentiates their demand. Some online pharmacies also offer free online consultation with experts, attracting more customers. They refill the monthly medications, eliminating the need to order medications every month.
North America held the major share of the cystic fibrosis therapeutics market in 2024. The rising incidences of cystic fibrosis, technological advancements, and favorable regulatory frameworks drive the market. According to the Cystic Fibrosis Foundation organization, approximately 40,000 people are living with cystic fibrosis. The increasing number of clinical trials also contribute to market. North America conducts the highest number of clinical trials related to cystic fibrosis globally.
The presence of key players potentiate the market in North America. AbbVie, Gilead Sciences, and ReCode Therapeutics are likely to dominate the market. Furthermore, the state-of-the-art research and development facilities lead to latest innovations in the treatment of cystic fibrosis.
Asia-Pacific is anticipated to grow at the fastest rate in the market during the forecast period. The growing research and development activities, increasing investments, and favorable government policies drive the market. The Japanese government’s Ministry of Health announced to add cystic fibrosis drugs to the list of reimbursable medicines. In Asia-Pacific, Australia and New Zealand are the countries with the highest number of cystic fibrosis cases. The increasing collaboration and mergers & acquisitions among companies, institutions, and healthcare professionals hold promise for further progress in cystic fibrosis treatment and care. The rising number of startups in the region, owing to the rapidly expanding biotechnology and pharmaceutical sectors. Moreover, the Australian government announced an investment of $5.5 million for newborn screening platform to screen hundreds of life-threatening genetic disorders.
Shehnaz Suliman, CEO of ReCode Therapeutics, commented on the receipt of $15 million funding from Cystic Fibrosis Foundation that the funding will support gene correction approach to accelerate research in collaboration with Intellia and to advance the development of treatment for cystic fibrosis. He added that the company aims to build a therapeutic franchise, expanding disease-modifying treatment options for patients.
By Drug Class
By Route of Administration
By Distribution Channel
By Region
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