Cystic Fibrosis Market Size, Share, Growth, Trends, and Global Industry Analysis: Drugs (CFTR Modulators, Bronchodilators, Mucolytics, Antibiotics) Route of Administration (Oral, Inhalation) Distribution Channel (Hospital Pharmacies, Retail Pharmacies) and Regional Forecast 2020-2030

Cystic Fibrosis Market was valued at US$ 23,416.6 million in 2024 and grew at 54.0 % CAGR over the historical years (2020 - 2024). The market is projected to reach US$ 30,884.5 million by 2030 at a CAGR of 4.5%. Moreover, the U.S. Global market is projected to grow significantly, reaching an estimated value of 10,259.5 million by 2030. The market focuses on the treatment of cystic fibrosis (CF), which is an inherited genetic disorder that affects the lungs, pancreas, and other organs, causing thick, sticky mucus to build up in these areas, leading to various health problems. CF is an autosomal recessive disorder, meaning both parents must carry a mutated CFTR gene for their child to inherit CF.

The market is growing significantly owing to the advancements in drug development, such as the introduction of innovative therapies. For instance, CFTR modulators have significantly improved patient outcomes and expanded treatment options. Moreover, the availability of reimbursement policies is playing a significant role in driving the growth of CFTR modulators in the market. The rising prevalence of cystic fibrosis in the United States is emerging as the leading genetic problem across the globe. For instance, according to the Cystic Fibrosis Patient Registry, it is estimated that around 40,000 children and adults are living with cystic fibrosis in the United States, and an estimated 105,000 people have been diagnosed with cystic fibrosis across 94 countries.

Facts & Figures:

• 162,428 individuals have cystic fibrosis, where North America and Europe now make up over 50% of the cystic fibrosis population.
• CF is now recognized as a global health issue with increasing prevalence in Asia, Africa, Latin America, and the Middle East.
• According to a comprehensive global analysis published in 2022, an estimated 162,428 individuals across 94 countries are currently living with cystic fibrosis, of which approximately 105,000 have been formally diagnosed. Alarmingly, an additional 57,000 individuals remain undiagnosed, underscoring the critical need for expanded diagnostic capabilities and awareness campaigns worldwide.
• In the United States, the incidence of CF is approximately 1 in 3,500 births, while Canada and Australia report similar rates of 1 in 3,300 and 1 in 3,000, respectively.
• Europe showcases considerable Regional variability, with Ireland exhibiting the highest global incidence of 1 in 1,353 births, while countries like Finland report much lower rates, around 1 in 25,000. Importantly, previously underrepresented Region such as India, where the estimated incidence ranges from 1 in 10,000 to 1 in 100,000 births.

Key Developments:

• In March 2025, the FDA granted Orphan Drug Designation to RCT2100 for treating cystic fibrosis. This inhaled mRNA therapy, targeting Class I CFTR mutations, is delivered via ReCode’s SORT LNP platform. The designation provides incentives such as market exclusivity, tax credits, fee waivers, and access to FDA grant support.
• In February 2025, The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for an expanded label of KAFTRIO®(elexacaftor/tezacaftor/ivacaftor) by Vertex Pharmaceuticals in combination with ivacaftor, aiming to benefit a broader range of CF patients.
• In December 2024, FDA Approval of TRIKAFTA Label Expansion, by Vertex Pharmaceuticals, approved for children aged 2 years and older with at least one F508del mutation or another responsive mutation; 94 new mutations added to label.
• In November 2024, Spirovant launched the SAAVe Phase 1/3 trial for SP-101, an inhaled AAV gene therapy for CF patients unserved by current modulators. Co-administered with doxorubicin, the trial focuses on safety, tolerability, lung function, and quality of life to assess the therapeutic potential in gene restoration.

Cystic Fibrosis Market Segmentation:

Based on the drugs:

• CFTR Modulators
• Bronchodilators
• Mucolytics
• Antibiotics

The cystic fibrosis (CF) market is evolving fast, with CFTR modulators taking center stage. Sales have soared from $2.76 billion in 2020 to $19.54 billion in 2024, and they’re expected to keep growing as more patients gain access and new approvals expand eligibility. While traditional symptom-based treatments are becoming less common, regions like Asia-Pacific still face challenges in accessing modulators, opening doors for market expansion. This shift is reshaping CF care, bringing hope for better treatments and improved patient outcomes worldwide.

Based on the route of administration:

• Oral
• Inhalation

The cystic fibrosis (CF) treatment landscape is evolving, with oral therapies taking over as the preferred option. CFTR modulators, like Trikafta, have made treatment more effective and convenient, boosting patient compliance and reducing dependence on inhaled drugs. As newborn screening and early diagnoses expand, more patients can access these life-changing therapies, reshaping how CF is managed worldwide. While inhaled treatments still play a supporting role, the future of CF care is shifting toward targeted, disease-modifying solutions that improve quality of life and long-term outcomes.

Based on the distribution channel:

• Hospital Pharmacy
• Retail Pharmacy

The way cystic fibrosis (CF) treatments are distributed is evolving, with retail pharmacies remaining the go-to option for most patients, though their share is slightly declining from 55.2% in 2020 to 52.6% by 2030. At the same time, hospital pharmacies are becoming more important as CF treatments grow more complex, requiring specialized care and clinical oversight. The retail pharmacy market is still expanding, expected to reach $16.26 billion by 2030, but hospitals are playing a bigger role in ensuring patients get the right treatment under proper supervision. This shift reflects how CF care is adapting to meet patients' changing needs.