United States Cystic Fibrosis Market NEWS 2025: FDA Approval of VX-548 by Vertex Pharmaceuticals Expands Treatment Eligibility for Cystic Fibrosis Patients with Rare Mutations

The Global Cystic Fibrosis Market reached USD 10.96 billion in 2023 and is expected to reach USD 26.58 billion by 2031, growing at a CAGR of 11.8% during the forecast period 2024-2031.

The cystic fibrosis market is centered on therapies, diagnostics, and devices that manage this genetic disorder affecting the lungs and digestive system. Treatments include gene modulators, mucus-thinning drugs, and antibiotics to control infections. Diagnostic tools support early detection and personalized treatment plans. Increasing prevalence and awareness of cystic fibrosis drive market demand. Research into novel therapies and improved patient care continues to expand the market.

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☛ Cystic Fibrosis Market Recent Developments 2025:

United States: Recent Industry Developments

✅ In August 2025, Vertex Pharmaceuticals announced FDA approval for VX-548, a next-generation CFTR modulator for cystic fibrosis patients with rare mutations. The therapy is expected to expand treatment eligibility to thousands of patients previously lacking options.

✅ In July 2025, Proteostasis Therapeutics initiated Phase 3 trials for PTI-428, targeting improved lung function in cystic fibrosis patients with advanced disease stages. Early Phase 2 data demonstrated significant improvements in FEV1 and reduced pulmonary exacerbations.

✅ In June 2025, Moderna began development of an mRNA-based therapy for cystic fibrosis, aiming to correct CFTR gene expression. Preclinical results show potential for long-term restoration of lung function, representing a novel approach in CF management.

Europe: Recent Industry Developments

✅ In July 2025, AstraZeneca and CF Foundation Therapeutics launched a multi-center European study on triple combination CFTR modulators, focusing on children aged 6-11. The study aims to assess safety and efficacy in younger populations.

✅ In June 2025, GlycoMira Therapeutics received EMA orphan drug designation for GM-201, a nasal spray therapy designed to reduce chronic airway inflammation in CF patients. Regulatory support accelerates potential market access across the EU.

✅ In May 2025, Vertex Pharmaceuticals expanded its European access program, providing VX-770/tezacaftor/elexacaftor therapy to underserved cystic fibrosis populations. The initiative includes patient support, education, and reimbursement assistance.

Japan: Recent Industry Developments

✅ In July 2025, Takeda Pharmaceutical initiated clinical trials for a next-generation CFTR modulator targeting Japanese cystic fibrosis patients with rare mutations. The study focuses on safety and efficacy in adolescent and adult populations.

✅ In June 2025, Chugai Pharmaceutical partnered with Vertex Pharmaceuticals to expand access to triple combination CFTR therapy in Japan. The collaboration aims to accelerate patient enrollment in post-marketing programs.

✅ In May 2025, Astellas Pharma launched a patient support initiative providing comprehensive care programs for cystic fibrosis, including home therapy monitoring and genetic counseling, addressing gaps in rare disease management.

☛ Cystic Fibrosis Market Trends:

Targeted therapies such as CFTR modulators dominate the market trend. Gene therapy and mRNA-based treatments are emerging for more permanent solutions. Personalized treatment plans based on genetic profiling are increasing patient outcomes. Digital health tools are being adopted for monitoring lung function and adherence. Rising awareness and screening programs in newborns are contributing to early diagnosis.

Recent Pipelines:

New-generation CFTR modulators are under late-stage trials. mRNA therapies aiming to correct CFTR protein expression are being evaluated. Inhaled antibiotics and anti-inflammatory agents are in clinical studies. Gene editing technologies like CRISPR-Cas9 are in preclinical development. Adjunct therapies for mucus clearance and lung function improvement are progressing in trials.

☛ Cystic Fibrosis Market Competitve Landscape:

Vertex Pharmaceuticals Inc., AbbVie Inc, F. Hoffmann-La Roche Ltd, Novartis AG, Teva Pharmaceutical Industries Ltd, Gilead sciences, Genentech Inc, AstraZeneca, Alcresta Therapeutics Inc., Merck & Co. Inc.

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We follow a hybrid research approach, combining qualitative insights with rigorous quantitative analysis to deliver reliable and comprehensive market intelligence. Our process begins with extensive secondary research, drawing on trusted industry reports, proprietary databases, and credible market sources. This is then reinforced through targeted primary research, including structured surveys and in-depth interviews with industry leaders, subject matter experts, and key market participants.

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☛ Segment Covered in the Cystic Fibrosis Market:

By Therapeutic drug class: CFTR modulators, Pancreatic enzyme supplements, Mucolytics, Bronchodilators.

By Route of administration: Oral, Inhaled, Parenteral.

By End-user: Hospitals, Clinics, Cancer centers, Others.

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☛ Regional Analysis for Cystic Fibrosis Market:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

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