Cystic Fibrosis Pipeline Insight, 2025: Emerging Therapies Aim to Expand Treatment Access and Address Underlying Genetic Causes | DelveInsight

Cystic fibrosis (CF) is a rare, progressive genetic disorder that primarily affects the respiratory and digestive systems, leading to persistent lung infections and reduced life expectancy. Despite significant advances in CF care over the past decade-particularly with the advent of CFTR modulators-there remains a pressing need to develop more inclusive therapies for the broader CF population, including those with rare or unaddressed mutations.

As of 2025, the cystic fibrosis treatment pipeline is rapidly evolving, with over 75 active candidates in clinical and preclinical development. The pipeline reflects a robust diversification of therapeutic strategies, ranging from next-generation CFTR modulators and gene therapies to anti-inflammatories, mRNA-based treatments, and inhaled antibiotics. Biopharma leaders such as Ligand Pharmaceuticals, Boehringer Ingelheim, OrPro Therapeutics, Protalix Biotherapeutics, Laurent Pharmaceuticals, Arcturus Therapeutics, Enterprise Therapeutics, Affinia Therapeutics, and AlgiPharma AS are at the forefront of this innovation wave, working to broaden treatment reach and improve long-term disease management.

DelveInsight's "Cystic Fibrosis - Pipeline Insight, 2025" offers a comprehensive review of these pipeline candidates, tracking key clinical milestones, regulatory updates, and company strategies. The report emphasizes novel approaches aimed at modifying disease progression, reducing treatment burden, and addressing unmet needs in pediatric and underserved patient subgroups.

With gene therapy and personalized medicine gaining traction, the CF treatment paradigm is on the brink of a new era. The expanding pipeline holds the promise of more effective, durable solutions that not only manage symptoms but potentially offer curative options, marking a significant leap forward in the fight against cystic fibrosis.

Interested in learning more about the current treatment landscape and the key drivers shaping the cystic fibrosis pipeline? Click here: https://www.delveinsight.com/report-store/cystic-fibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Cystic Fibrosis Pipeline Report
• DelveInsight's cystic fibrosis pipeline analysis depicts a strong space with 75+ active players working to develop 80+ pipeline drugs for cystic fibrosis treatment.
• The leading cystic fibrosis companies include Eloxx Pharmaceuticals, Arrowhead Pharmaceuticals, SolAeroMed, Parion Sciences, Translate Bio, Inc., Path BioAnalytics, Aridis Pharmaceuticals, Novartis, Vertex Pharmaceuticals, AlgiPharma, Proteostasis Therapeutics, Inc., Galapagos NV, Santhera Pharmaceuticals, Calithera Biosciences, Inc., Spyryx Biosciences, Inc., Verona Pharma, Ionis Pharmaceuticals, Inc., Chiesi Farmaceutici S.p.A., Ligand Pharmaceuticals, Boehringer Ingelheim, OrPro Therapeutics, Protalix Biotherapeutics, Laurent Pharmaceuticals, Arcturus Therapeutics, Enterprise Therapeutics, Affinia Therapeutics, AlgiPharma AS, Sound Pharmaceuticals, Spirovant, 4D Molecular Therapeutics, Santhera Pharmaceuticals, Armata Pharmaceuticals, and others are evaluating their lead assets to improve the cystic fibrosis treatment landscape.
• Key cystic fibrosis pipeline therapies in various stages of development include KB407, MRT5005, P1037, Cavosonstat, QBW276, VX121, OligoG, Ensifentrine, Renzapride, CB280, SPL84231, Bacteriophage therapeutics, Sodium pyruvate, GLPG 3067, GLPG2737, Brensocatib, Ravicti, Tobramycin, and others.
• In March 2025, ReCode Therapeutics announced that the FDA granted Orphan Drug Designation for RCT2100, an investigational mRNA therapy developed to treat cystic fibrosis.
• In February 2025, the FDA granted orphan drug designation to Porosome Therapeutics' new treatment strategy for cystic fibrosis, based on the porosome, a type of cellular structure.
• In February 2025, Insmed Incorporated announced that the FDA informed the company it does not plan to hold an advisory committee meeting to discuss the New Drug Application (NDA) for brensocatib for patients with non-cystic fibrosis bronchiectasis.
• In February 2025, Porosome Therapeutics announced that the FDA granted Orphan Drug Designation to its revolutionary cystic fibrosis therapy, marking a significant step in secretory defect therapeutics.
• In January 2025, the FDA approved a new triple-combination CFTR modulator therapy-vanzacaftor, tezacaftor, and deutivacaftor. Developed by Vertex Pharmaceuticals and marketed as Alyftrek, the therapy is indicated for cystic fibrosis in patients aged 6 and older with at least one copy of a mutation responsive to the treatment, including the F508del mutation.
• In December 2024, Inogen received FDA 510(k) clearance for its SIMEOX 200 Airway Clearance Device, expanding its respiratory product portfolio in the U.S. The upgraded version of the original Simeox uses high-frequency oscillatory vibrations and intermittent negative pressure during exhalation to improve bronchial drainage in patients with chronic lung diseases like Bronchiectasis, COPD, Cystic Fibrosis, and Primary Ciliary Dyskinesia.
• In December 2024, Vertex Pharmaceuticals Incorporated announced that the FDA approved the expanded use of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in individuals aged 2 and older. The approval applies to those with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or a mutation responsive to TRIKAFTA based on clinical and/or in vitro data.

Request a sample and discover the recent breakthroughs happening in the cystic fibrosis pipeline landscape at https://www.delveinsight.com/report-store/cystic-fibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Cystic Fibrosis Overview
Cystic fibrosis (CF) is a rare, inherited, life-shortening disorder that primarily affects the lungs and digestive system. It is caused by mutations in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene, which leads to the production of thick, sticky mucus that clogs airways and traps bacteria, resulting in chronic lung infections, inflammation, and progressive respiratory decline. The disease also affects the pancreas, impairing digestion and nutrient absorption.

CF affects approximately 70,000-100,000 people worldwide, with the highest prevalence seen in North America and Europe. It is typically diagnosed in infancy through newborn screening programs or early signs such as persistent coughing, frequent lung infections, and poor weight gain. Although historically considered a pediatric disease, advances in treatment have significantly improved life expectancy, enabling many individuals to live into adulthood.

Treatment has evolved significantly over the years-from symptomatic relief with airway clearance techniques and antibiotics to the introduction of CFTR modulators that target the underlying genetic defect. Despite this progress, not all patients benefit from existing therapies, especially those with rare CFTR mutations. As a result, there is an ongoing push for more inclusive and transformative therapies, including gene therapy, mRNA-based treatments, and anti-inflammatory agents, aimed at improving quality of life and potentially offering a functional cure.

Find out more about cystic fibrosis medication at https://www.delveinsight.com/report-store/cystic-fibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Cystic Fibrosis Treatment Analysis: Drug Profile
VX-121: Vertex Pharmaceuticals
Vertex is advancing a triple combination therapy-VX-121, tezacaftor, and VX-561 (deutivacaftor)-as a once-daily treatment for cystic fibrosis patients with specific CFTR mutations. VX-121 and tezacaftor work to correct protein processing and trafficking defects by increasing mature CFTR protein presence at the cell surface, while VX-561, a CFTR potentiator, helps keep the protein channel open longer to enhance chloride transport and mucus clearance. This investigational regimen is currently in Phase III clinical trials.

Brensocatib: AstraZeneca/Insmed
Brensocatib is an oral DPP1 inhibitor under development for inflammatory lung diseases such as cystic fibrosis. DPP1 activates neutrophil serine proteases (NSPs), which in excess contribute to airway inflammation and tissue damage. By blocking DPP1, brensocatib may reduce NSP activity and related lung damage. It is currently being studied in a Phase II clinical trial to assess its pharmacokinetics in CF patients.

ELX-02: Eloxx Pharmaceuticals
ELX-02 is an investigational read-through therapy targeting cystic fibrosis caused by nonsense mutations, particularly G542X. It is an aminoglycoside analog designed to enable the ribosome to bypass premature stop codons, thereby producing functional full-length CFTR proteins. The drug has received orphan drug and fast track designations in the U.S. and Europe and is being evaluated in Phase II open-label studies in CF patients carrying at least one G542X mutation.

Learn more about the novel and emerging cystic fibrosis pipeline therapies at https://www.delveinsight.com/report-store/cystic-fibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Cystic Fibrosis Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical

By Molecule Type
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Scope of the Cystic Fibrosis Pipeline Report
• Coverage: Global
• Key Cystic Fibrosis Companies: Eloxx Pharmaceuticals, Arrowhead Pharmaceuticals, SolAeroMed, Parion Sciences, Translate Bio, Inc., Path BioAnalytics, Aridis Pharmaceuticals, Novartis, Vertex Pharmaceuticals, AlgiPharma, Proteostasis Therapeutics, Inc., Galapagos NV, Santhera Pharmaceuticals, Calithera Biosciences, Inc., Spyryx Biosciences, Inc., Verona Pharma, Ionis Pharmaceuticals, Inc., Chiesi Farmaceutici S.p.A., Ligand Pharmaceuticals, Boehringer Ingelheim, OrPro Therapeutics, Protalix Biotherapeutics, Laurent Pharmaceuticals, Arcturus Therapeutics, Enterprise Therapeutics, Affinia Therapeutics, AlgiPharma AS, Sound Pharmaceuticals, Spirovant, 4D Molecular Therapeutics, Santhera Pharmaceuticals, Armata Pharmaceuticals, and others.
• Key Cystic Fibrosis Pipeline Therapies: KB407, MRT5005, P1037, Cavosonstat, QBW276, VX121, OligoG, Ensifentrine, Renzapride, CB280, SPL84231, Bacteriophage therapeutics, Sodium pyruvate, GLPG 3067, GLPG2737, Brensocatib, Ravicti, Tobramycin, and others.

Dive deep into rich insights for drugs used for cystic fibrosis treatment, visit: https://www.delveinsight.com/report-store/cystic-fibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Cystic Fibrosis Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Cystic Fibrosis Pipeline Therapeutics
6. Cystic Fibrosis Pipeline: Late-Stage Products (Phase III)
7. Cystic Fibrosis Pipeline: Mid-Stage Products (Phase II)
8. Cystic Fibrosis Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

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This release was published on openPR.