Cystic Fibrosis Pipeline: Pioneering Progress with 75+ Leading Companies Developing Groundbreaking Therapies | DelveInsight

The Cystic Fibrosis market is rapidly advancing and is fueled by groundbreaking research and innovative therapies from companies such as Krystal Biotech, Vertex Pharmaceuticals, Translate Bio, Novartis, Algi Pharma, Verona Pharma, and Atlantic Healthcare. These industry pioneers are transforming treatment strategies and redefining the future of Cystic Fibrosis, bringing new hope to patients worldwide.

DelveInsight's "Cystic Fibrosis Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the Cystic Fibrosis market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.

For emerging Cystic Fibrosis drugs, the Cystic Fibrosis pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.

Key Takeaways from the Cystic Fibrosis Pipeline Report
• DelveInsight's Cystic Fibrosis Pipeline analysis depicts a robust space with 75+ active players working to develop 80+ pipeline drugs for Cystic Fibrosis treatment.
• The leading Cystic Fibrosis companies include Krystal Biotech, Vertex Pharmaceuticals, Translate Bio, Novartis, Algi Pharma, Verona Pharma, Atlantic Healthcare, Calithera Biosciences, Horizon Therapeutics, Santhera Pharmaceuticals, Reveragen Biopharma, Spli Sense, GlaxoSmithKline, EmphyCorp, Abbvie, Galapagos NV, PathBio Analytics, AstraZeneca, AxentisPharma AG, 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 Feb 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, Lupin Limited announced that it has received tentative approval from the FDA for its Abbreviated New Drug Application (ANDA) for Ivacaftor Oral Granules in 25 mg, 50 mg, and 75 mg per unit dose packet. This product is a generic version of Kalydeco Oral Granules by Vertex Pharmaceuticals.
• In January 2025, the U.S. 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 (CF) 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, the FDA approved ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) for the treatment of cystic fibrosis in patients aged 6 years and older. This approval is for individuals who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to the treatment.
• In December 2024, Vertex Pharmaceuticals Incorporated announced that the FDA has 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.

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Cystic Fibrosis Overview
Cystic fibrosis (CF) is a progressive genetic disorder that leads to persistent lung infections and gradually impairs breathing. The condition affects over 30,000 children and adults in the United States and approximately 70,000 people worldwide, impacting individuals across all racial and ethnic backgrounds. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which result in a defective CFTR protein. This protein plays a crucial role in moving chloride-a key component of salt-to the cell surface. When it malfunctions, chloride cannot attract water to the cell surface, causing mucus in various organs to become thick and sticky.

In the lungs, this buildup obstructs airways, traps harmful bacteria, and leads to infections, inflammation, respiratory failure, and other serious complications. Due to the heightened risk of infections, minimizing exposure to germs is essential for individuals with CF. Additionally, CF can affect fertility in men. Symptoms vary in severity and may include very salty-tasting skin, persistent coughing with mucus, frequent lung infections such as pneumonia or bronchitis, wheezing or shortness of breath, poor growth or weight gain despite a healthy appetite, greasy or bulky stools, digestive difficulties, nasal polyps, and chronic sinus infections. Because CF presents differently in each individual, symptom severity and complications can vary widely.

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Cystic Fibrosis Treatment Analysis: Drug Profile
OligoG: Algi Pharma
OligoG, Algi Pharma's lead drug candidate, is a dry powder formulation designed for inhalation. Derived from seaweed, this alginate oligosaccharide represents a novel class of drugs that modulate mucus and restore its normal rheology. OligoG is being developed to aid individuals with cystic fibrosis (CF) in clearing mucus from their lungs, potentially slowing disease progression. Additionally, it has demonstrated the ability to disrupt infectious biofilms commonly found in the lungs of CF patients. By breaking down these biofilms, OligoG is believed to enhance antibiotic effectiveness by increasing bacterial exposure to treatment. Currently, it is in Phase 2 clinical development for CF treatment.

Ensifentrine: Verona Pharma
Ensifentrine is a dual-action compound that combines bronchodilator and anti-inflammatory properties, making it a promising treatment for chronic obstructive pulmonary disease (COPD) and other respiratory conditions, including asthma and CF. It is designed to optimize efficacy while minimizing adverse effects by exhibiting high selectivity for PDE3 and PDE4 enzymes, thereby reducing off-target interactions. Delivered directly to the lungs via inhalation, Ensifentrine maximizes pulmonary exposure while limiting systemic distribution to reduce potential side effects. Additionally, it activates the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), which helps decrease mucus viscosity and improve mucociliary clearance-key factors in CF management. Ensifentrine is currently in Phase 2 clinical development for CF treatment.

MRT5005: Translate Bio
MRT5005 is the first clinical-stage mRNA therapy developed to address the root cause of CF. This investigational treatment delivers mRNA encoding fully functional CFTR protein directly to lung epithelial cells via nebulization. Unlike traditional CF therapies, MRT5005 is designed to benefit all CF patients, regardless of their genetic mutation, including those with minimal or no CFTR protein production. The U.S. Food and Drug Administration (FDA) has granted MRT5005 Orphan Drug, Fast Track, and Rare Pediatric Disease designations. It is currently in Phase 1/2 clinical development for CF treatment.

Key Cystic Fibrosis Therapies and Companies
• VX-121/TEZ/ VX-561: Vertex Pharmaceuticals
• Ensifentrine: Verona Pharmaceuticals
• OligoG: Algi Pharma
• MRT5005: Translate Bio
• CB280: Calithera Biosciences
• KB407 : Krystal Biotech
• SPL84231: SpliSense

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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
• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

By Molecule Type
• Oligonucleotide
• Peptide
• Small molecule

Scope of the Cystic Fibrosis Pipeline Report
• Coverage: Global
• Key Cystic Fibrosis Companies: Krystal Biotech, Vertex Pharmaceuticals, Translate Bio, Novartis, Algi Pharma, Verona Pharma, Atlantic Healthcare, Calithera Biosciences, Horizon Therapeutics, Santhera Pharmaceuticals, Reveragen Biopharma, Spli Sense, GlaxoSmithKline, EmphyCorp, Abbvie, Galapagos NV, PathBio Analytics, AstraZeneca, AxentisPharma AG, 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.

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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: Late-Stage Products (Phase III)
8. Cystic Fibrosis Pipeline: Mid-Stage Products (Phase II)
9. Cystic Fibrosis Pipeline: Early Stage Products (Phase I)
10. Therapeutic Assessment
11. Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Key Companies
14. Key Products
15. Unmet Needs
16. Market Drivers and Barriers
17. Future Perspectives and Conclusion
18. Analyst Views
19. Appendix

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