Cystic Fibrosis Pipeline constitutes 75+ key companies continuously working towards developing 80+ Cystic Fibrosis treatment therapies, analyzes DelveInsight.
Cystic Fibrosis Overview:
Cystic fibrosis (CF) is a progressive genetic disorder that leads to chronic lung infections and gradually reduces the ability to breathe. It affects over 30,000 individuals in the United States and around 70,000 globally, impacting people from all racial and ethnic backgrounds. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which results in a malfunctioning CFTR protein. This defective protein is unable to transport chloride — a component of salt — to the cell surface. Without chloride to draw water to the surface, mucus in various organs becomes thick and sticky. In the lungs, this mucus blocks airways, trapping bacteria and other germs, leading to infections, inflammation, respiratory failure, and other complications. As a result, avoiding infections is a major concern for those with CF. In men, CF can cause infertility. Symptoms of CF can vary but commonly include: skin that tastes very salty, persistent coughing (sometimes with mucus), frequent lung infections like pneumonia or bronchitis, wheezing or shortness of breath, poor growth or weight gain despite a good appetite, frequent greasy or bulky stools, difficulty with bowel movements, nasal polyps, chronic sinus infections, and more. CF is a complex condition, and the types and severity of symptoms can vary significantly between individuals.
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“Cystic Fibrosis Pipeline Insight 2025” report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Cystic Fibrosis Therapeutics Market.
Key Takeaways from the Cystic Fibrosis Pipeline Report
DelveInsight’s Cystic Fibrosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for Cystic Fibrosis treatment.
In January 2025, Verona Pharma PLC announced that the primary goal of its study is to evaluate the effect of ensifentrine versus placebo, in addition to standard care, on pulmonary exacerbations, symptoms, and quality of life in participants with non-cystic fibrosis bronchiectasis (NCFBE). The study is designed to be an event-driven trial focused on pulmonary exacerbations, with participants receiving treatment for at least 24 weeks, or until 120 subjects experience at least one protocol-defined pulmonary exacerbation.
In January 2025, Sanofi launched the ACT18018 study, a multinational, randomized, double-blind, placebo-controlled, parallel-group Phase 2 trial with three treatment groups. This study aims to assess the efficacy, safety, and tolerability of two dosing regimens of itepekimab compared to placebo in male and/or female participants with non-cystic fibrosis bronchiectasis (NCFB), ranging in age from 18 to 85 years.
In November 2024, Spirovant Sciences, a clinical-stage gene therapy company focused on inherited respiratory diseases, announced that the first patient had been treated in its SAAVe Phase 1/2 clinical trial, which is evaluating SP-101 in combination with Augmenter for cystic fibrosis. SP-101, administered via inhalation, is an investigational recombinant adeno-associated virus (AAV) gene therapy designed to target human airway epithelial cells. The chosen augmenter, doxorubicin, boosts the expression of the functional CFTR transgene to levels that are expected to provide significant clinical benefits.
In July 2024, Armata Pharmaceuticals announced the completion of full enrollment in its Tailwind Phase II clinical study, which is evaluating inhaled AP-PA02 in patients with non-cystic fibrosis bronchiectasis (NCFB) and chronic pulmonary Pseudomonas aeruginosa (P. aeruginosa) infection.
In June 2024, 4DMT announced plans to begin the Phase II dose expansion phase of its AEROW clinical trial, following positive interim results for its gene therapy, 4D-710. This therapy, which combines an AAV vector with a CFTR transgene, is being tested in cystic fibrosis (CF) patients, particularly those who are either ineligible for or unable to tolerate CFTR modulator therapies.
In May 2024, Insmed Incorporated revealed positive topline results from the ASPEN study, a global, randomized, double-blind, placebo-controlled Phase III trial designed to assess the efficacy, safety, and tolerability of brensocatib in patients with non-cystic fibrosis bronchiectasis.
In April 2024, 30 Technology announced that the European Medicines Agency (EMA) had approved a Phase I/IIa trial to evaluate the safety, tolerability, and efficacy of nebulized Nitric Oxide Formulations in non-cystic fibrosis bronchiectasis (NCFB) patients with Pseudomonas aeruginosa (Pa) or other potentially pathogenic microorganisms (PPMs) — the NOPA trial.
Key Cystic Fibrosis companies such as 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, Vertex Pharmaceuticals, PathBio Analytics, AstraZeneca, AxentisPharma AG, and others are evaluating new drugs for Cystic Fibrosis to improve the treatment landscape.
Promising Cystic Fibrosis pipeline therapies in various stages of development include OligoG, Ensifentrine, MRT5005, CB280, KB407, SPL84231, and others.
Cystic Fibrosis Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the key companies that are developing therapies in the Cystic Fibrosis Market.
The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Cystic Fibrosis treatment.
It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Cystic Fibrosis market.
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Cystic Fibrosis Emerging Drugs
OligoG: Algi Pharma
Ensifentrine: Verona Pharma
MRT5005: Translate Bio
CB280: Calithera Biosciences
KB407 : Krystal Biotech
SPL84231: SpliSense
Cystic Fibrosis Companies
Over 75 key companies are working on developing therapies for Cystic Fibrosis. Among them, Algi Pharma has its drug candidates for Cystic Fibrosis in the most advanced stage, Phase II.
DelveInsight’s report covers around 80+ products under different phases of clinical development like
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
Cystic Fibrosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Intravenous
Subcutaneous
Oral
Intramuscular
Cystic Fibrosis Products have been categorized under various Molecule types such as
Monoclonal antibody
Small molecule
Peptide
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Cystic Fibrosis Pipeline Therapeutic Assessment
• Cystic Fibrosis Assessment by Product Type
• Cystic Fibrosis By Stage
• Cystic Fibrosis Assessment by Route of Administration
• Cystic Fibrosis Assessment by Molecule Type
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Table of Content
1. Report Introduction
2. Executive Summary
3. Cystic Fibrosis Current Treatment Patterns
4. Cystic Fibrosis – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Cystic Fibrosis Late-Stage Products (Phase-III)
7. Cystic Fibrosis Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Cystic Fibrosis Discontinued Products
13. Cystic Fibrosis Product Profiles
14. Cystic Fibrosis Key Companies
15. Cystic Fibrosis Key Products
16. Dormant and Discontinued Products
17. Cystic Fibrosis Unmet Needs
18. Cystic Fibrosis Future Perspectives
19. Cystic Fibrosis Analyst Review
20. Appendix
21. Report Methodology
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