Cystic fibrosis (CF) is an autosomal recessive disease characterized by pancreatic insufficiency and chronic endobronchial airway infection. The disease is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. The most common mutation is the deletion of phenylalanine at codon 508 (known as F508del). Recent progress in the understanding of the disease, increased newborn screening, and discovery of disease-modifying drugs have revolutionized the treatment approach and improved the quality of life and life expectancy of CF patients.
Marketed drugs currently available for CF target multiple aspects of the disease. Inhaled antibiotics are used for the treatment of chronic lung infections, mucolytic agents are used for reducing the viscosity of the mucus in the lungs, pancreatic enzyme replacement therapies (PERTs) are used for the treatment of CF-related Exocrine pancreatic insufficiency (EPI), and CFTR modulators enhance CFTR function, targeting the underlying cause of disease. The disease-modifying nature of CFTR modulators has transformed the treatment paradigm for CF over the last decade, a trend that is likely to persist.
Over the past decade, there have been significant changes in the CF market due to the development of CFTR modulator therapies that target the underlying cause of CF. The most recently launched CFTR modulator, Vertex Therapeutics’ Trikafta/Kaftrio, is the first triple drug combination treatment and quickly became the gold standard soon after approval. However, the publisher is expecting the emergence of a next-generation triple-combination CFTR modulator, Vertex’s VX-121 + tezacaftor + VX-561 (deutivacaftor), to take over the market by the end of the forecast period.
The publisher projects that the global CF market, which comprises the seven major pharmaceutical markets (7MM) (US, France, Germany, Italy, Spain, UK, and Canada), will experience growth during the forecast period, at a Compound Annual Growth Rate (CAGR) of 4.5%, with sales growing from $8.2B in 2020 to $12.7B in 2030.
Marketed drugs currently available for CF target multiple aspects of the disease. Inhaled antibiotics are used for the treatment of chronic lung infections, mucolytic agents are used for reducing the viscosity of the mucus in the lungs, pancreatic enzyme replacement therapies (PERTs) are used for the treatment of CF-related Exocrine pancreatic insufficiency (EPI), and CFTR modulators enhance CFTR function, targeting the underlying cause of disease. The disease-modifying nature of CFTR modulators has transformed the treatment paradigm for CF over the last decade, a trend that is likely to persist.
Over the past decade, there have been significant changes in the CF market due to the development of CFTR modulator therapies that target the underlying cause of CF. The most recently launched CFTR modulator, Vertex Therapeutics’ Trikafta/Kaftrio, is the first triple drug combination treatment and quickly became the gold standard soon after approval. However, the publisher is expecting the emergence of a next-generation triple-combination CFTR modulator, Vertex’s VX-121 + tezacaftor + VX-561 (deutivacaftor), to take over the market by the end of the forecast period.
The publisher projects that the global CF market, which comprises the seven major pharmaceutical markets (7MM) (US, France, Germany, Italy, Spain, UK, and Canada), will experience growth during the forecast period, at a Compound Annual Growth Rate (CAGR) of 4.5%, with sales growing from $8.2B in 2020 to $12.7B in 2030.
The greatest drivers of growth in the global CF market include:
- The continued uptake of high-priced Trikafta/Kaftrio and label expansion to a growing population of CF patients
- The launch of VX-121 + tezacaftor + VX-561 (deutivacaftor) in 2025 in the US and in 2026 in the 5EU, a high-priced next-generation triple combination.
- Continued growth of the CF patient population due to overall population growth across the 7MM and increasing life expectancy of CF patients.
The main barrier to growth in the CF market include:
- Slow reimbursement for novel CFTR modulators in some 5EU countries and Canada.
- Decreased usage of symptomatic drug classes such antibiotics, mucolytics, and anti-inflammatory drugs due to overall improvements to lung health following use of Trikafta/Kaftrio.
- Lack of novel products entering the CF market in the mucolytic or inhaled drug class.
- Key unmet needs include the lack of curative therapies, the need for better antibiotic regimens to fight lung infections, limited choice in mucolytic products, and low patient adherence to treatment.
Key Questions Answered
- Which unmet needs are limiting the treatment of CF in the 7MM?
- What strategies can the pharmaceutical industry employ to increase treatment rates for CF? How should these strategies differ across different geographical markets?
- What effect will the launch of generics have on the sales of branded agents?
- What are the main R&D trends in the CF market and which companies are leading the way?
- Are there major differences in the mechanisms of action used by therapies in late-stage versus early-stage clinical development?
Scope
- Overview of CF including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
- Topline CF market revenue, annual cost of therapy, and major pipeline product sales in the forecast period.
- Key topics covered include current treatment and pipeline therapies, unmet needs and opportunities, and the drivers and barriers affecting CF therapeutics sales in the 7MM.
- Pipeline analysis: Comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
- Analysis of the current and future market competition in the global CF therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.
Reasons to Buy
The report will enable you to:
- Develop and design your in-licensing and out-licensing strategies, using a detailed overview of current pipeline products and technologies to identify companies with the most robust pipelines.
- Develop business strategies by understanding the trends shaping and driving the global CF therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global CF market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Track drug sales in the global CF therapeutics market from 2020-2030.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
Table of Contents
1 Cystic Fibrosis: Executive Summary
2 Introduction
3 Disease Overview
4 Epidemiology
5 Disease Management
6 Competitive Assessment
7 Unmet Needs and Opportunity Assessment
8 R&D Strategies
9 Pipeline Assessment
10 Pipeline Valuation Analysis
11 Current and Future Players
12 Market Outlook
List of Tables
List of Figures
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- AbbVie
- Chiesi
- Genentech (Roche)
- Gilead
- Horizon Therapeutics
- Laurent Pharmaceuticals
- Nestle HealthScience
- Pharmaxis
- Teva
- Vertex Pharmaceuticals
- Viatris (formerly Mylan)
- Vivus Pharmaceuticals
