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Cystic Fibrosis – Global Drug Forecast and Market Analysis to 2030

The cystic fibrosis (CF) market is expected to grow from $8.2bn in 2020 to $12.7bn in 2030 in the seven major markets (7MM*) at a compound annual growth rate (CAGR) of 4.5%. GlobalData notes that significant growth in this market will be driven by the uptake of Vertex Pharmaceutical’s CF transmembrane conductance regulator (CFTR) modulators, Trikafta/Kaftrio and VX-121 + tezacaftor + VX-561 (deutivacaftor), both high-priced triple combination therapies.

This report reveals that another key factor driving an increase in the CF market is the continued growth of the patient population, as well as the improving life expectancy of CF patients.

Mandana Emamzadeh, PhD, Healthcare Analyst at GlobalData, comments: “CFTR modulators are preferred over other CF drugs, because they target the underlying cause of the disease. Vertex is the first and only company to bring these types of therapies to market and has revolutionized the space yet again with the recent launch of Trikafta/Kaftrio. With its unparalleled efficacy across a wide range of CF patients, Trikafta/Katrio is quickly becoming the gold standard.”

GlobalData expects that increasing uptake of Trikafta/Kaftrio over the forecast period will cannibalize sales from Vertex’s other three CFTR modulator products, with sales of Kalydeco, Orkambi, and Symdeko/Symkevi shrinking from $2.2bn in 2020 to $46.9m by 2030. Global sales for Trikafta/Kaftrio are projected to peak at $7.2bn in 2024 before the launch of Vertex’s next-generation triple-combination therapy, VX-121 + tezacaftor + VX-561 (deutivacaftor), in 2025. Once launched, this drug is expected to show strong uptake and achieve global sales of $8.8bn in 2030, representing nearly 70% of the total market size.

Emamzadeh continues: “Based on insight from key opinion leaders (KOLs) interviewed by GlobalData, increasing uptake of these triple-combination CFTR modulator therapies is expected to greatly improve the overall lung health of CF patients and consequently decrease the use of many symptomatic therapies including inhaled antibiotics and mucolytics. Total sales for inhaled antibiotics and mucolytic drugs will drop from 17.9% ($1.1bn) and 7.3% ($465.5m) of total sales in 2020 to 10.7% ($917.4m) and 4.9% ($424.1m) of total sales in 2030, respectively. In contrast, the prescribing rate of pancreatic enzyme replacement therapy is not expected to be greatly impacted by triple combinations during the forecast period.”

GlobalData’s report highlights five products within the late-stage pipeline for CF: four CFTR modulators, two by Vertex, VX-561 (deutivacaftor) and VX-121 + tezacaftor + VX-561, and two by AbbVie, ABBV-3067 and ABBV-3067 + galicaftor (ABBV-2222), as well as an anti-inflammatory drug, LAU-7b (fenretinide) by Laurent Pharmaceuticals.

Emamzadeh explains: “Vertex’s VX-121 + tezacaftor + VX-561 demonstrated favorable tolerability and strong efficacy compared to Trikafta/Kaftrio in a late-stage Phase II trial. KOLs believed that this therapy could actually top the stellar clinical profile of Trikafta/Kaftiro.

“In contrast, KOLs did not expect that AbbVie’s dual combination product, ABBV-3067 + galicaftor, would be superior to Trikafta/Kaftrio and believed that AbbVie’s pipeline products would more likely be positioned as alternatives for patients who cannot tolerate established CFTR modulator therapies. Finally, due to a lack of publicly available data, uncertainty remains regarding the overall clinical profile of Laurent’s LAU-7b (fenretinide), the only anti-inflammatory pipeline agent in late-stage clinical development.”

Anticipated barriers to growth in the CF market between 2020 and 2030 include slow reimbursement for novel CFTR modulators in some 5EU countries and Canada, lack of novel products in the mucolytic or inhaled antibiotic class, and decreased usage of symptomatic drug classes following widening adoption of triple combination therapies.

Emamzadeh concludes: “Although important, these barriers will have a limited impact on growth. The market is expanding at an impressive rate and ample opportunities exist for developers to further improve the CF treatment landscape.”

*7MM: US, France, Germany, Italy, Spain, UK, and Canada

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.

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?

Key Highlights

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.

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.

Companies mentioned

AbbVie

Chiesi

Genentech (Roche)

Gilead

Horizon Therapeutics

Laurent Pharmaceuticals

Nestle HealthScience

Pharmaxis

Teva

Vertex Pharmaceuticals

Viatris (formerly Mylan)

Vivus Pharmaceuticals

Table of Contents

| About GlobalData

1 Cystic Fibrosis: Executive Summary

1.1 CF Transmembrane Conductance Regulator Modulators Will Drive Substantial Growth in the Cystic Fibrosis Market from 2020-2030

1.2 Vertex’s Disease-Modifying Drugs Monopolize the CF Market

1.3 Marketed CFTR Modulators Will Provide Appropriate Treatment for the Majority of the CF Population, but the High Unmet Needs Will Remain

1.4 Opportunity for Pipeline Agents

1.5 What Do Physicians Think?

2 Introduction

2.1 Catalyst

2.2 Related Reports

2.3 Upcoming Reports

3 Disease Overview

3.1 Etiology and Pathophysiology

3.1.1 Etiology

3.1.2 Pathophysiology

3.1.3 Biomarkers/Targets of Interest

3.2 Classification

3.3 Symptoms

3.4 Prognosis

3.5 Quality of Life

4 Epidemiology

4.1 Disease Background

4.2 Risk Factors and Comorbidities

4.3 Global and Historical Trends

4.3.1 Registry-Based Diagnosed Prevalence of CF

4.3.2 Registry-Based Diagnosed Prevalent Cases Adjusted for Underestimation

4.4 Forecast Methodology

4.4.1 Sources

4.4.2 Sources Not Used

4.4.3 Forecast Assumptions and Methods

4.4.4 Registry-Based Diagnosed Prevalent Cases of CF

4.4.5 Registry-Based Diagnosed Prevalent Cases of CF by Specific Mutation

4.4.6 Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection

4.4.7 Registry-Based Diagnosed Prevalent Cases Adjusted for Underestimation

4.5 Epidemiological Forecast for CF (2020-2030)

4.5.1 Registry-Based Diagnosed Prevalent Cases of CF

4.5.2 Age-Specific Registry-Based Diagnosed Prevalent Cases of CF

4.5.3 Sex-Specific Registry-Based Diagnosed Prevalent Cases of CF

4.5.4 Registry-Based Diagnosed Prevalent Cases of CF with Specific Mutations

4.5.5 Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection

4.5.6 Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation

4.5.7 Age-Specific Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation

4.5.8 Sex-Specific Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation

4.5.9 Registry-Based Diagnosed Prevalent Cases of CF with Specific Mutations Adjusted for Underestimation

4.5.10 Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection Adjusted for Underestimation

4.6 Discussion

4.6.1 Epidemiological Forecast Insight

4.6.2 COVID-19 Impact

4.6.3 Limitations of Analysis

4.6.4 Strengths of Analysis

5 Disease Management

5.1 Diagnosis and Treatment Overview

5.1.1 Diagnosis

5.1.2 Treatment Guidelines and Leading Prescribed Drugs

5.2 KOL Insights on Disease Management

5.2.1 Earlier Diagnosis and More Treatment Options Have Led to a CF Patient Population with Milder Disease

5.2.2 Evaluation of Disease Severity

5.2.3 Treatment Approach

5.2.4 Transition from Pediatric to Adult Care

6 Competitive Assessment

6.1 Overview

7 Unmet Needs and Opportunity Assessment

7.1 Overview

7.2 Development of More Effective or Curative Therapies

7.3 Development of Chronic Lung Infection Therapies

7.4 Development of Mucolytic Therapies

7.5 Improvement of Treatment Compliance

8 R&D Strategies

8.1 Overview

8.1.1 Reformulation Strategies

8.1.2 CFTR Modulators and Disease-Modifying Therapies

8.1.3 Development of Anti-Inflammatory Drugs

8.2 Clinical Trials Design

8.2.1 Current Clinical Trials

8.2.2 CFTR Modulator Clinical Trials

9 Pipeline Assessment

9.1 Overview

9.2 Promising Drugs in Clinical Development

10 Pipeline Valuation Analysis

10.1 Overview

10.2 Competitive Assessment

10.2.1 CFTR Modulators

10.2.2 Anti-inflammatory Agents

11 Current and Future Players

11.1 Overview

11.2 Deal-Making Trends

12 Market Outlook

12.1 Global Markets

12.1.1 Forecast

12.1.2 Drivers and Barriers – Global Issues

12.2 US

12.2.1 Forecast

12.2.2 Key Events

12.3 5EU

12.3.1 Forecast

12.3.2 Key Events

12.4 Canada

12.4.1 Forecast

12.4.2 Key Events

13 Appendix

13.1 Bibliography

13.2 Abbreviations

13.3 Methodology

13.3.1 Forecasting Methodology

13.4 Primary Research – KOLs and Payers Interviewed for This Report

13.4.1 KOLs

13.4.2 Payers

13.5 Primary Research – Prescriber Survey

13.6 About the Authors

13.6.1 Analysts

13.6.2 Therapy Area Directors

13.6.3 Epidemiologist

13.6.4 Managing Epidemiologist

13.6.5 Global Director of Therapy Analysis and Epidemiology

13.6.6 Global Head and EVP of Healthcare Operations and Strategy

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List of Tables

Table 1: CF: Key Metrics in the 7MM

Table 2: Onset of CF by Approximate Age

Table 3: Classification and Criteria of the CFTR Gene Mutations in CF

Table 4: Symptoms of CF

Table 5: Risk Factors and Comorbidities for CF

Table 6: Treatment Guidelines for CF

Table 7: Leading Treatments for CF, 2021

Table 8: CF Market – Global Drivers and Barriers, 2020-2030

Table 9: Key Events Impacting Sales for CF in the US, 2020‒2030

Table 10: Key Events Impacting Sales for CF in the US, 2020‒2030

Table 11: Key Events Impacting Sales for CF in Canada, 2020-2030

Table 12: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

List of Figures

Figure 1: Global (7MM) Sales Forecast by Country for CF in 2020 and 2030

Figure 2: Analysis of the Company Portfolio Gap in CF During the Forecast Period

Figure 3: Competitive Assessment of the Late-Stage Pipeline Agents that GlobalData Expects to Be Licensed for the Treatment of CF During the Forecast Period

Figure 4: CFTR Mutation Classes in CF

Figure 5: 7MM, Registry-Based Diagnosed Prevalence of CF, Men and Women, All Ages, 2010-2030 (%)

Figure 6: 7MM, Registry-Based Diagnosed Prevalence of CF Adjusted for Underestimation, Men and Women, All Ages, 2010-2030 (%)

Figure 7: Sources Used for Registry-Based Diagnosed Prevalent Cases of CF

Figure 8: Sources Used for Registry-Based Diagnosed Prevalent Cases of CF by Specific Mutation

Figure 9: Sources Used for Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection

Figure 10: Sources Used for Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation

Figure 11: Registry-Based Diagnosed Prevalent Cases of CF, 7MM, Men and Women, All Ages, 2020

Figure 12: 7MM, Age-Specific Registry-Based Diagnosed Prevalent Cases of CF, Men and Women, 2020 (N)

Figure 13: 7MM, Sex-Specific Registry-Based Diagnosed Prevalent Cases of CF, All Ages, 2020 (N)

Figure 14: 7MM, Registry-Based Diagnosed Prevalent Cases of CF with Specific Mutations, Men and Women, All Ages, 2020 (N)

Figure 15: 7MM, Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection, Men and Women, All Ages, 2020 (N)

Figure 16: Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation, 7MM, Men and Women, All Ages, 2020

Figure 17: 7MM, Age-Specific Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation, Men and Women, 2020 (N)

Figure 18: 7MM, Sex-Specific Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation, All Ages, 2020 (N)

Figure 19: 7MM, Registry-Based Diagnosed Prevalent Cases of CF with Mutations Adjusted for Underestimation, Men and Women, All Ages, 2020 (N)

Figure 20: 7MM, Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection Adjusted for Underestimation, Men and Women, All Ages, 2020 (N)

Figure 21: Genetics of CF

Figure 22: Unmet Needs and Opportunities in CF

Figure 23: Overview of the Development Pipeline (Phase I and II) in CF

Figure 24: Key Late-Stage Trials for the Promising Pipeline Agents that GlobalData Expects to Be Licensed for CF in the 7MM During the Forecast Period

Figure 25: Competitive Assessment of Pipeline CFTR Modulators Benchmarked Against the SOCs, Kalydeco and Trikafta/Kaftrio

Figure 26: Competitive Assessment of the Pipeline Anti-inflammatory Drug, LAU-7b, Benchmarked Against the SOC, Azithromycin

Figure 27: Analysis of the Company Portfolio Gap in CF During the Forecast Period

Figure 28: Global (7MM) Sales Forecast by Country for CF in 2020 and 2030

Figure 29: Sales Forecast by Class for CF in the US in 2020 and 2030

Figure 30: Sales Forecast by Class for CF in the 5EU in 2020 and 2030

Figure 31: Sales Forecast by Class for CF in Canada in 2020 and 2030

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