Cystic Fibrosis (CF) is a rare genetic disorder caused by a mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene located on chromosome 7. This disorder primarily affects the lungs and digestive system and causes secretions to become thick and sticky. Individuals with Cystic Fibrosis develop lung disease resulting from a cycle of mucus retention, infection, and inflammation, as well as pancreatic dysfunction resulting in calorie malabsorption. The sweat glands and reproductive organs are also affected. The sequence of the CFTR gene was identified in 1989 and it encodes a 1480 amino acid protein. There are approximately 250 known disease-causing mutations that intervene with various stages of CFTR synthesis and function. Every year, approximately 70,000 children and young adults are affected with CF across the world.
The Cystic Fibrosis market is primarily driven by increasing CF prevalence, the rise in R&D expenditure by pharmaceutical companies and increase in funding by government bodies. The rise in the prevalence of Cystic Fibrosis is further attributed to reduced mortality rate due to treatment advancements and better disease management. Drugs such as Orkambi, Kalydeco, Symdeko etc. are expected to boost the market growth, while factors such as the high cost of treatment, and limited availability of medicine for all mutation type’s patients are expected to hamper market growth.
Key players in the Cystic Fibrosis market are Vertex Pharmaceuticals, Roche, Allergan, Novartis, AbbVie, Pharmaxis, Chiesi Farmaceutici, PARI Medical Holding and Gilead Sciences. Vertex Pharmaceuticals is the leading player in the market. The global Cystic Fibrosis market is expected to witness substantial growth during the forecast period. The market is anticipated to reach a value of $12.2 billion by 2028 from the estimated value of $3.8 billion in 2018, at a CAGR of 12.3 percent. This can be attributed to the great investment by market players and many non-profit organizations in the research and development of Cystic Fibrosis therapy. Another factor is the growing penetration of drugs including CFTR modulators during the forecast period and the existence of a strong pipeline.
The Cystic Fibrosis pipeline is comprised of diverse sets of molecules with a majority of products in early-stage development. In total, 124 molecules are in development either alone or in combination with other molecules. The majority of these molecules are in the pre-clinical development stage followed by Phase II and Phase I. Earlier, CF treatment was largely focused on the downstream effects of CFTR dysfunction (mucus retention, infection, and inflammation of the airways) and there have been many advancements in the treatment of those problems. However, new therapies such as CFTR modulators are able to address the underlying abnormalities of Cystic Fibrosis rather than its downstream effects.
The efficacy of these treatments has been established recently. Currently, there are just three FDA-approved molecules targeting specific mutation types that are commercially available in the market.
The majority of companies are focused on the modulators of the CFTR gene, followed by anti-infectives. The Cystic Fibrosis drug market is very competitive and many small players are researching and developing products in this field. Vertex Pharmaceuticals, Galapagos, Flatley Discovery Lab, Ockham Biotech, Proteostasis Therapeutics and Druggability Technologies Holdings are leading the research space.
Key Questions Answered and insights delivered in the Report:
- Global Cystic Fibrosis market size by value from 2016 to 2028.
- Global Cystic Fibrosis market size by product sales; 2016 and 2017.
- Global Cystic Fibrosis Market by Therapeutic Approach; 2017.
- Comprehensive product landscape by therapeutic class, company, originator, approval date, pharma class, molecule type, mutation type and therapy type.
- Pipeline analysis by stage of development, therapeutic class, molecule type, therapy type, route of administration and company.
- Merger & acquisition and collaboration by the key players in the market.
- Growth drivers, future opportunities and challenges in the market.
- Earliest priority country filing trend in Cystic Fibrosis market; 2007-2018.
- Cystic Fibrosis patent trend by focus area, route of administration and composition; 2007-2018.
- Cystic Fibrosis patent trend by key patent holder; 2007-2018.
- Key patents in Cystic Fibrosis market; 2007-2018.
- Top inventors in Cystic Fibrosis market; 2007-2018.
- Detailed company profiling of key market players.
Executive Summary
1. Cystic Fibrosis: Disease Overview
I. Clinical Manifestations and Complications of Cystic Fibrosis
II. Etiology and Genetics of Cystic Fibrosis
a. Basics of Genetics and the CFTR Gene
b. Role of CFTR Protein in Ion Transport
c. The CFTR Gene Mutations
d. Classification of Mutations of CFTR Gene
III. Diagnosis of Cystic Fibrosis
IV. Treatment Methodology of Cystic Fibrosis
a. Treatment of Lung Complications
b. Treatment for Advanced Lung Disease
c. Treatment for Digestive Problems
2. Market Sizing: Growth and Forecast
I. Global Cystic Fibrosis Market by Value; 2016-2028
II. Cystic Fibrosis Market by Product Sales
III. Cystic Fibrosis Market by Therapeutic Approach
a. CFTR Modulator
b. Mucociliary Clearance
c. Anti-Infective
d. Pancreatic Enzyme Supplement
3. Product Landscape
I. CFTR Modulator
II. Mucociliary Clearance
III. Anti-Infective
IV. Pancreatic Enzyme Supplement
4. Pipeline Analysis of Cystic Fibrosis
I. Cystic Fibrosis Drug Pipeline by Stage of Development
II. Cystic Fibrosis Drug Pipeline by Therapeutic Class
a. Anti-Infective
b. Anti-Inflammatory
c. CFTR Modulator
d. Mucociliary Clearance
e. Nutritional
f. Miscellaneous
III. Cystic Fibrosis Drug Pipeline by Molecule Type
IV. Cystic Fibrosis Drug Pipeline by Route of Administration
V. Cystic Fibrosis Drug Pipeline by Therapy Type
VI. Cystic Fibrosis Drug Pipeline by Company
5. Merger & Acquisition Activities
6. Major Collaborations
7. Market Dynamics
I. Growth Drivers
a. Rise in Prevalence of Cystic Fibrosis
b. Treatment Advancements
c. Increase in Investments and Funding
II. Challenges
a. High Cost of Medications
b. Limited Medications for All Mutation Typess
III. Trends
a. Next-Generation CFTR Modulators
b. CFTR Amplifiers
c. Gene Editing
d. Bypassing the CFTR channel
8. Patent Landscape
I. Overall IP Trends in Cystic Fibrosis Market
II Patent Trends by Focus Area
III. Key Patents in Cystic Fibrosis Market
IV. Cystic Fibrosis Market Patent Trend by Key Players
V. Cystic Fibrosis Market Patent Trend by Top Inventors
9. Company Profiling
I. Vertex Pharmaceuticals
a. Business Overview
b. Financial Overview
c. Product Pipeline
d. Business Strategy
II. Galapagos
a. Business Overview
b. Financial Overview
c. Product Pipeline
d. Business Strategy
III. AbbVie
a. Business Overview
b. Financial Overview
c. Product Pipeline
d. Business Strategy
IV. Chiesi Farmaceutici
a. Business Overview
b. Financial Overview
c. Product Pipeline
V. Anthera Pharmaceuticals
a. Business Overview
b. Financial Overview
c. Product Pipeline
Fig 1.1 Clinical Manifestations and Complications of Cystic Fibrosis
Fig 1.2 Mode of Inheritance of Cystic Fibrosis
Fig 2.1 Global Cystic Fibrosis Market by Value; 2016-2028
Fig 2.2 Key Product Sales in Cystic Fibrosis Market; 2016-2017
Fig 2.3 Cystic Fibrosis Market by Therapeutic Approach; 2017
Fig 2.4 Global CFTR Modulator Class Market by Value; 2012-2018
Fig 2.5 Global Mucociliary Clearance Class Market by Value; 2012-2018
Fig 2.6 Global Anti-Infective Class Market by Value; 2012-2018
Fig 2.7 Global Pancreatic Enzyme Supplement Class Market by Value; 2012-2018
Fig 3.1 Cystic Fibrosis Products Approval Timeline
Fig 4.1 Cystic Fibrosis Drug pipeline by Stage of Development
Fig 4.2 Cystic Fibrosis Drug Pipeline by Therapeutic Class
Fig 4.3 Cystic Fibrosis Drug Pipeline by Anti-Infective Class
Fig 4.4 Cystic Fibrosis Drug Pipeline by Anti-Inflammatory Class
Fig 4.5 Cystic Fibrosis Drug Pipeline by CFTR Modulator Class
Fig 4.6 Cystic Fibrosis Drug Pipeline by Mucociliary Clearance Class
Fig 4.7 Cystic Fibrosis Miscellaneous Drug Pipeline
Fig 4.8 Cystic Fibrosis Drug Pipeline by Molecule Type
Fig 4.9 Cystic Fibrosis Drug Pipeline by Route of Administration
Fig 4.10 Cystic Fibrosis Drug Pipeline by Therapy Type6
Fig 4.11 Cystic Fibrosis Drug Pipeline by Company
Fig 7.1 Prevalence of Cystic Fibrosis in the US; 2001-2016
Fig 7.2 Annual Mortality Rate of Cystic Fibrosis Patients in the US; 2001-2016
Fig 7.3 Predicted median survival of Cystic Fibrosis Patients in the US; 2001-2016
Fig 7.4 Global Pharmaceutical R&D Expenditure; 2016-2022
Fig 7.5 Average Monthly Medicare Price of Drugs in the US; 1994-2008
Fig 8.1 Overall IP Trends in Cystic Fibrosis Market; 2007-2018
Fig 8.2 Earliest Priority Country Filing Trend in Cystic Fibrosis Market; 2007-2018
Fig 8.3 Cystic Fibrosis Patent Trend by Focus Area; 2007-2018
Fig 8.4 Cystic Fibrosis Patent Trend by Composition; 2007-2018
Fig 8.5 Cystic Fibrosis Patent Trend by Route of Administration; 2007-2018
Fig 8.6 Cystic Fibrosis Patent Trend by Key Patent Holder (Legal Assignees); 2007-2018
Fig 8.7 Top inventor in Cystic Fibrosis Market; 2007-2018
Fig 9.1 Vertex Pharmaceuticals Revenue; 2013-2017
Fig 9.2 Vertex Pharmaceuticals Cystic Fibrosis Revenue; 2013-2017
Fig 9.3 Vertex Pharmaceuticals Product Share by Revenue; 2013-2017
Fig 9.4 Vertex Pharmaceuticals Research & Development Expenses; 2013-2017
Fig 9.5 Galapagos Revenue; 2013-2017
Fig 9.6 Galapagos Research & Development Expenses; 2013-2017
Fig 9.7 AbbVie Revenue; 2013-2017
Fig 9.8 AbbVie Research & Development Expenses; 2013-2017
Fig 9.9 Chiesi Farmaceutici Revenue; 2012-2016
Fig 9.10 Chiesi Farmaceutici Research & Development Expenses; 2012-2016
Fig 9.11 Anthera Pharmaceuticals Research & Development Expenses; 2013-2017
Table 1.1 Classification of CFTR Mutations
Table 3.1 Key Products in CFTR Modulator Class
Table 3.2 Key Products in Mucociliary Clearance Class
Table 3.3 Key Products in Anti-Infective Class
Table 3.4 Key Products in Pancreatic Enzyme Supplement Class
Table 4.1 Cystic Fibrosis Drug Pipeline by Stage of Development
Table 4.2 Cystic Fibrosis Drug Pipeline by Anti-Infective Class
Table 4.3 Expanded Label Cystic Fibrosis Drug Pipeline by Anti-Infective Class
Table 4.4 Cystic Fibrosis Drug Pipeline by Anti-Inflammatory Class
Table 4.5 Cystic Fibrosis Drug Pipeline by CFTR Modulator Class
Table 4.6 Expanded Label Cystic Fibrosis Drug Pipeline by CFTR Modulator Class
Table 4.7 Cystic Fibrosis Drug Pipeline by Mucociliary Clearance Class
Table 4.8 Cystic Fibrosis Drug Pipeline by Mucociliary Clearance Class for Different Geographies
Table 4.9 Cystic Fibrosis Drug Pipeline by Nutritional Class
Table 4.10 Cystic Fibrosis Miscellaneous Drug Pipeline
Table 4.11 Cystic Fibrosis Drug Pipeline by Company
Table 5.1 Cystic Fibrosis Merger & Acquisition Activities
Table 6.1 Cystic Fibrosis Major Collaborations
Table 7.1 Programs Funded by the Cystic Fibrosis Foundation
Table 8.1 Cystic Fibrosis Patent Trend by Focus Area (Year Wise Filing); 2007-2018
Table 8.2 Cystic Fibrosis Patent Trend by Composition (Year Wise Filing); 2007-2018
Table 8.3 Key Patents in Cystic Fibrosis Market
Table 8.4 Key Patent Holder Year Wise Patent Filing in Cystic Fibrosis Market; 2007-2018
Table 8.5 Key Player Patent Filing by Focus area
Table 8.6 Key Player Patent Filling by Composition
Table 8.7 Top Collaborations of the Assignees
Table 8.8 Top Inventors Association with Key Players
Table 9.1 Cystic Fibrosis Product Pipeline of Vertex Pharmaceuticals
Table 9.2 Cystic Fibrosis Product Pipeline of Galapagos
Table 9.3 Cystic Fibrosis Product Pipeline of AbbVie
Table 9.4 Cystic Fibrosis Product Pipeline of Chiesi Farmaceutici
Table 9.5 Cystic Fibrosis Product Pipeline of Anthera Pharmaceuticals