Cystic Fibrosis: Market Research Report
This report analyzes the worldwide markets for Cystic Fibrosis in US$ Million. The report provides separate comprehensive analytics for the US, Canada, Europe, and Rest of World. Annual estimates and forecasts are provided for the period 2014 through 2022. Market data and analytics are derived from primary and secondary research.
Company profiles are primarily based on public domain information including company URLs.
The report profiles 56 companies including many key and niche players such as -
Company profiles are primarily based on public domain information including company URLs.
The report profiles 56 companies including many key and niche players such as -
- Advanced Inhalation Therapies (AIT) Ltd.
- AbbVie Inc.
- Galapagos NV
- Genentech, Inc.
- Gilead Sciences, Inc.
I. INTRODUCTION, METHODOLOGY & PRODUCT DEFINITIONS
II. EXECUTIVE SUMMARY
1. MARKET OVERVIEW
Cystic Fibrosis – A Prelude
CF Disease Prevalence: A Brief Note
Treatment Modalities for Cystic Fibrosis
CFTR Modulators: Key Drug Category
Competitive Scenario
Vertex: At the Forefront of Cystic Fibrosis Therapeutics
2. RESEARCH IN CYSTIC FIBROSIS SPACE
Areas of Research & Development
Gene Therapy – A Step Towards Cure
Research Findings & Innovations
Updated CF Diagnosis Guidelines to Aid in Diagnosis and Personalized Treatment
Pipeline Drugs – An Overview
Select Pipeline Drugs for Cystic Fibrosis Treatment by Development Stage, 2016
Description of Select Pipeline Drugs
Ataluren
VX-661 + Ivacaftor
Riociguat
QBW251
CTP-656
Cavosonstat (N91115)
VX-440
QR-010
pGM169/GL67A
Acebilustat
Resunab
Inhaled Mannitol
VX-371
Inhaled Levoflaxacin (Quinsair)
AeroVanc (Vancomycin Inhalation Powder)
3. DISEASE OVERVIEW
Cystic Fibrosis – A Hereditary Disease
Mutation in CFTR Gene – A Major Cause of CF
(F508 – The Most Common Mutation that Affects CFTR Protein
Hereditary Risk of the Disease
Pathophysiology of the Disease
Symptoms of Cystic Fibrosis
Common Symptoms in Early Childhood
Common Symptoms in Grownups or Adults:
Complications Associated with Cystic Fibrosis
Sinus and Lung Illness
Pancreatic, Gastrointestinal, and Liver Disease
Endocrine Disorders
Infertility
Diagnosis of Cystic Fibrosis
Newborn Test
Sweat Test
The IRT Test
Carrier Test
Antenatal Test
Other Common Tests
Prenatal Diagnosis of Cystic Fibrosis
Tests to Diagnose CF-Related Complications
Cystic Fibrosis in Children
4. CYSTIC FIBROSIS TREATMENT: AN OVERVIEW
5. CLINICAL STUDIES
Vertex Pharmaceuticals Announces Positive Phase 3 Study Results of ORKAMBI
ProQR Therapeutics Announces Positive Proof-of-Concept Study for QR-010
Concert Pharmaceuticals Presents Positive Food Effect Results for CTP-656
Vertex Pharmaceuticals Initiates Phase 2 Studies for VX-440 & VX-152
Vertex Pharmaceuticals Provides Update on Phase 3 Study of VX-661 with ivacaftor
Protalix BioTherapeutics Announces Start of Phase II Trial of AIR DNase (PRX-110)
Nivalis Therapeutics Announces Completion of Enrollment for Phase 2 Study of N91115
Alaxia SAS Announces Agreement and Grant from CFFT for ALX-009 Studies
Polyphor Completes Successful Phase I Study of Inhaled POL6014
AlgiPharma Receives Grant from CFFT for Phase 2 Trials of OligoG
Health Canada Grants Clearance to ProMetic for its PBI-4050
Cystic Fibrosis Canada Finances Laurent Pharmaceuticals’ LAU-7b Phase 2 Trial
Galapagos NV Begins Phase 2 Study of GLPG1837
Pharmaxis Announces Positive Phase II Trial Results of Bronchitol
PTC Therapeutics Completes Enrollment for second Phase 3 clinical trial of Translarna
Anthera Pharmaceuticals Begins Phase 3 Study of Oral Liprotamase
Parion Sciences Announces Expanded Enrollment Criteria for CLEAN-CF Trial
Laurent Announces Positive Phase 1b Trial Results for LAU-7b
Celtaxsys Announces Positive Phase 1 Clinical Trial Data of CTX-4430
Corbus Pharmaceuticals Receives USFDA Clearance to Initiate Phase 2 Study of Resunab
Savara Pharmaceuticals Unveils Positive Phase 2 Trial Results of AeroVanc
KaloBios Reports Top-line Data from Phase 2 Study of KB001-A
Galapagos NV Nominates GLPG2222 for Combination Therapy with GLPG1837
Cystic Fibrosis Trust and NovaBiotics Partner for the Phase IIa Study of Lynovex
PTC Therapeutics Initiates Phase 3 Trial of Translarna/ataluren
Kamada to Start Clinical Study of Alpha-1 Antitrypsin (AAT) for Cystic Fibrosis
6. PRODUCT LAUNCHES/APPROVALS
EC Grants Orphan Drug Designation to Corbus Pharmaceuticals’ Resunab
FDA Approves Vertex’s ORKAMBI for Extended Age Group
Pharmaxis Announces Approval of Bronchitol in Russia
FDA Grants Orphan Drug Status to Pulmatrix’s CF Drug PUR1900
ProQR Therapeutics Announces FDA Fast Track Designation for QR-010
Alcresta Therapeutics Launches Relizorb Cartridge
EC Grants Orphan Medicinal Product Designation to Arch Biopartners’ AB569
TGV-Inhalonix Receives Orphan Drug Designation for its Mul-1867
RespirTech Launches Development of Remote Monitoring System for CF
Vertex Pharmaceuticals Receives TGA Approval of ORKAMBI
AlgiPharma’s OligoG Receives Orphan Drug Designation by FDA
Proteostasis Therapeutics Receives FDA Fast Track designation for PTI-428
Proteostasis Therapeutics Expands CF Drug Pipeline
Vertex Pharmaceuticals Receives Two EU Approvals for KALYDECO
USPTO Issues Patent to OrPro Therapeutics’ Theradux
FDA Approves Vertex’s ORKAMBI
Advanced Inhalation Therapies Receives Orphan Medicinal Product Designation for AIT-CF from EMA
FDA Approves New Age Group Indication for Vertex Pharmaceuticals’ KALYDECO
Sandoz Launches Tobramycin Inhalation Solution in the US
FDA Approves sNDA for KALYDECO
EC Grants Orphan Designation to Macrolide CF Drug by Synovo GmbH
7. RECENT INDUSTRY ACTIVITY
Pharmaxis Enters into Research Collaboration with Woolcock Institute
Horizon Pharma Acquires Raptor Pharmaceutical
AstraZeneca to Sell Drug Commercialization Rights to Pfizer
Vertex Pharmaceuticals and Moderna Therapeutics Enter into Research Collaboration
Editas Medicine Enters into Agreement with CFFT for CRISPR/Cas9-based CF Drugs
Galapagos and AbbVie Expand Agreement for CF Portfolio
Raptor Pharmaceutical Acquires Quinsair from Tripex Pharmaceuticals
Galapagos NV Enters into Licensing Agreement with the HUB Foundation
Zambon SpA Acquires Nigaard Pharma
ManRos and Cyclacel Enter into Licensing Agreement for Seliciclib
Vertex and Parion Sciences Collaborate to Develop Investigational CF Treatment
Ruthigen and Pulmatrix Enter into Merger Agreement
N30 Pharmaceuticals Changes Name to Nivalis Therapeutics
Anthera Pharmaceuticals Acquires Sollpura/liprotamase from Eli Lilly
OPKO Health Acquires Inspiromatic dry powder inhaler from Inspiro Medical
CURx Pharmaceuticals Enters into Global License Agreement with Gilead Sciences
8. SELECT CLINICAL-STAGE PLAYERS
Advanced Inhalation Therapies (AIT) Ltd (Israel)
Alaxia SAS (France)
AlgiPharma AS (Norway)
Anthera Pharmaceuticals, Inc. (USA)
Arch BioPartners (Canada)
Celtaxsys, Inc. (USA)
Concert Pharmaceuticals, Inc. (USA)
Corbus Pharmaceuticals Holdings, Inc. (USA)
CURx Pharmaceuticals (USA)
Nivalis Therapeutics, Inc. (USA)
NovaBiotics Ltd (UK)
Parion Sciences, Inc. (USA)
Pharmaxis Ltd (Australia)
PTC Therapeutics, Inc. (USA)
Pulmatrix, Inc. (USA)
9. FOCUS ON SELECT GLOBAL PLAYERS
AbbVie Inc. (USA)
Galapagos NV (Belgium)
Genentech, Inc. (USA)
Gilead Sciences, Inc. (USA)
Novartis AG (Switzerland)
Vertex Pharmaceuticals (USA)
10. GLOBAL MARKET PERSPECTIVE
III. MARKET
1. THE UNITED STATES
A. Market Analysis
Snapshots of Cystic Fibrosis (CF) Prevalence in the US
CFFT: NGO Dedicated to Cystic Fibrosis
List of Drugs (Companies) in the US Funded by CFFT for Cystic Fibrosis (2016)
Drug Pricing in Wake of Orkambi’s High Price
Clinical Studies
Product Launches
Strategic Corporate Developments
Select Key Players
Clinical-Stage Players
B. Market Analytics
2. CANADA
A. Market Analysis
Clinical Studies
Product Launch
Arch BioPartners: Player Review
B. Market Analytics
3. EUROPE
A. Market Analysis
Clinical Studies
Product Launches
Strategic Corporate Developments
Select Key Players
B. Market Analytics
4. REST OF WORLD
A. Market Analysis
Australia – High Out-of-Pocket Expenditure by CF Patients
Clinical Studies
Product Launch
Strategic Corporate Development
Select Key Players
B. Market Analytics
Table 13: Rest of World Recent Past, Current & Future Analysis for Cystic Fibrosis Therapeutics Analyzed with Annual Sales Figures in US$ Million for Years 2014 through 2022 (includes corresponding Graph/Chart)III-26IV. COMPETITIVE LANDSCAPE
Total Companies Profiled: 56 (including Divisions/Subsidiaries - 58)
The United States (30)
Canada (4)
Europe (20)
France (2)
Germany (2)
The United Kingdom (3)
Italy (2)
Rest of Europe (11)
Asia-Pacific (Excluding Japan) (1)
Middle East (3)
II. EXECUTIVE SUMMARY
1. MARKET OVERVIEW
Cystic Fibrosis – A Prelude
CF Disease Prevalence: A Brief Note
Table 1. Prevalence of Cystic Fibrosis across Select Countries
Treatment Modalities for Cystic Fibrosis
CFTR Modulators: Key Drug Category
Competitive Scenario
Table 2. Leading CF Drugs on the Market (2016): Percentage Share Breakdown by Sales (includes corresponding Graph/Chart)
Vertex: At the Forefront of Cystic Fibrosis Therapeutics
2. RESEARCH IN CYSTIC FIBROSIS SPACE
Areas of Research & Development
Gene Therapy – A Step Towards Cure
Research Findings & Innovations
Updated CF Diagnosis Guidelines to Aid in Diagnosis and Personalized Treatment
Pipeline Drugs – An Overview
Select Pipeline Drugs for Cystic Fibrosis Treatment by Development Stage, 2016
Description of Select Pipeline Drugs
Ataluren
VX-661 + Ivacaftor
Riociguat
QBW251
CTP-656
Cavosonstat (N91115)
VX-440
QR-010
pGM169/GL67A
Acebilustat
Resunab
Inhaled Mannitol
VX-371
Inhaled Levoflaxacin (Quinsair)
AeroVanc (Vancomycin Inhalation Powder)
3. DISEASE OVERVIEW
Cystic Fibrosis – A Hereditary Disease
Mutation in CFTR Gene – A Major Cause of CF
(F508 – The Most Common Mutation that Affects CFTR Protein
Table 3. Most Common Mutations and Worldwide Frequency (includes corresponding Graph/Chart)
Hereditary Risk of the Disease
Pathophysiology of the Disease
Symptoms of Cystic Fibrosis
Common Symptoms in Early Childhood
Common Symptoms in Grownups or Adults:
Complications Associated with Cystic Fibrosis
Sinus and Lung Illness
Pancreatic, Gastrointestinal, and Liver Disease
Endocrine Disorders
Infertility
Diagnosis of Cystic Fibrosis
Newborn Test
Sweat Test
The IRT Test
Carrier Test
Antenatal Test
Other Common Tests
Prenatal Diagnosis of Cystic Fibrosis
Tests to Diagnose CF-Related Complications
Cystic Fibrosis in Children
4. CYSTIC FIBROSIS TREATMENT: AN OVERVIEW
5. CLINICAL STUDIES
Vertex Pharmaceuticals Announces Positive Phase 3 Study Results of ORKAMBI
ProQR Therapeutics Announces Positive Proof-of-Concept Study for QR-010
Concert Pharmaceuticals Presents Positive Food Effect Results for CTP-656
Vertex Pharmaceuticals Initiates Phase 2 Studies for VX-440 & VX-152
Vertex Pharmaceuticals Provides Update on Phase 3 Study of VX-661 with ivacaftor
Protalix BioTherapeutics Announces Start of Phase II Trial of AIR DNase (PRX-110)
Nivalis Therapeutics Announces Completion of Enrollment for Phase 2 Study of N91115
Alaxia SAS Announces Agreement and Grant from CFFT for ALX-009 Studies
Polyphor Completes Successful Phase I Study of Inhaled POL6014
AlgiPharma Receives Grant from CFFT for Phase 2 Trials of OligoG
Health Canada Grants Clearance to ProMetic for its PBI-4050
Cystic Fibrosis Canada Finances Laurent Pharmaceuticals’ LAU-7b Phase 2 Trial
Galapagos NV Begins Phase 2 Study of GLPG1837
Pharmaxis Announces Positive Phase II Trial Results of Bronchitol
PTC Therapeutics Completes Enrollment for second Phase 3 clinical trial of Translarna
Anthera Pharmaceuticals Begins Phase 3 Study of Oral Liprotamase
Parion Sciences Announces Expanded Enrollment Criteria for CLEAN-CF Trial
Laurent Announces Positive Phase 1b Trial Results for LAU-7b
Celtaxsys Announces Positive Phase 1 Clinical Trial Data of CTX-4430
Corbus Pharmaceuticals Receives USFDA Clearance to Initiate Phase 2 Study of Resunab
Savara Pharmaceuticals Unveils Positive Phase 2 Trial Results of AeroVanc
KaloBios Reports Top-line Data from Phase 2 Study of KB001-A
Galapagos NV Nominates GLPG2222 for Combination Therapy with GLPG1837
Cystic Fibrosis Trust and NovaBiotics Partner for the Phase IIa Study of Lynovex
PTC Therapeutics Initiates Phase 3 Trial of Translarna/ataluren
Kamada to Start Clinical Study of Alpha-1 Antitrypsin (AAT) for Cystic Fibrosis
6. PRODUCT LAUNCHES/APPROVALS
EC Grants Orphan Drug Designation to Corbus Pharmaceuticals’ Resunab
FDA Approves Vertex’s ORKAMBI for Extended Age Group
Pharmaxis Announces Approval of Bronchitol in Russia
FDA Grants Orphan Drug Status to Pulmatrix’s CF Drug PUR1900
ProQR Therapeutics Announces FDA Fast Track Designation for QR-010
Alcresta Therapeutics Launches Relizorb Cartridge
EC Grants Orphan Medicinal Product Designation to Arch Biopartners’ AB569
TGV-Inhalonix Receives Orphan Drug Designation for its Mul-1867
RespirTech Launches Development of Remote Monitoring System for CF
Vertex Pharmaceuticals Receives TGA Approval of ORKAMBI
AlgiPharma’s OligoG Receives Orphan Drug Designation by FDA
Proteostasis Therapeutics Receives FDA Fast Track designation for PTI-428
Proteostasis Therapeutics Expands CF Drug Pipeline
Vertex Pharmaceuticals Receives Two EU Approvals for KALYDECO
USPTO Issues Patent to OrPro Therapeutics’ Theradux
FDA Approves Vertex’s ORKAMBI
Advanced Inhalation Therapies Receives Orphan Medicinal Product Designation for AIT-CF from EMA
FDA Approves New Age Group Indication for Vertex Pharmaceuticals’ KALYDECO
Sandoz Launches Tobramycin Inhalation Solution in the US
FDA Approves sNDA for KALYDECO
EC Grants Orphan Designation to Macrolide CF Drug by Synovo GmbH
7. RECENT INDUSTRY ACTIVITY
Pharmaxis Enters into Research Collaboration with Woolcock Institute
Horizon Pharma Acquires Raptor Pharmaceutical
AstraZeneca to Sell Drug Commercialization Rights to Pfizer
Vertex Pharmaceuticals and Moderna Therapeutics Enter into Research Collaboration
Editas Medicine Enters into Agreement with CFFT for CRISPR/Cas9-based CF Drugs
Galapagos and AbbVie Expand Agreement for CF Portfolio
Raptor Pharmaceutical Acquires Quinsair from Tripex Pharmaceuticals
Galapagos NV Enters into Licensing Agreement with the HUB Foundation
Zambon SpA Acquires Nigaard Pharma
ManRos and Cyclacel Enter into Licensing Agreement for Seliciclib
Vertex and Parion Sciences Collaborate to Develop Investigational CF Treatment
Ruthigen and Pulmatrix Enter into Merger Agreement
N30 Pharmaceuticals Changes Name to Nivalis Therapeutics
Anthera Pharmaceuticals Acquires Sollpura/liprotamase from Eli Lilly
OPKO Health Acquires Inspiromatic dry powder inhaler from Inspiro Medical
CURx Pharmaceuticals Enters into Global License Agreement with Gilead Sciences
8. SELECT CLINICAL-STAGE PLAYERS
Advanced Inhalation Therapies (AIT) Ltd (Israel)
Alaxia SAS (France)
AlgiPharma AS (Norway)
Anthera Pharmaceuticals, Inc. (USA)
Arch BioPartners (Canada)
Celtaxsys, Inc. (USA)
Concert Pharmaceuticals, Inc. (USA)
Corbus Pharmaceuticals Holdings, Inc. (USA)
CURx Pharmaceuticals (USA)
Nivalis Therapeutics, Inc. (USA)
NovaBiotics Ltd (UK)
Parion Sciences, Inc. (USA)
Pharmaxis Ltd (Australia)
PTC Therapeutics, Inc. (USA)
Pulmatrix, Inc. (USA)
9. FOCUS ON SELECT GLOBAL PLAYERS
AbbVie Inc. (USA)
Galapagos NV (Belgium)
Genentech, Inc. (USA)
Gilead Sciences, Inc. (USA)
Novartis AG (Switzerland)
Vertex Pharmaceuticals (USA)
10. GLOBAL MARKET PERSPECTIVE
Table 4. World Recent Past, Current and Future Analysis for Cystic Fibrosis Therapeutics by Geographic Region - US, Canada, Europe, and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2014 through 2022 (includes corresponding Graph/Chart)
Table 5. World 7-Year Perspective for Cystic Fibrosis Therapeutics by Geographic Region - Percentage Breakdown of Dollar Sales for US, Canada, Europe, and Rest of World Markets for Years 2016 and 2022 (includes corresponding Graph/Chart)
Table 6. World Recent Past, Current and Future Analysis for Cystic Fibrosis Therapeutics by Drug Type - Mucolytics, CFTR Modulators, Anti-Infectives, PERT, and Bronchodilators Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2014 through 2022 (includes corresponding Graph/Chart)
Table 7. World 7-Year Perspective for Cystic Fibrosis Therapeutics by Drug Type - Percentage Breakdown of Dollar Sales for Mucolytics, CFTR Modulators, Anti-Infectives, PERT, and Bronchodilators Markets for Years 2016 and 2022 (includes corresponding Graph/Chart)
III. MARKET
1. THE UNITED STATES
A. Market Analysis
Snapshots of Cystic Fibrosis (CF) Prevalence in the US
CFFT: NGO Dedicated to Cystic Fibrosis
List of Drugs (Companies) in the US Funded by CFFT for Cystic Fibrosis (2016)
Drug Pricing in Wake of Orkambi’s High Price
Clinical Studies
Product Launches
Strategic Corporate Developments
Select Key Players
Clinical-Stage Players
B. Market Analytics
Table 8. US Recent Past, Current & Future Analysis for Cystic Fibrosis Therapeutics Analyzed with Annual Sales Figures in US$ Million for Years 2014 through 2022 (includes corresponding Graph/Chart)
2. CANADA
A. Market Analysis
Clinical Studies
Product Launch
Arch BioPartners: Player Review
B. Market Analytics
Table 9. Canadian Recent Past, Current & Future Analysis for Cystic Fibrosis Therapeutics Analyzed with Annual Sales Figures in US$ Million for Years 2014 through 2022 (includes corresponding Graph/Chart)
3. EUROPE
A. Market Analysis
Table 10. Number of Cystic Fibrosis Patients in Europe by Country (2014) (includes corresponding Graph/Chart)
Table 11. Proportion of CF Patients in Europe (Adults Vs Children) by Country (2014) (includes corresponding Graph/Chart)
Clinical Studies
Product Launches
Strategic Corporate Developments
Select Key Players
B. Market Analytics
Table 12. European Recent Past, Current & Future Analysis for Cystic Fibrosis Therapeutics Analyzed with Annual Sales Figures in US$ Million for Years 2014 through 2022 (includes corresponding Graph/Chart)
4. REST OF WORLD
A. Market Analysis
Australia – High Out-of-Pocket Expenditure by CF Patients
Clinical Studies
Product Launch
Strategic Corporate Development
Select Key Players
B. Market Analytics
Table 13: Rest of World Recent Past, Current & Future Analysis for Cystic Fibrosis Therapeutics Analyzed with Annual Sales Figures in US$ Million for Years 2014 through 2022 (includes corresponding Graph/Chart)III-26IV. COMPETITIVE LANDSCAPE
Total Companies Profiled: 56 (including Divisions/Subsidiaries - 58)
The United States (30)
Canada (4)
Europe (20)
France (2)
Germany (2)
The United Kingdom (3)
Italy (2)
Rest of Europe (11)
Asia-Pacific (Excluding Japan) (1)
Middle East (3)
