This report analyzes the US market for Cystic Fibrosis in Millions of US$. Annual estimates and forecasts are provided for the period of 2007 through 2015. Also, a six-year historic analysis is provided for this market. The report profiles 56 companies including many key and niche players such as Abbott Laboratories, Axcan Pharma, Inc., Digestive Care, Inc., Gilead Sciences, Inc., Eurand N.V., Inspire Pharmaceuticals, Inc., KaloBios Pharmaceuticals, Inc., Nektar Therapeutics, PTC Therapeutics, Inc., Transave, Inc., Vertex Pharmaceuticals, Inc., Actelion Ltd., F. Hoffmann'La Roche Ltd., Novartis AG, and Pharmaxis Ltd. Market data and analytics are derived from primary and secondary research. Company profiles are mostly extracted from URL research and reported select online sources.

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US Cystic Fibrosis Industry
Published on June 2010
Report Summary
This report analyzes the US market for Cystic Fibrosis in Millions of US$. Annual estimates and forecasts are provided for the period
of 2007 through 2015. Also, a six-year historic analysis is provided for this market. The report profiles 56 companies including many
key and niche players such as Abbott Laboratories, Axcan Pharma, Inc., Digestive Care, Inc., Gilead Sciences, Inc., Eurand N.V.,
Inspire Pharmaceuticals, Inc., KaloBios Pharmaceuticals, Inc., Nektar Therapeutics, PTC Therapeutics, Inc., Transave, Inc., Vertex
Pharmaceuticals, Inc., Actelion Ltd., F. Hoffmann'La Roche Ltd., Novartis AG, and Pharmaxis Ltd. Market data and analytics are
derived from primary and secondary research. Company profiles are mostly extracted from URL research and reported select online
sources.
Table of Content
CYSTIC FIBROSISMCP-6233
A US MARKET REPORT
CONTENTS
I. INTRODUCTION, METHODOLOGY & PRODUCT DEFINITIONS
Study Reliability and Reporting Limitations I-1
Disclaimers I-2
Data Interpretation & Reporting Level I-3
Quantitative Techniques & Analytics I-3
Product Definitions and Scope of Study I-3
II. A US MARKET REPORT
1. MARKET OVERIVEW II-1
A Global Fact Sheet on Cystic Fibrosis II-1
Current and Future Analysis II-1
Cystic Fibrosis: A Life-Limiting Genetic Disease II-1
Cystic Fibrosis Scenario in the US II-2
Narrowing Mortality in the US II-2
Median Survival Age (MSA) of Patients with CF in United States II-2
Caucasians at Greater Risk II-3
A Market with Huge Unmet Medical Needs II-3
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Pulmozyme and TOBI Dominate CF Market II-3
Table 1: US Market for Cystic Fibrosis by Leading Drugs
(2008): Annual Sales in US$ Million for Pulmozyme and TOBI II-3
Genentech/Roche and Novartis Lead the Pack II-4
Other Potential Market Contenders II-4
Mega Deals for Niche Local Players II-4
2. CYSTIC FIBROSIS TREATMENT: AN OVERVIEW II-5
EXISTING TREATMENT OPTIONS FOR CYSTIC FIBROSIS II-5
A Quick Glance at Common Drugs for Cystic Fibrosis Management II-6
Analysis of Drugs Indicated for Management of Cystic Fibrosis II-7
Antibiotic Therapy for Lung Infections II-7
Select Antibiotics Approved for CF Treatment II-7
TOBI (Tobramycin inhalation solution) II-7
Other Common Cystic Fibrosis Infections and Recommended
Antibiotics II-7
Cayston (Aztreonam for inhalation solution) II-8
Combination Antibiotic Therapy II-8
Pulmozyme - The Effective Mucolytic Agent II-8
Pancreatic Enzyme Replacement Therapy (PERT) for EPI II-8
FDA Approval Made Mandate for PI Products II-9
Most Commonly Prescribed Pes II-9
Leading PE Drugs Worldwide for Treating Pancreatic
Insufficiency Associated with Cystic Fibrosis II-10
ULTRASE (Axcan Pharma) II-10
VIOKASE (Axcan Pharma) II-10
CREON® (Solvay Pharmaceuticals) II-10
PANZYTRAT (Axcan Pharma) II-10
ZENPEP TM (Eurand N.V.) II-11
Additional Treatment for Patients with CF II-11
Surgical and Other Techniques II-11
Lung Transplantation - Recommended for Rare Cases of CF II-11
Feeding Tube - For Extra Nutrition II-11
Bowel Surgery - To Remove any Blockage in Bowel II-11
3. RESEARCH IN CYSTIC FIBROSIS SPACE II-12
Pitfalls in Current Cystic Fibrosis Treatment II-12
Areas of Research & Development II-12
Major Companies Active in Cystic Fibrosis Research II-13
Companies and Area of Research II-13
Gene Therapy- A Step Towards Cure II-14
PLASmin' - The Only Investigational Drug Based on Gene Therapy II-14
Rising Concerns over Animal-Derived Drugs II-14
4. PIPELINE ANALYSIS II-15
Active Cystic Fibrosis Pipeline - A Ray of Hope II-15
Cystic Fibrosis Pipeline (As of January 2010) II-15
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Gene Therapy II-15
Protein Assist/Repair Therapy: II-15
Restoration of Salt Transport Therapy: II-16
Anti-Inflammatory Agents: II-16
Anti-infective Agents: II-16
Other Developmental Drugs: II-17
5. DISEASE OVERVIEW II-18
Cystic Fibrosis - A Hereditary Disease II-18
Mutation in CFTR Gene - A Major Cause of CF II-18
DF508 - The Most Common Mutation that Affects CFTR Protein II-18
Table 2: Most Common Mutations and Worldwide Frequency
(includes corresponding Graph/Chart) II-18
Hereditary Risk of the Disease II-19
Pathophysiology of the Disease II-19
Symptoms of Cystic Fibrosis II-19
Common Symptoms in Early Childhood II-19
Common Symptoms in Grownups or Adults: II-20
Complications Associated with Cystic Fibrosis II-20
Sinus and Lung Illness II-20
Pancreatic, Gastrointestinal, and Liver Disease II-20
Endocrine Disorders II-21
Infertility II-21
Diagnosis of Cystic Fibrosis II-21
Newborn Test II-22
Sweat Test II-22
The IRT Test II-22
Carrier Test II-22
Antenatal Test II-22
Other Common Tests II-23
Prenatal Diagnosis of Cystic Fibrosis II-23
Tests to Diagnose CF-Related Complications II-23
Cystic Fibrosis in Kids II-24
6. RESEARCH AND DEVELOPMENT INITIATIVES OF COMPANIES II-25
VERTEX ANNOUNCES PHASE IIA RESULTS OF VX-809 II-25
Actelion's Miglustat Proves Effective for Treating Cystic
Fibrosis II-25
Transave Announces Phase II Clinical Trial Results of Arikace' II-25
Alnara Announces Positive Phase III Results of Liprotamase II-26
Novartis Announces Phase III Results of Tobramycin Inhalation
Powder II-26
KaloBios Announces Encouraging Phase I/II Results of KB001 II-27
PTC Therapeutics Initiates Ataluren's Phase III Trial II-27
Mpex Posts Positive Results of Phase 2b Study of Aeroquin II-28
Pharmaxis Announces Positive Results for Bronchitol II-28
Inspire Pharmaceuticals Provides Update on Denufosol
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Tetrasodium's Phase III Clinical Trial Program II-28
Vertex Announces Promising Phase 2a Clinical Trial Results of
VX-770 II-28
RegeneRx Biopharmaceuticals to Develop Cystic Fibrosis Drug II-29
Venus Remedies Introduces Tobracef for CF Induced Lung Infection II-29
Axentis Pharma Commences Phase IIa Trial with Fluidosome® II-29
Kamada Announces Positive Phase 2 Results of Alpha-1 Antitrypsin II-29
Actelion Initiates Phase 2a Proof-of-Concept Clinical Trial
with Miglustat in Cystic Fibrosis Patients II-30
Forest Labs Commences Phase III Trials of New Dry Powder
Formulation for Lung Infection Associated with CF II-30
7. RECENT INDUSTRY ACTIVITY II-31
Abbott Acquires Solvay Pharmaceuticals II-31
KaloBios Signs Deal with Sanofi Pasteur for KB001 II-31
Roche Acquires Genentech II-32
Altus Pharmaceuticals Announces Strategic Realignment,
Withdraws Product Development Activities of Trizytek II-32
Gilead Sciences Receives License for P-680 from Parion Sciences II-33
NIH Awards Additional Grant to DiscoveryBioMed for CF Product
Development II-33
Lonza Group and Alnara Pharmaceuticals Sign Agreement II-33
Alnara Pharmaceuticals Acquires Rights to Liprotamase II-34
Protea Biosciences Sign Agreement with Mayoly Spindler II-34
Axentis Pharma Obtains Orphan Drug Status for Liposomal
Formulation of Tobramycin II-34
Novartis Completes Acquisition of Nektar's Pulmonary Business II-35
Gilead Sciences Submits MAA to EMEA for Aztreonam Lysine II-35
VGX Pharmaceuticals and CFT Sign Agreement II-35
Parion Sciences Receives Funds to Develop GSP9411 II-36
Genzyme to Acquire Marketing Rights Over PTC124 II-36
EPIX Pharmaceuticals Expands Partnership Deal with CFFTI II-36
8. PRODUCT APPROVALS II-37
Gilead Receives US FDA Approval for Marketing Cayston® in the US II-37
McNeil Pediatrics Obtains Approval for Pancreaze II-37
FDA Approves CREON® for Treating EPI in Patients with CF II-37
Gilead Wins Conditional Marketing Authorization for Cayston®
from EC II-38
Eurand Launches ZENPEP for Pacreatic Insufficiency Associated
with CF II-38
Pharmaxis Submits Application to TGA for Approval of Brochitol
in Australia II-39
Aradigm Receives Orphan Drug Status for Inhaled Liposomal
Ciprofloxacin II-39
Eurand Obtains Complete Response Letter from US FDA for
Zentase, a Pancreatic Enzyme Product II-39
Pharmaxis Files Application with EMA for Bronchitol II-39
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9. FOCUS ON SELECT GLOBAL PLAYERS II-40
Abbott Laboratories (USA) II-40
Axcan Pharma Inc. (Canada) II-40
Digestive Care, Inc. (USA) II-41
Gilead Sciences, Inc. (USA) II-41
Eurand N.V. (The Netherlands) II-41
Inspire Pharmaceuticals, Inc. (USA) II-42
KaloBios Pharmaceuticals, Inc. (USA) II-42
Nektar Therapeutics (USA) II-42
PTC Therapeutics, Inc. (USA) II-43
Transave, Inc. (USA) II-44
Vertex Pharmaceuticals, Inc. (USA) II-44
Actelion Ltd. (Switzerland) II-45
F. Hoffmann-La Roche Ltd. (Switzerland) II-45
Novartis AG (Switzerland) II-46
Pharmaxis Ltd. (Australia) II-46
10. US MARKET PERSPECTIVE II-48
Table 3: US Recent Past, Current & Future Analysis for Cystic
Fibrosis Therapeutics Independently Analyzed with Annual Sales
Figures in US$ Million for Years 2007 through 2015 (includes
corresponding Graph/Chart) II-48
Table 4: US Historic Review for Cystic Fibrosis Therapeutics
Independently Analyzed with Annual Sales Figures in US$
Million for Years 2001 through 2006 (includes corresponding
Graph/Chart) II-48
III. COMPETITIVE LANDSCAPE
Total Companies Profiled: 56 (including Divisions/Subsidiaries - 59)
------------------------------------------
Region/Country Players
------------------------------------------
The United States 36
Canada 1
Japan 1
Europe 18
France 2
Germany 2
The United Kingdom 2
Italy 1
Rest of Europe 11
Asia-Pacific (Excluding Japan) 2
Middle East 1
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