Title: Reports and Intelligence: Cystic Fibrosis Therapeutics Market - Size, Share, Global Trends 2019
1 Cystic Fibrosis Therapeutics in Major Developed
Markets to 2019 CFTR Modulators Initiate Drive
towards Personalized Treatment and Market Growth
Publisher GBI Research
No of pages 76
Publish Date Feb 2014
2Description
GBI Research, a leading business intelligence
provider, has released its latest research report
Cystic Fibrosis Therapeutics in Major Developed
Markets to 2019 - CFTR Modulators Initiate Drive
towards Personalized Treatment and Market
Growth. The value of the Cystic Fibrosis (CF)
market is expected to increase significantly in
value over the forecast period across the leading
eight developed nations, from 695.6 million in
2012 to almost 4.5 billion in 2019. This
equates to a Compound Annual Growth Rate (CAGR)
of 30.4. Novel treatments with disease-modifying
mechanisms of action are the primary factor
driving the growth of the value of the market.
The positive impact of new market entrants will
offset the effects of key patent losses during
the forecast period. Cystic Fibrosis
Transmembrane conductance Regulators (CFTR)
modulators are the first type of CF therapy to
treat the root cause of the disease rather than
the symptoms, and as a result, they offer
patients improved relief and quality of life.
3Description
This effect will be repeated on a larger scale
when lumacaftor and Ataluren (PTC124) enter the
market, as these treatments can be used for a
much larger proportion of the CF population.
Vertex Pharmaceuticals Inc. are the developers of
Kalydeco, lumacaftor and other early phase CFTR
modulators, and are therefore expected to play a
key role in the development of the future CF
market. The pricing of CFTR modulators will be
premium due to the clinical benefits and
disease-modifying effects that they represent.
Symptomatic therapies will be used in conjunction
with CFTR modulators, and will still be a
necessary part of the CF treatment plan. The
antibiotics segment in particular is expected to
become overcrowded and competitive over the
forecast period, due to cheap, generic tobramycin
products and novel market entrants competing for
market share. Request for discount at
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uiry/132421
4Scope
- A disease introduction, which defines the
disease, including symptoms, diagnosis and
treatment. - An analysis of the CF marketed landscape,
including a comparison of the efficacy and safety
of the most prominent brands, as well as the
unmet needs of CF treatment. - A detailed analysis of the CF pipeline,
detailing, among other parameters, drug
distribution by phase, molecule type and
mechanism of action. The CF clinical trial
landscape is then analyzed, with a particular
emphasis on failure rates across different trial
phases as well as the trends in clinical trial
size and duration. This section also includes
profiles and single product forecasts for the
most promising pipeline drugs. - An epidemiological forecast of the major CF
markets. The projected values include total and
treated populations.
5Reasons to buy
- Understand the CF pipeline and the clinical needs
it is addressing. In particular, the importance
and benefits of CFTR modulators are highlighted
and compared with symptomatic treatments. - Develop their knowledge of key products that may
enter the market before 2019. Detailed profiles
of these products are provided, with a focus on
their clinical trial performance, as well as how
they can be incorporated into the CF treatment
plan and what competition they face. -
- Assess the risk associated with CF clinical
trials compared with both industry and therapy
area averages. In addition, in order to
understand the trends in both trial size and
duration according to mechanism of action or
molecule type. - Identify patterns of growth in the CF market over
the forecast period and understand the underlying
causes, as well as observing the contributions
made by each major market to this growth.
6Table Of Content
1 Table of Contents1 Table of Contents 41.1
List of Tables 51.2 List of Figures 62
Introduction 72.1 Disease Introduction 72.2
Symptoms 82.3 Etiology 82.4 Pathophysiology
92.5 Diagnosis 92.6 Prognosis 102.7 Assessment
of Disease Severity 102.8 Treatment 103
Marketed Products 123.1 Overview 123.2 Product
Profiles 13 3.2.1 Pulmozyme (dornase alfa) -
Genentech 13
7Table Of Content
3.2.2 Bronchitol (mannitol) - Pharmaxis 143.2.3
TOBI (tobramycin) - Novartis 153.2.4 TOBI
Podhaler (tobramycin) - Novartis 163.2.5
Bramitob/Bethkis (tobramycin) - Chiesi
Farmaceutici 173.2.6 Colobreathe (colistimethate
sodium) - Forest Laboratories 173.2.7 Cayston
(aztreonam) - Gilead Sciences 183.2.8 Kalydeco
(ivacaftor) - Vertex Pharmaceuticals 193.2.9
Pancreatic Enzyme Replacement Therapies 203.2.10
Other Successful Products 213.3 Heat Map of
Marketed Products 213.4 Conclusion 234
Developmental Pipeline 254.1 Overview 254.2
Mechanisms of Action in the Pipeline 274.3
Clinical Trials 30 4.3.1 Failure Rate 304.3.2
Clinical Trial Duration 31
8Table Of Content
4.3.3 Clinical Trial Size 324.4 Promising
Pipeline Molecules 354.4.1 Lumacaftor (VX809) -
Vertex Pharmaceuticals 354.4.2 Ataluren (PTC124)
- PTC Therapeutics 374.4.3 Aeroquin
(levofloxacin) - Aptalis Pharma 384.4.4 Arikace
(amikacin liposomal) - Insmed 404.4.5 Other
Promising Molecules 414.5 Heat Map of Pipeline
Products 424.6 Conclusion 445 Market Forecast
to 2019 455.1 Geographical Markets 455.1.1
Global Markets 46 Get full TOC at
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sis-therapeutics-in-major-developed-to-2019-cftr-m
odulators-initiate-drive-towards-personalized-trea
tment-and-growth-market/table-of-contents
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