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Cystic Fibrosis: Will New Agents Emerge as First-Line Therapies?


Description: The cystic fibrosis (CF) market is poised for significant change. The next generation of CF treatments all target the central mechanism responsible for organ damage in CF. These agents, as one thought leader states, “represent the closest we have come to a cure for CF” and will almost certainly bring about a change in the treatment algorithm of the disease. As a result, new market leaders will emerge, and current first-line therapies will find themselves relegated to second- or third-line options.

Get the Answers You Need to Shape Your Strategy

Physicians use a variety of medications and non-pharmacological treatments to address the needs of:

-CF patients. What are the strengths and weaknesses of these treatments? Which work best in specific subpopulations? Do thought leaders believe these treatments will still have a role in the treatment algorithm over the next five to ten years?

-Several drugs with trusted and novel mechanisms of action are in development for CF.
What new agents are in development? What are thought leaders’ opinions of the potential of these agents in the treatment of CF? What impact will these agents have on treatment algorithms and the CF market?

-Emerging agents target the central mechanism responsible for organ damage in CF and have the potential to be disease modifying rather than just treating symptoms. What unmet needs remain in drug development? Will the formulations of these emerging therapies improve patient compliance? What more is required to effectively treat the paediatric subpopulation?

Overview of - CF: disease development, symptoms, complications, and methods for diagnosis.

- Epidemiology: disease defi nition; incidence by race; growth in the number of prevalent cases.

- Current therapies: mucolytics, antibiotics, pancreatic enzyme replacement therapy, as well as thought leaders’ opinions on where these agents fi t into the treatment algorithm.

- Emerging therapies: strengths and weaknesses of agents in development; novel mechanisms of action; an analysis of market position for these agents.

- CF market: details on the 2007 market and prescribing trends.

- Outlook: the impact of novel agents on the CF market.

- Orphan Disease Series: As competition increases in larger disease states, companies are turning to orphan diseases that offer substantial pricing advantages and lower regulatory hurdles because of high unmet need. The Orphan Disease Series provides critical market information, including disease prevalence, profi les of marketed and emerging agents, and insight into the prescribing choices of disease specialists for diseases with smaller patient populations. These reports will help you to maximize the potential of your agents in these challenging markets, including the regulatory
benefi ts available and the potential for partnership with charitable organizations.


Contents: Executive Summary
Strategic Considerations
Stakeholder Implications
Introduction
Overview of Cystic Fibrosis
Anatomy of the Lung
The Role of CFTR in the Development of Cystic Fibrosis
Diagnosis of Cystic Fibrosis
Symptoms and Complications
Pulmonary Symptoms/Complications Pancreatic Symptoms/Complications
Epidemiology and Patient Populations
Overview
Disease Defi nition
Current Treatments
Nebulized Mucolytics
Genentech/Roche’s Pulmozyme
Hypertonic Saline
Comparison of Pulmozyme and Hypertonic Saline
Inhaled Antibiotics
Pfi zer’s Zithromax
Pancreatic Enzyme Replacement Therapy
Emerging Therapies
PTC Therapeutics’ PTC124
Vertex Pharmaceuticals’ VX770
Inspire Pharmaceuticals’ Denufosol Tetrasodium
Gilead’s Cayston (Inhaled Aztreonam)
Novartis/Nektar’s Tobramycin Inhaled Powder
New Formulations of Pancreatic Enzymes
2007 Cystic Fibrosis Market and Outlook for Emerging Therapies
Appendix A Incentive Programs for the Research, Development, and Marketing of
Orphan Drugs
FDA
Expanded Market Exclusivity
Tax Credits
Support During the Drug Approval Process
Grants
EMEA
Private Organization Incentives
Appendix B Epidemiology Methodology
Incidence
Prevalence
Appendix C Bibliography—Cystic Fibrosis
Appendix D Experts Interviewed—Cystic Fibrosis

Tables
ES1 Thought Leaders’ Opinions on Cystic Fibrosis Issues
1 Classifi cations for Cystic Fibrosis Mutations
2 Symptoms and Complications of Cystic Fibrosis
3 Common Treatments for Cystic Fibrosis
4 Emerging Treatments for Cystic Fibrosis
5 Ongoing Cystic Fibrosis Clinical Trials of Launched Agents
6 US Sales of Leading Agents to Treat the Pulmonary Complications of Cystic
Fibrosis, 2007
B1 Studies Reviewed But Not Used for the Epidemiological Analyses

Figures
1 Anatomy of the Lung
2 Total Incident Cases of Cystic Fibrosis in Live Births in the United States, 2007,
2012, and 2017
3 Sales Growth of MarketLeading Cystic Fibrosis Agents and Total Cystic Fibrosis
Market, 20002007
A1 Number of Drugs Receiving FDA OrphanDrug Designation and Approval, 19832007
A2 Growth in the Number of Orphan Drugs Available, 19832007


Companies Mentioned - Abbott - Aerovance - Altus Pharmaceuticals - Axcan - Bayer - Cellular Sciences - Copernicus Therapeutics - Eurand - Genentech - Gilead - GlaxoSmithKline - Immunsystem - Inspire Pharmaceuticals - Johnson & Johnson - Lantbio - Nektar - Novartis - Novo Nordisk - Parion Sciences - Pfi zer - PTC Therapeutics - Roche - Solvay - Sucampo Pharmaceuticals - Vertex Pharmaceuticals


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