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

Published by: Decision Resources

Published: Jun. 20, 2008 - 39 Pages


Table of 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 Definition

Current Treatments

Nebulized Mucolytics

Genentech/Roche’s Pulmozyme

Hypertonic Saline

Comparison of Pulmozyme and Hypertonic Saline

Inhaled Antibiotics

Pfizer’s Zithromax

Pancreatic Enzyme Replacement Therapy

Emerging Therapies

PTC Therapeutics’ PTC-124

Vertex Pharmaceuticals’ VX-770

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. Classifications 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. U.S. 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 Market-Leading Cystic Fibrosis Agents and Total Cystic Fibrosis Market, 2000-2007

A1. Number of Drugs Receiving FDA Orphan-Drug Designation and Approval, 1983-2007

A2. Growth in the Number of Orphan Drugs Available, 1983-2007

Abstract

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 nonpharmacological 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 pediatric subpopulation?
Scope
  • Overview of CF: disease development, symptoms, complications, and methods for diagnosis.
  • Epidemiology: disease definition; 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 fit 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, profiles 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 benefits available and the potential for partnership with charitable organizations.


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