Will the Approval of New Agents Redefine the Cystic Fibrosis Market?
Decision Resources, Inc, April 2011, Pages: 38
The cystic fibrosis (CF) pipeline includes agents that target not only the symptoms of the disease but also the main cause of the disease - defective chloride transport. Interviewed key opinion leaders are eagerly waiting the approval of these agents; such approval is bound to change the treatment of CF. Agents that can prevent the onset of the disease rather than simply treat the associated respiratory and pulmonary complications of CF are likely to become first-line therapies.
Questions answered in this report:
- CF is an orphan disease, and, despite a relatively static incidence, we anticipate that the prevalence of this disease will rise. Why do we expect the prevalence to increase? What is the projected prevalence of CF over the next ten years?
- Several drugs with novel mechanisms of action are in development for CF. What are the new agents in development? How many target the CFTR gene? What do interviewed thought leaders see as the potential of these agents in the treatment of CF? What impact will these agents have on the current CF market?
- Approximately 85-90% of patients with CF have pancreatic damage significant enough to leave them unable to digest food and absorb nutrients. The FDA now imposes mandatory approval for pancreatic enzyme therapies for CF patients; only three such agents have received approval. Does the reduced number of approved agents affect the CF population? Which agents are currently approved? Which agents are still awaiting approval?
- In March 2010, the FDA approved Gilead Sciences’ Cayston to treat Pseudomonas aeruginosa infections in CF patients. What has been the uptake of Cayston in the CF market? What do interviewed thought leaders say about the use of Cayston? How often do they prescribe it to their patients? Do they prefer it over Novartis’s Tobi?
- In addition to frequent lung infections, most CF patients are treated for pancreatic insufficiency. What are the other complications of CF? What treatments are used for managing these complications?
- Vertex’s VX-770 and PTC Therapeutics’ Ataluren are the emerging agents for CF that are furthest in development. How promising are these two agents? Will their success change the dynamics of the CF market? How will they affect the treatment of CF?
Scope of the report:
- Markets covered: United States, France, Germany, Italy, Spain, United Kingdom, and Japan.
- Primary research: Interviews with thought-leading U.S. specialists with expertise in pulmonology, pediatrics, and CF who have worked closely with CF patients in CF centers.
- Epidemiology: Prevalent and incident cases of CF over a ten-year forecast period in the United States, France, Germany, Italy, Spain, the United Kingdom, and Japan.
- Exhibits: 8 tables and 5 figures.
- Market assessment: Major industry players in the CF market and considerations for drug developers.
- Emerging therapies: Phase II: 12 drugs; Phase III: 4 drugs; preregistration: 4 drugs. Extended coverage of select non-industry-sponsored trials.
- Benefits of orphan-drug status: Financial and marketing incentives associated with orphan-drug status.
Executive Summary
- Strategic Considerations
- Stakeholder Implications
- Introduction
Overview of CF
- Anatomy of the Lung
- Role of CFTR in the Development of CF
- Diagnosis of CF
- Symptoms and Complications
- Pulmonary Symptoms/Complications
- Pancreatic Symptoms/Complications
Epidemiology and Patient Populations
- Overview
- Disease Definition
- Methodology
- Incidence
- Prevalence
Current Treatments
- Inhaled Mucolytics
- Roche’s Pulmozyme
- Hypertonic Saline
- Inhaled Antibiotics
- Novartis’s Tobi
- Gilead Sciences’ Cayston
- Macrolide Antibiotics
- Pfizer’s Zithromax
- Pancreatic Enzyme Replacements
Emerging Therapies
- Vertex Pharmaceuticals’ VX-770 and VX-809
- PTC Therapeutics’ Ataluren (PTC-124)
- Pharmaxis’s Bronchitol
- Emerging Antibiotics
- Novartis’s Tobramycin Inhaled Powder (TIP)
- Mpex Pharmaceuticals’ Aeroquin (MP-376)
- Other Agents
Outlook for CF: Strategies and Considerations for Drug Makers
- 2010 U.S. Market Estimates
- Unmet Needs and Potential for New Agents to Enter the CF Market
- Emerging Dynamics in the CF Market and Effect on Established Treatments
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
- European Medicines Agency
- Private Organization Incentives
Appendix B: Bibliography
About the Authors
- Abbott
- Aerovance
- AstraZeneca
- Axcan Pharmaceuticals
- Bayer Schering Pharma
- Copernicus Therapeutics
- Digestive Care
- Discovery Laboratories
- Eli Lilly
- EmphyCorp
- Eurand Pharmaceuticals
- Forest Laboratories
- Gilead Sciences
- GlaxoSmithKline
- Insmed
- Johnson & Johnson
- Kamada
- Mpex Pharmaceuticals
- Novartis
- Pari Pharmaceuticals
- Parion Sciences
- Pfizer
- Pharmaxis
- PTC Therapeutics
- Roche
- Vectura Group
- Vertex Pharmaceuticals
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