Orphan Drugs Pipeline Analysis: Wider Incentives Encourage Orphan Drug R&D
Decision Resources, Inc, July 2005, Pages: 107
This report examines the pipeline of drugs in development under the guidance of orphan drug legislation in the major markets of the United States, Europe, and Japan. Each of these markets has orphan drug legislation with different provisions, and we compare and contrastthese regulatory factors. The contrasting requirements and different time frames in which this legislation has been enacted has also resulted in considerable differences in the numbers of designated and approved orphan drugs within each market.
More than 6,000 rare diseases could potentially be designated as eligible for development of orphan drugs. However, this report focuses on diseases that have attracted the most attention from orphan drug developers—namely, genetic diseases, uncommon autoimmune diseases, and cancer. We also discuss which cancers are potentially eligible for gaining orphan drug designation. Given the large number of orphan drugs targeting various cancers, we devote an entire section of this report solely to cancer therapies. We group other orphan drugs in this report according to their primary mode of action: enzymes and enzyme inhibitors, hormones, gene therapies, gene modulators, monoclonal antibodies, and receptor and ion channel modulators.
We then profile companies that are active in the development of orphan drugs. We separately profile the small number of orphan drug specialist companies and the larger number of biotechnology companies for whom orphan drug development constitutes a significant proportion of their current pipeline. Finally, we discuss how the orphan drug market is likely to evolve over the next ten years by considering the influence of such factors as patent issues, pharmacogenomics, and the reimbursement environment.
Why Buy This Report?
- Learn what the leading players in the orphan drug market are doing via detailed analyses of their pipelines.
- Gain valuable insight regarding emerging companies and the agents in their orphan drug pipelines.
- Explore key trends in the orphan drug market and their potential impact on the commercial outlook for this market.
- Assess marketed and pipeline drugs with respect to development phase and peak-year sales potential.
- Analyze orphan drug alliance activity presented in comprehensive company profiles and evaluate company financial information
Executive Summary
Which companies are the major players in the orphan drug market?
Which companies are the emerging players in the orphan drug market?
Why seek orphan designation?
How has orphan drug legislation affected the pharmaceutical market?
Which indications represent the best opportunities for developers of orphan drugs?
What is the commercial outlook for the orphan drug market?
Chapter 1. Introduction .
Overview
Scope of This Report
Chapter 2. Regulatory Factors .
United States
Europe
Japan
Chapter 3. Orphan Drug Indications .
Genetic Diseases
Cystic Fibrosis
Alpha-1 Antitrypsin Deficiency
Sickle Cell Anemia
Hereditary Angioedema
Phenylketonuria
Lysosomal Storage Disorders
Gaucher’s Disease
Fabry’s Disease.
Other Lysosomal Storage Disorders
Autoimmune Diseases
Multiple Sclerosis
Myasthenia Gravis
Cancer
Prevalence of Cancers
Cancers Eligible for Orphan Drug Designation
Chapter 4. Classes of Orphan Drugs .
Oncology Drugs: Small-Molecule Therapeutics, Natural Products,Recombinant Proteins
Marketed Agents
Emerging Agents
Leukemia and Myeloma
Melanoma
Glioma
Renal Cell Carcinoma
Pancreatic Cancer
Other Cancers
Monoclonal Antibodies
Marketed Products
Emerging Agents
Oncology Indications
Other Indications
Enzymes and Enzyme Inhibitors
Hormones
Gene Therapy
Gene Modulators
Receptor and Ion-Channel Modulators
Chapter 5. Pipeline Analysis of Companies Developing Orphan Drugs .
Biotechnology Companies
Amicus Therapeutics
Select Alliances in the Orphan Drugs Area
Financial Details
Ark Therapeutics
Select Alliances in the Orphan Drugs Area
Financial Details
BioMarin Pharmaceutical
Select Alliances in the Orphan Drugs Area
Financial Details
Genzyme
Select Alliances in the Orphan Drugs Area
Financial Details
Seattle Genetics
Select Alliances in the Orphan Drugs Area
Financial Details
Sigma Tau
Select Alliances in the Orphan Drugs Area
Financial Details
Transkaryotic Therapies
Select Alliances in the Orphan Drugs Area
Financial Details
ZyStor Therapeutics
Select Alliances in the Orphan Drugs Area
Financial Details
Orphan Drug Specialist Companies
Orphan Medical
Select Alliances in the Orphan Drugs Area
Financial Details
Rare Disease Therapeutics
Significant Alliances
Financial Details
AOP Orphan Pharmaceuticals
Select Alliances in the Orphan Drugs Area
Financial Details
Orphan Europe
Select Alliances in the Orphan Drugs Area
Financial Details
Orphan Pharma International
Significant Alliances
Financial Details
Swedish Orphan International
Select Alliances in the Orphan Drugs Area
Financial Details
Chapter 6. Outlook for Orphan Drugs .
Patent and Exclusivity Issues
Challenging Indications (Cancer)
Pharmacogenomics
Big Pharma Strategy
Reimbursement and Legislation
Commercial Outlook
Bibliography
Tables and Figures
Table 1. Comparison of Orphan Drug Legislation in the United States, the
European Union, and Japan
Table 2. Prevalent Cases of Multiple Sclerosis, 2003-2013
Table 3. Prevalent Cases of Cancers Not Eligible for Orphan Drug Status in the Major
Pharmaceutical Markets, 2003-2013
Table 4. Prevalent Populations of Cancers Eligible for Orphan Drug Status in the Major Pharmaceutical Markets, 2003-2013
Table 5. Marketed Anticancer Agents with Orphan Drug Status in the United States
and/or Europe, 2005
Table 6. Anticancer Agents in Development with Orphan Drug Status in the United
States and/or Europe, 2005
Table 7. Marketed Monoclonal Antibodies with Orphan Drug Status in the United
States and/or Europe, 2005
Table 8. Monoclonal Antibodies in Development with Orphan Drug Status in the
United States and/or Europe, 2005
Table 9. Marketed Enzymes and Enzyme Inhibitors with Orphan Drug Status in the
United States and/or Europe, 2005
Table 10. Enzymes and Enzyme Inhibitors in Development with Orphan Drug Status
in the United States and/or Europe, 2005
Table 11. Marketed Hormone Products with Orphan Drug Status in the United States
and/or Europe
Table 12. Gene Therapeutics in Development with Orphan Drug Status in the
United States and/or Europe, 2005
Table 13. Gene Modulators with Orphan Drug Status in the United States and/or
Europe, 2005
Table 14. Marketed Receptor and Ion-Channel Modulators with Orphan Drug
Status in the United States and/or Europe, 2005
Table 15. Receptor and Ion-Channel Modulators in Development with Orphan
Drug Status in the United States and/or Europe, 2005
Table 16. Companies with Multiple Orphan Drug Designations
Table 17. Pipeline of Orphan Drugs—Select Companies
Table 18. Pipeline of Development Compounds—Ark Therapeutics
Table 19. Ark Therapeutics’ Alliances in the Orphan Drug Area
Table 20. Pipeline of Orphan Drugs—BioMarin
Table 21. BioMarin Pharmaceutical’s Alliances in the Orphan Drug Area
Table 22. BioMarin’s Financial Information, 2000-2004
Table 23. Pipeline of Orphan Drugs—Genzyme
Table 24. Genzyme’s Alliances in the Orphan Drug Area
Table 25. Pipeline of Orphan Drugs—Seattle Genetics
Table 26. Seattle Genetics’ Alliances in the Orphan Drug Area
Table 27. Seattle Genetics’ Financial Information, 2000-2004
Table 28. Pipeline of Orphan Drugs—Sigma Tau
Table 29. Pipeline of Orphan Drugs—Transkaryotic Therapies
Table 30. Transkaryotic Therapies’ Alliances in the Orphan Drug Area
Table 31. Transkaryotic Therapies’ Financial Information, 2000-2004
Table 32. Pipeline of Orphan Drugs—Orphan Medical
Table 33. Orphan Medical’s Alliances in the Orphan Drug Area
Table 34. Orphan Medical’s Financial Information, 2000-2004
Table 35. Pipeline of Orphan Drugs—Rare Disease Therapeutics
Table 36. AOP Orphan Pharmaceutical’s Alliances in the Orphan Drug Area
Table 37. Pipeline of Orphan Drugs—Orphan Europe
Table 38. Pipeline of Orphan Drugs—OPi
Table 39. OPi’s Alliances in the Orphan Drug Area
Table 40. Pipeline of Orphan Drugs—Swedish Orphan
Figure 1. Worldwide Sales of Leading Orphan Drugs, 2004
Figure 2. Blockage of Capillaries by Abnormally Shaped Sickle Cells
Figure 3. Clinical Features of MPS I
Figure 4. Multiple Sclerosis Immune Pathophysiology
Figure 5. Genzyme’s Financial Information, 2000-2004
Which companies are the major players in the orphan drug market?
The orphan drug market represents a highly fragmented market spanning many indications. The major players in these markets—based on total sales from drugs with orphan drug status—are also the leading biotechnology companies: Amgen, Biogen Idec, and Genentech. None of these companies, however, is strategically organized around the goal of exploiting orphan drug markets. This report focuses on the companies for which targeting orphan diseases is a major strategic aim. Genzyme is the largest and most established among these companies, focusing its research on enzyme replacement therapies for certain rare genetic diseases. We have also identified eight smaller companies specializing in orphan drug development
Why seek orphan designation?
Orphan drug status provides a number of advantages. Most significantly, it provides tax incentives for the developer and, for an approved product, a period of market exclusivity against similar competing products. Thus, Biogen Idec was able to delay the U.S. market entry of Serono’s interferon product Rebif because of the orphan status of Biogen Idec’s interferon product, Avonex. The race between Genzyme and Transkaryotic Therapies for the first U.S. approval of an enzyme replacement therapy for Fabry’s disease resulted in Genzyme’s agalsidase (Fabrazyme) earning first approval, thereby gaining seven years exclusivity at the expense of Transkaryotic’s agalsidase alfa (Replagal).
How has orphan drug legislation affected the pharmaceutical market?
The introduction of orphan drug legislation has provided a distinct regulatory framework for the introduction of such agents and has provided financial incentives to encourage their development. At the time of this writing, the FDA had recorded a total of 1,455 orphan drug designations. Europe and Japan have granted 256 and 263 such designations, respectively. Despite differences in the incentives offered by each major market’s orphan drug legislation, the advantages of market exclusivity, tax credits, and rapid regulatory review combine to increase the financial attractiveness of unprofitable low-prevalence diseases.
Although orphan drugs still represent a niche market that larger pharmaceutical companies enter on only a limited basis, orphan drug legislation has enabled biotechnology and specialty pharmaceutical companies to focus on the development of these drugs as a viable new business model.
Which indications represent the best opportunities for developers of orphan drugs?
The diseases for which orphan drugs are currently marketed and/or in development are grouped into three therapeutic categories: genetic diseases, including cystic fibrosis and lysosomal storage disorders; autoimmune diseases, including multiple sclerosis and myasthenia gravis; and cancer.
One issue that must be considered by prospective developers of an orphan drug is growing disease prevalence. Each major markets’ orphan drug legislation sets prevalence criteria that determine which indications are appropriate for orphan drug development. Indications with rapidly expanding prevalence, such as malignant melanoma and bladder cancer, may soon exceed these prevalence criteria.
What is the commercial outlook for the orphan drug market?
The commercial outlook for the orphan drug market is promising. In 2004, nine products with orphan drug status generated annual sales in excess of $1 billion. The fact that orphan drugs are typically developed for serious or life-threatening diseases confers pricing power substantially greater than is possible for many other indications. Increased awareness of the potential advantages of orphan drug status, the opportunity to develop orphan drugs in all major markets, and the increasing number of marketed recombinant products will all enhance the growth of the orphan drug market in the near term.
In the long term, the application of pharmacogenomic approaches should provide enhanced opportunities to develop orphan drugs because they will provide a scientific basis for market segmentation and, in turn, further stimulate the growth of the orphan drug market.
- Orphan Medical
- Rare Therapeutics
- Orphan Pharma International
- Orphan Europe,
- AOP Orphan Pharmaceuticals,
- Swedish Orphan
- Orphan Australia
- Genzyme
- Transkaryotic Therapeutics,
- Sigma Tau, Amicus Therapeutics,
- Ark Therapeutics
- BioMarin Pharmaceutical
- Seattle Genetics
- ZyStor Therapeutics.
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