Orphan Drugs for Cancer: R&D and Market 2013-2023
Orphan cancer drugs - new study shows you trends, R&D, and sales forecasts
What's the future of orphan medicines to treat cancers? Visiongain's new report gives you revenue predictions from 2013. There you explore results, R&D, opportunities, and potential sales.
Orphan drugs can be profitable. In our analysis you see forecasted sales to 2023 at overall world market, submarket, product, and national level. You see the potential of treatments for rare cancers. Try our report now, helping you stay ahead.
Trends and revenue potentials of that rising pharma segment
Many opportunities remain for orphan medicines in cancer care. In our new study you assess treatments and their potential returns. You also investigate what affects their sales.
Read on, then, to explore that industry and see what its future market could be worth.
Forecasts 2013-2023 and other analyses - find commercial opportunities
Besides revenue forecasting to 2023, our new work shows historical data, growth rates, and market shares. There you discover original analysis, seeing business outlooks and developments. You also get 64 tables, 52 charts, and two interviews with companies.
It can be hard to find data on rare diseases - all that searching. But now you can stay ahead in knowledge for applied oncology, benefiting your research, analyses, and decisions.
Finding information you need on rare cancer indications just got easier. You save time too. In our study you hear what's going on, also seeing where needs and money lie.
Avoid falling behind then. The following sections show what you get in our new investigation.
Prospects from 2013 for the world market and submarkets
Our new report shows revenue to 2023 for the overall world market. It also shows you individual revenue forecasts to 2023 for five orphan cancer submarkets:
What's the secret of making money there? Our study gives you a feel, then, for what stimulates and restrains that industry and market. See what's possible and likely.
Find sales predictions by product, too, hearing how they can succeed.
Forecasts from 2013 for orphan anticancer brands
How will orphan cancer drugs perform to 2023 at world level? Our report forecasts revenues of 12 leading drugs and six recently-approved products, including these brands:
Our study also divides its overall world forecast into those for geographical regions.
National markets - what demand for orphan anticancer agents?
From 2013 to 2023 the US, the EU, and Japan offer the greatest opportunity for companies developing and producing therapies for rare cancer indications.
Our analyses show you individual revenue forecasts to 2023 for 11 national markets:
Research and development - you assess trends, possibilities, and innovations
What about R&D - pipelines for new drugs? You assess trends and technologies there, especially in these applications:
You hear, then, about innovations in oncological R&D. Many molecular pathways and targets exist. Discover progress and find what it means. Our study explains, discussing issues to help your work.
Forces affecting developers, producers, and sellers
Our report shows you issues and events affecting the orphan cancer medicines industry and market from 2013, including these:
Companies and 2017 market value - what happens next?
From 2013, new treatments hold great potential for investments, medical advances and revenues. Our analysis predicts the world market for orphan cancer drugs will reach $50.3bn in 2017, with high growth from 2013 to 2023.
See what's possible there - the expected gains. Our report shows what technologies and players hold greatest potential. You explore activities of these companies and many others:
Information found nowhere else
In particular, then, our investigation gives you these advantages:
With that report you are less likely to fall behind in knowledge or miss sales opportunity. See there how you could benefit your research, analysis, and decisions. Explore progress and possibilities. Also find how you can save time and get recognition for insight.
Orphan anticancer drugs - assess potentials now, seeing what you can gain
Our new report is for everyone analysing biopharma and orphan diseases. Find sales predications with discussions. Avoid missing out then - please order the report now.
What's the future of orphan medicines to treat cancers? Visiongain's new report gives you revenue predictions from 2013. There you explore results, R&D, opportunities, and potential sales.
Orphan drugs can be profitable. In our analysis you see forecasted sales to 2023 at overall world market, submarket, product, and national level. You see the potential of treatments for rare cancers. Try our report now, helping you stay ahead.
Trends and revenue potentials of that rising pharma segment
Many opportunities remain for orphan medicines in cancer care. In our new study you assess treatments and their potential returns. You also investigate what affects their sales.
Read on, then, to explore that industry and see what its future market could be worth.
Forecasts 2013-2023 and other analyses - find commercial opportunities
Besides revenue forecasting to 2023, our new work shows historical data, growth rates, and market shares. There you discover original analysis, seeing business outlooks and developments. You also get 64 tables, 52 charts, and two interviews with companies.
It can be hard to find data on rare diseases - all that searching. But now you can stay ahead in knowledge for applied oncology, benefiting your research, analyses, and decisions.
Finding information you need on rare cancer indications just got easier. You save time too. In our study you hear what's going on, also seeing where needs and money lie.
Avoid falling behind then. The following sections show what you get in our new investigation.
Prospects from 2013 for the world market and submarkets
Our new report shows revenue to 2023 for the overall world market. It also shows you individual revenue forecasts to 2023 for five orphan cancer submarkets:
- Leukaemia
- Multiple myeloma
- Lymphoma
- Glioma
- Other disorders.
What's the secret of making money there? Our study gives you a feel, then, for what stimulates and restrains that industry and market. See what's possible and likely.
Find sales predictions by product, too, hearing how they can succeed.
Forecasts from 2013 for orphan anticancer brands
How will orphan cancer drugs perform to 2023 at world level? Our report forecasts revenues of 12 leading drugs and six recently-approved products, including these brands:
- Rituxan
- Glivec
- Revlimid
- Velcade
- Cometriq
- Stivarga.
Our study also divides its overall world forecast into those for geographical regions.
National markets - what demand for orphan anticancer agents?
From 2013 to 2023 the US, the EU, and Japan offer the greatest opportunity for companies developing and producing therapies for rare cancer indications.
Our analyses show you individual revenue forecasts to 2023 for 11 national markets:
- US
- Japan
- Germany, France, UK, Italy, and Spain (EU5), also with EU regional prediction
- Brazil, Russia, India, and China (BRIC).
Research and development - you assess trends, possibilities, and innovations
What about R&D - pipelines for new drugs? You assess trends and technologies there, especially in these applications:
- Leukaemia
- Myelodysplastic syndromes (MDS)
- Lymphoma
- Multiple myeloma
- Glioma.
You hear, then, about innovations in oncological R&D. Many molecular pathways and targets exist. Discover progress and find what it means. Our study explains, discussing issues to help your work.
Forces affecting developers, producers, and sellers
Our report shows you issues and events affecting the orphan cancer medicines industry and market from 2013, including these:
- Regulations - regional and national
- Antibodies, toxin fragments, and other novel immunotherapies
- Potential of tyrosine kinase inhibitors
- Therapeutic vaccines
- Expansions of indications.
- Costs of therapy and issues for regulators, governments, and other payers
- Biosimilars and generics as competition for original drugs
- Challenges and opportunities in patient recruitment
- Companion diagnostics and personalised therapies.
Companies and 2017 market value - what happens next?
From 2013, new treatments hold great potential for investments, medical advances and revenues. Our analysis predicts the world market for orphan cancer drugs will reach $50.3bn in 2017, with high growth from 2013 to 2023.
See what's possible there - the expected gains. Our report shows what technologies and players hold greatest potential. You explore activities of these companies and many others:
- Novartis
- Celgene
- Onyx Pharmaceuticals
- Bristol-Myers Squibb
- Johnson & Johnson
- Merck & Co.
Information found nowhere else
In particular, then, our investigation gives you these advantages:
- Revenues to 2023 for orphan cancer drugs at world level, with forecasting of 5 submarkets and 18 products - assess potentials for investments and sales
- Forecasts to 2023 for 11 national markets in the Americas, Europe, and Asia - investigate countries for revenues and expected growth
- Prospects for established competitors and new entrants - explore activities, results, R&D, and outlooks for future success.
With that report you are less likely to fall behind in knowledge or miss sales opportunity. See there how you could benefit your research, analysis, and decisions. Explore progress and possibilities. Also find how you can save time and get recognition for insight.
Orphan anticancer drugs - assess potentials now, seeing what you can gain
Our new report is for everyone analysing biopharma and orphan diseases. Find sales predications with discussions. Avoid missing out then - please order the report now.
1. EXECUTIVE SUMMARY
1.1 Orphan Drugs for Cancer: Market Overview 2013
1.2 Report Contents
1.3 Research and Analysis Methods
2. INTRODUCTION TO ORPHAN DRUGS IN CANCER
2.1 Defining Orphan Diseases
2.2 What Are the Major Orphan Cancer Indications?
2.2.1 Leukaemia: The Most Common Blood Cancer
2.2.2 Lymphoma
2.2.3 Multiple Myeloma
2.2.4 Other Orphan Cancer Indications
2.3 Orphan Drug Designation
2.3.1 What Benefits Come with Orphan Drug Designation?
2.4 Demand for Orphan Drugs
2.4.1 Development of Orphan Drugs
3. THE WORLD ORPHAN CANCER DRUGS MARKET 2013-2023
3.1 How Large is the Orphan Drugs Market in 2013?
3.1.1 Cancer is the Largest Sector of the Orphan Drugs Market
3.2 The Orphan Cancer Drugs Market 2013-2023
3.2.1 What Will Drive Growth in the Market to 2023?
3.2.2 Market Restraints 2013-2023
3.3 Orphan Drugs in Leukaemia: Submarket 2013-2023
3.3.1 Leukaemia: Submarket Forecast 2013-2023
3.3.2 Next Generation Tyrosine Kinase Inhibitors to Drive Growth
3.4 Orphan Drugs in Lymphoma: Submarket 2013-2023
3.4.1 Lymphoma: Submarket Forecast 2013-2023
3.4.2 Monoclonal Antibody Therapies Will Dominate the Submarket 2013-2023
3.5 Orphan Drugs in Multiple Myeloma: Submarket 2013-2023
3.5.1 Multiple Myeloma: Submarket Forecast 2013-2023
3.5.2 There Are Many New Therapies in Development
3.6 Orphan Drugs in Glioma: Submarket 2013-2023
3.6.1 Glioma: Submarket Forecast 2013-2023
3.6.2 Therapeutic Vaccines Will Provide a Novel Treatment Strategy 2016-2023
4. LEADING AND EMERGING NATIONAL MARKETS FOR RARE CANCER INDICATIONS 2013-2023
4.1 Leading National Markets 2013
4.1.1 National Market Forecasts 2013-2023
4.2 The US: The Leading Market for Orphan Cancer Drugs 2013-2023
4.2.1 Orphan Drug Regulations in the US
4.2.2 Benefits for Orphan Drug Developers
4.2.3 US Orphan Cancer Drugs Market Forecast 2013-2023
4.3 Orphan Drugs in the EU 2013-2023
4.3.1 The Incentives for Orphan Drug Development in the EU
4.3.2 The EU Orphan Cancer Drugs Market Forecast 2013-2023
4.3.3 Orphan Cancer Drugs in the EU5 Submarkets 2013-2023
4.4 The Japanese Orphan Cancer Drugs Market 2013-2023
4.4.1 Orphan Drug Regulations and Benefits
4.4.2 Japanese Orphan Cancer Drugs Submarket Forecast 2013-2023
4.5 The Potential for Orphan Cancer Drugs in Emerging Markets 2013-2023
4.5.1 Brazil
4.5.2 Russia: Attempts to Introduce Orphan Designation and Incentives 2010-2013
4.5.3 India: Generics Will Limit Branded Rare Cancer Drug Uptake
4.5.4 China
5. OUTLOOK FOR LEADING ORPHAN DRUGS 2013-2023
5.1 Rituxan (Roche/Biogen Idec) Is the Bestselling Orphan Cancer Drug
5.1.1 Roche is Developing a Next Generation Rituxan
5.1.2 Rituxan: Revenue Forecast 2013-2023
5.2 Glivec (Novartis): A Leukaemia Market Leader
5.2.1 The Challenge of Tasigna and Generics
5.2.2 Glivec: Revenue Forecast 2013-2023
5.3 Revlimid (Celgene) Is the Leading Therapy for Multiple Myeloma
5.3.1 Celgene Is Adding New Indications for Revlimid
5.3.2 Revlimid: Revenue Forecast 2013-2023
5.4 Alimta (Eli Lilly)
5.4.1 Revenue Growth Driven by Uptake in NSCLC 2008-2012
5.4.2 Alimta: Revenue Forecast 2013-2023
5.5 Velcade (J&J/Takeda)
5.5.1 Strong Revenue Growth in Markets Worldwide 2008-2012
5.5.2 Velcade Faces Significant Competition in Multiple Myeloma 2013-2023
5.6 Nexavar (Bayer/Onyx Pharmaceuticals)
5.6.1 Nexavar Has Shown Promise in Thyroid Cancer
5.6.2 Nexavar: Revenue Forecast 2013-2023
5.7 Tasigna (Novartis)
5.7.1 Tasigna Is More Effective than Glivec and Sprycel
5.7.2 Will Tasigna Replace Glivec 2013-2023?
5.8 Temodar (Merck & Co.)
5.8.1 Temodar Competes with Generics in the EU
5.8.2 Temodar: Continued Revenue Decline 2013-2023
5.9 Vidaza (Celgene/Nippon Shinyaku)
5.9.1 Vidaza: Revenue Growth 2008-2012
5.9.2 Celgene Is Developing an Oral Azacitidine Formulation
5.9.3 Vidaza Revenue Forecast 2013-2023: When Will Generic Competition Arrive?
5.10 Sprycel (Bristol-Myers Squibb/Otsuka Pharmaceutical)
5.10.1 Sprycel: Financial Performance 2008-2012
5.10.2 Sprycel: Revenue Forecast 2013-2023
5.11 Afinitor (Novartis)
5.11.1 Approval in Breast Cancer Drives Revenue Growth 2012
5.11.2 Afinitor: Revenue Forecast
5.12 Yervoy
5.12.1 Yervoy May Be Effective as a Combination Therapy
5.12.2 Yervoy: Revenue Forecast 2013-2023
5.13 Outlook for Orphan Cancer Drugs Approved in 2012 and 2013
5.13.1 New Therapies for Multiple Myeloma
5.13.1.1 Pomalyst: A Next Generation Thalidomide Derivative
5.13.1.2 Kyprolis (Onyx Pharmaceuticals)
5.13.1.3 Pomalyst versus Kyprolis
5.13.1.4 Pomalyst and Kyprolis: Revenue Forecast 2013-2023
5.13.2 Three New Drugs Approved for CML in 2012
5.13.2.1 Bosulif (Pfizer): A TKI Targeting New Mutations
5.13.2.2 Iclusig (ARIAD Pharmaceuticals)
5.13.2.3 Bosulif and Iclusig: Revenue Forecast 2013-2023
5.13.2.4 Synribo (Teva)
5.13.3 Commercial Prospects for Marqibo (Talon Therapeutics)
5.13.4 Cometriq (Exelixis)
5.13.4.1 Cometriq: Revenue Forecast 2013-2023
5.13.5 Stivarga (Bayer/Onyx Pharmaceuticals): Opportunities in Stomach and Liver Cancer
5.13.5.1 Stivarga: Revenue Forecast 2013-2023
5.14 Other Leading Cancer Therapies Approved for Orphan Indications 2012
5.14.1 Avastin (Roche)
5.14.1.1 Avastin Shows Promise in Glioblastoma
5.14.2 Erbitux (Bristol-Myers Squibb/Eli Lilly/Merck Serono)
5.14.2.1 Attempts to Expand Approved Indications
5.14.3 Herceptin (Roche)
6. ORPHAN CANCER DRUGS: R&D PIPELINE 2013
6.1 Orphan Drugs in the Leukaemia Pipeline 2013
6.1.1 Obinutuzumab (Roche)
6.1.2 Dacogen: Eisai Seeks Approval for AML in the US
6.1.3 CPX-351 (Celator Pharmaceuticals)
6.1.4 Dinaciclib (Merck & Co.)
6.1.5 EZN-2285 (Sigma-Tau Pharmaceuticals): Reformulation for Improved Dosing
6.1.6 Ibrutinib (J&J/Pharmacyclics): Awarded Breakthrough Status in Multiple Indications
6.1.7 Midostaurin (Novartis): Targeting the FLT3 Gene
6.1.8 Moxetumomab Pasudotox (MedImmune): Combining Antibodies and Toxin Fragments
6.1.9 Vosaroxin (Sunesis Pharmaceuticals)
6.1.10 Alvocidib (Tolero Pharmaceuticals)
6.1.11 Blinatumomab (Amgen): Complete Response in Two Thirds of Patients
6.1.12 CNDO-109 (Coronado Biosciences)
6.1.13 Tosedostat (Chroma Therapeutics/Cell Therapeutics)
6.1.14 TRU-016 (Emergent BioSolutions)
6.1.15 Veltuzumab (Immunomedics)
6.2 Orphan Drugs in the MDS Pipeline 2013
6.2.1 Rigosertib (Onconova Therapeutics/Baxter)
6.2.2 Sapacitabine (Cyclacel Pharmaceuticals)
6.2.3 StemEx (Gamida Cell): A Regenerative Medicine Approach to Treating Cancer
6.2.4 Pacritinib (Cell Therapeutics): Approval Possible in 2016
6.2.5 Telintra (Telik)
6.3 Orphan Drugs in the Lymphoma Pipeline 2013
6.3.1 Mechlorethamine Gel (Ceptaris Therapeutics): Fast and Accurate Dosing for Patients
6.3.2 BiovaxID (BioVest International): A Therapeutic Vaccine for Follicular Lymphoma
6.3.3 Belinostat (Topotarget/Spectrum Pharmaceuticals)
6.3.4 Epratuzumab (Immunomedics)
6.3.5 Faridak (Novartis): Approval in Myeloma in 2014?
6.3.6 Mogamulizumab (Kyowa Hakko Kirin/Amgen)
6.3.7 Darinaparsin (ZIOPHARM Oncology)
6.3.8 Mocetinostat: MethylGene Is Seeking a Development Partner
6.3.9 Resminostat (4SC)
6.4 Orphan Drugs in the Multiple Myeloma Pipeline 2013
6.4.1 Aplidin (PharmaMar)
6.4.2 Elotuzumab (AbbVie/Bristol-Myers Squibb)
6.4.3 Ixazomib (Millennium Pharmaceuticals): An Oral Proteasome Inhibitor to Replace Velcade?
6.4.4 Captisol-Enabled Melphalan (Spectrum Pharmaceuticals)
6.4.5 Siltuximab (J&J)
6.5 Orphan Glioma Pipeline: Led by Novel Immunotherapies
6.5.1 Rindopepimut (Celldex Therapeutics)
6.5.2 DCVax-L (Northwest Biotherapeutics)
6.5.3 ICT-107 (ImmunoCellular Therapeutics)
6.5.4 Trans-Sodium Crocetinate (TSC, Diffusion Pharmaceuticals): Phase II Results Expected in 2014
6.5.5 Cotara (Peregrine Pharmaceuticals): Over a Decade in Clinical Development
6.5.6 VAL-083 (Del Mar Pharmaceuticals): Avoiding MGMT Resistance Pathways
7. ORPHAN DRUGS IN CANCER: INDUSTRY TRENDS 2013-2023
7.1 Orphan Drugs in Cancer: Strengths and Weaknesses 2013
7.2 Orphan Drugs in Cancer: Opportunities and Threats 2013-2023
7.3 Orphan Drugs in Cancer: STEP Analysis 2013-2023
7.3.1 Social Factors
7.3.1.1 Ethical Considerations for Orphan Drug Development
7.3.2 Technological Developments
7.3.2.1 Increased Understanding of Disease Will Drive Innovation
7.3.3 Economic Pressures Influencing Orphan Drug Uptake
7.3.3.1 Orphan Drugs Tend to be Expensive
7.3.4 Political Issues: The Role of Government and Regulatory Bodies in the Market
7.3.4.1 Harmonising Global Regulatory Pathways
7.4 Trends in Orphan Cancer Drug Development
7.4.1 Clinical Trials for Orphan Drugs
7.4.1.1 Are Adverse Events More Acceptable for Orphan Drugs?
7.4.1.2 Challenges and Opportunities in Patient Recruitment
7.4.2 Biomarkers for Orphan Cancer Indications
7.4.2.1 Companion Diagnostics
7.4.3 The Role of Public Funding in Orphan Drug Development
7.5 Commercialising Orphan Drugs in Cancer
7.5.1 Pricing of Orphan Drugs May Become Crucial to Success
7.5.2 Market and Patient Access Are Crucial for Orphan Drug Success
7.5.3 Repositioning Drugs for Orphan Cancer Indications
7.5.3.1 Expanding Beyond Orphan Indications
7.5.4 Big Pharma and Orphan Cancer Drugs
7.5.4.1 Orphan Drugs Fit the Personalised Medicine Model
7.5.5 Common Market Entry Strategies
7.5.6 Orphan Indications as an Opportunity for Generic Drug Manufacturers
8. RESEARCH INTERVIEWS
8.1 Jeffrey Bacha, President and CEO, Del Mar Pharmaceuticals
8.1.1 Del Mar Pharmaceuticals
8.1.2 Applying for Orphan Drug Status
8.1.3 The Benefits of Orphan Drug Designation
8.1.4 Clinical Trials for Orphan Drugs
8.1.5 Market Access in Glioblastoma
8.1.6 VAL-083 and the Future Glioblastoma Market
8.1.7 Immunotherapies for Glioblastoma
8.1.8 Development of VAL-083
8.1.9 Future Development of VAL-083
8.2 David Kalergis, CEO, Diffusion Pharmaceuticals
8.2.1 Diffusion Pharmaceuticals and TSC
8.2.2 Seeking Orphan Drug Status
8.2.3 Challenges in Orphan Drug Development
8.2.4 TSC and the Glioblastoma Market
9. CONCLUSIONS FROM OUR STUDY
9.1 The State of the Orphan Cancer Drugs Market 2012-2013
9.2 There Will Be Strong Growth in the Market 2013-2023
9.3 The US is the Largest National Market 2013-2023
9.4 There Are Long Pipelines in Many Disease Sectors in 2013
1.1 Orphan Drugs for Cancer: Market Overview 2013
1.2 Report Contents
1.3 Research and Analysis Methods
2. INTRODUCTION TO ORPHAN DRUGS IN CANCER
2.1 Defining Orphan Diseases
2.2 What Are the Major Orphan Cancer Indications?
2.2.1 Leukaemia: The Most Common Blood Cancer
2.2.2 Lymphoma
2.2.3 Multiple Myeloma
2.2.4 Other Orphan Cancer Indications
2.3 Orphan Drug Designation
2.3.1 What Benefits Come with Orphan Drug Designation?
2.4 Demand for Orphan Drugs
2.4.1 Development of Orphan Drugs
3. THE WORLD ORPHAN CANCER DRUGS MARKET 2013-2023
3.1 How Large is the Orphan Drugs Market in 2013?
3.1.1 Cancer is the Largest Sector of the Orphan Drugs Market
3.2 The Orphan Cancer Drugs Market 2013-2023
3.2.1 What Will Drive Growth in the Market to 2023?
3.2.2 Market Restraints 2013-2023
3.3 Orphan Drugs in Leukaemia: Submarket 2013-2023
3.3.1 Leukaemia: Submarket Forecast 2013-2023
3.3.2 Next Generation Tyrosine Kinase Inhibitors to Drive Growth
3.4 Orphan Drugs in Lymphoma: Submarket 2013-2023
3.4.1 Lymphoma: Submarket Forecast 2013-2023
3.4.2 Monoclonal Antibody Therapies Will Dominate the Submarket 2013-2023
3.5 Orphan Drugs in Multiple Myeloma: Submarket 2013-2023
3.5.1 Multiple Myeloma: Submarket Forecast 2013-2023
3.5.2 There Are Many New Therapies in Development
3.6 Orphan Drugs in Glioma: Submarket 2013-2023
3.6.1 Glioma: Submarket Forecast 2013-2023
3.6.2 Therapeutic Vaccines Will Provide a Novel Treatment Strategy 2016-2023
4. LEADING AND EMERGING NATIONAL MARKETS FOR RARE CANCER INDICATIONS 2013-2023
4.1 Leading National Markets 2013
4.1.1 National Market Forecasts 2013-2023
4.2 The US: The Leading Market for Orphan Cancer Drugs 2013-2023
4.2.1 Orphan Drug Regulations in the US
4.2.2 Benefits for Orphan Drug Developers
4.2.3 US Orphan Cancer Drugs Market Forecast 2013-2023
4.3 Orphan Drugs in the EU 2013-2023
4.3.1 The Incentives for Orphan Drug Development in the EU
4.3.2 The EU Orphan Cancer Drugs Market Forecast 2013-2023
4.3.3 Orphan Cancer Drugs in the EU5 Submarkets 2013-2023
4.4 The Japanese Orphan Cancer Drugs Market 2013-2023
4.4.1 Orphan Drug Regulations and Benefits
4.4.2 Japanese Orphan Cancer Drugs Submarket Forecast 2013-2023
4.5 The Potential for Orphan Cancer Drugs in Emerging Markets 2013-2023
4.5.1 Brazil
4.5.2 Russia: Attempts to Introduce Orphan Designation and Incentives 2010-2013
4.5.3 India: Generics Will Limit Branded Rare Cancer Drug Uptake
4.5.4 China
5. OUTLOOK FOR LEADING ORPHAN DRUGS 2013-2023
5.1 Rituxan (Roche/Biogen Idec) Is the Bestselling Orphan Cancer Drug
5.1.1 Roche is Developing a Next Generation Rituxan
5.1.2 Rituxan: Revenue Forecast 2013-2023
5.2 Glivec (Novartis): A Leukaemia Market Leader
5.2.1 The Challenge of Tasigna and Generics
5.2.2 Glivec: Revenue Forecast 2013-2023
5.3 Revlimid (Celgene) Is the Leading Therapy for Multiple Myeloma
5.3.1 Celgene Is Adding New Indications for Revlimid
5.3.2 Revlimid: Revenue Forecast 2013-2023
5.4 Alimta (Eli Lilly)
5.4.1 Revenue Growth Driven by Uptake in NSCLC 2008-2012
5.4.2 Alimta: Revenue Forecast 2013-2023
5.5 Velcade (J&J/Takeda)
5.5.1 Strong Revenue Growth in Markets Worldwide 2008-2012
5.5.2 Velcade Faces Significant Competition in Multiple Myeloma 2013-2023
5.6 Nexavar (Bayer/Onyx Pharmaceuticals)
5.6.1 Nexavar Has Shown Promise in Thyroid Cancer
5.6.2 Nexavar: Revenue Forecast 2013-2023
5.7 Tasigna (Novartis)
5.7.1 Tasigna Is More Effective than Glivec and Sprycel
5.7.2 Will Tasigna Replace Glivec 2013-2023?
5.8 Temodar (Merck & Co.)
5.8.1 Temodar Competes with Generics in the EU
5.8.2 Temodar: Continued Revenue Decline 2013-2023
5.9 Vidaza (Celgene/Nippon Shinyaku)
5.9.1 Vidaza: Revenue Growth 2008-2012
5.9.2 Celgene Is Developing an Oral Azacitidine Formulation
5.9.3 Vidaza Revenue Forecast 2013-2023: When Will Generic Competition Arrive?
5.10 Sprycel (Bristol-Myers Squibb/Otsuka Pharmaceutical)
5.10.1 Sprycel: Financial Performance 2008-2012
5.10.2 Sprycel: Revenue Forecast 2013-2023
5.11 Afinitor (Novartis)
5.11.1 Approval in Breast Cancer Drives Revenue Growth 2012
5.11.2 Afinitor: Revenue Forecast
5.12 Yervoy
5.12.1 Yervoy May Be Effective as a Combination Therapy
5.12.2 Yervoy: Revenue Forecast 2013-2023
5.13 Outlook for Orphan Cancer Drugs Approved in 2012 and 2013
5.13.1 New Therapies for Multiple Myeloma
5.13.1.1 Pomalyst: A Next Generation Thalidomide Derivative
5.13.1.2 Kyprolis (Onyx Pharmaceuticals)
5.13.1.3 Pomalyst versus Kyprolis
5.13.1.4 Pomalyst and Kyprolis: Revenue Forecast 2013-2023
5.13.2 Three New Drugs Approved for CML in 2012
5.13.2.1 Bosulif (Pfizer): A TKI Targeting New Mutations
5.13.2.2 Iclusig (ARIAD Pharmaceuticals)
5.13.2.3 Bosulif and Iclusig: Revenue Forecast 2013-2023
5.13.2.4 Synribo (Teva)
5.13.3 Commercial Prospects for Marqibo (Talon Therapeutics)
5.13.4 Cometriq (Exelixis)
5.13.4.1 Cometriq: Revenue Forecast 2013-2023
5.13.5 Stivarga (Bayer/Onyx Pharmaceuticals): Opportunities in Stomach and Liver Cancer
5.13.5.1 Stivarga: Revenue Forecast 2013-2023
5.14 Other Leading Cancer Therapies Approved for Orphan Indications 2012
5.14.1 Avastin (Roche)
5.14.1.1 Avastin Shows Promise in Glioblastoma
5.14.2 Erbitux (Bristol-Myers Squibb/Eli Lilly/Merck Serono)
5.14.2.1 Attempts to Expand Approved Indications
5.14.3 Herceptin (Roche)
6. ORPHAN CANCER DRUGS: R&D PIPELINE 2013
6.1 Orphan Drugs in the Leukaemia Pipeline 2013
6.1.1 Obinutuzumab (Roche)
6.1.2 Dacogen: Eisai Seeks Approval for AML in the US
6.1.3 CPX-351 (Celator Pharmaceuticals)
6.1.4 Dinaciclib (Merck & Co.)
6.1.5 EZN-2285 (Sigma-Tau Pharmaceuticals): Reformulation for Improved Dosing
6.1.6 Ibrutinib (J&J/Pharmacyclics): Awarded Breakthrough Status in Multiple Indications
6.1.7 Midostaurin (Novartis): Targeting the FLT3 Gene
6.1.8 Moxetumomab Pasudotox (MedImmune): Combining Antibodies and Toxin Fragments
6.1.9 Vosaroxin (Sunesis Pharmaceuticals)
6.1.10 Alvocidib (Tolero Pharmaceuticals)
6.1.11 Blinatumomab (Amgen): Complete Response in Two Thirds of Patients
6.1.12 CNDO-109 (Coronado Biosciences)
6.1.13 Tosedostat (Chroma Therapeutics/Cell Therapeutics)
6.1.14 TRU-016 (Emergent BioSolutions)
6.1.15 Veltuzumab (Immunomedics)
6.2 Orphan Drugs in the MDS Pipeline 2013
6.2.1 Rigosertib (Onconova Therapeutics/Baxter)
6.2.2 Sapacitabine (Cyclacel Pharmaceuticals)
6.2.3 StemEx (Gamida Cell): A Regenerative Medicine Approach to Treating Cancer
6.2.4 Pacritinib (Cell Therapeutics): Approval Possible in 2016
6.2.5 Telintra (Telik)
6.3 Orphan Drugs in the Lymphoma Pipeline 2013
6.3.1 Mechlorethamine Gel (Ceptaris Therapeutics): Fast and Accurate Dosing for Patients
6.3.2 BiovaxID (BioVest International): A Therapeutic Vaccine for Follicular Lymphoma
6.3.3 Belinostat (Topotarget/Spectrum Pharmaceuticals)
6.3.4 Epratuzumab (Immunomedics)
6.3.5 Faridak (Novartis): Approval in Myeloma in 2014?
6.3.6 Mogamulizumab (Kyowa Hakko Kirin/Amgen)
6.3.7 Darinaparsin (ZIOPHARM Oncology)
6.3.8 Mocetinostat: MethylGene Is Seeking a Development Partner
6.3.9 Resminostat (4SC)
6.4 Orphan Drugs in the Multiple Myeloma Pipeline 2013
6.4.1 Aplidin (PharmaMar)
6.4.2 Elotuzumab (AbbVie/Bristol-Myers Squibb)
6.4.3 Ixazomib (Millennium Pharmaceuticals): An Oral Proteasome Inhibitor to Replace Velcade?
6.4.4 Captisol-Enabled Melphalan (Spectrum Pharmaceuticals)
6.4.5 Siltuximab (J&J)
6.5 Orphan Glioma Pipeline: Led by Novel Immunotherapies
6.5.1 Rindopepimut (Celldex Therapeutics)
6.5.2 DCVax-L (Northwest Biotherapeutics)
6.5.3 ICT-107 (ImmunoCellular Therapeutics)
6.5.4 Trans-Sodium Crocetinate (TSC, Diffusion Pharmaceuticals): Phase II Results Expected in 2014
6.5.5 Cotara (Peregrine Pharmaceuticals): Over a Decade in Clinical Development
6.5.6 VAL-083 (Del Mar Pharmaceuticals): Avoiding MGMT Resistance Pathways
7. ORPHAN DRUGS IN CANCER: INDUSTRY TRENDS 2013-2023
7.1 Orphan Drugs in Cancer: Strengths and Weaknesses 2013
7.2 Orphan Drugs in Cancer: Opportunities and Threats 2013-2023
7.3 Orphan Drugs in Cancer: STEP Analysis 2013-2023
7.3.1 Social Factors
7.3.1.1 Ethical Considerations for Orphan Drug Development
7.3.2 Technological Developments
7.3.2.1 Increased Understanding of Disease Will Drive Innovation
7.3.3 Economic Pressures Influencing Orphan Drug Uptake
7.3.3.1 Orphan Drugs Tend to be Expensive
7.3.4 Political Issues: The Role of Government and Regulatory Bodies in the Market
7.3.4.1 Harmonising Global Regulatory Pathways
7.4 Trends in Orphan Cancer Drug Development
7.4.1 Clinical Trials for Orphan Drugs
7.4.1.1 Are Adverse Events More Acceptable for Orphan Drugs?
7.4.1.2 Challenges and Opportunities in Patient Recruitment
7.4.2 Biomarkers for Orphan Cancer Indications
7.4.2.1 Companion Diagnostics
7.4.3 The Role of Public Funding in Orphan Drug Development
7.5 Commercialising Orphan Drugs in Cancer
7.5.1 Pricing of Orphan Drugs May Become Crucial to Success
7.5.2 Market and Patient Access Are Crucial for Orphan Drug Success
7.5.3 Repositioning Drugs for Orphan Cancer Indications
7.5.3.1 Expanding Beyond Orphan Indications
7.5.4 Big Pharma and Orphan Cancer Drugs
7.5.4.1 Orphan Drugs Fit the Personalised Medicine Model
7.5.5 Common Market Entry Strategies
7.5.6 Orphan Indications as an Opportunity for Generic Drug Manufacturers
8. RESEARCH INTERVIEWS
8.1 Jeffrey Bacha, President and CEO, Del Mar Pharmaceuticals
8.1.1 Del Mar Pharmaceuticals
8.1.2 Applying for Orphan Drug Status
8.1.3 The Benefits of Orphan Drug Designation
8.1.4 Clinical Trials for Orphan Drugs
8.1.5 Market Access in Glioblastoma
8.1.6 VAL-083 and the Future Glioblastoma Market
8.1.7 Immunotherapies for Glioblastoma
8.1.8 Development of VAL-083
8.1.9 Future Development of VAL-083
8.2 David Kalergis, CEO, Diffusion Pharmaceuticals
8.2.1 Diffusion Pharmaceuticals and TSC
8.2.2 Seeking Orphan Drug Status
8.2.3 Challenges in Orphan Drug Development
8.2.4 TSC and the Glioblastoma Market
9. CONCLUSIONS FROM OUR STUDY
9.1 The State of the Orphan Cancer Drugs Market 2012-2013
9.2 There Will Be Strong Growth in the Market 2013-2023
9.3 The US is the Largest National Market 2013-2023
9.4 There Are Long Pipelines in Many Disease Sectors in 2013
LIST OF TABLES
Table 1.1 Currency Exchange Rates
Table 1.2 Common Abbreviations Used in this Report
Table 2.1 EU Prevalence Statistics for Selected Rare Cancer Indications, 2013
Table 3.1 Global Orphan Cancer Drugs Market: Revenue ($bn) and Market Share (%) by Sector, 2012
Table 3.2 Global Orphan Cancer Drugs Market: Overall Market and Revenue Forecasts by Sector ($bn), 2012-2023
Table 3.3 Global Orphan Cancer Drug Market: Submarket Shares (%), 2012-2023
Table 3.4 Patent Expiries for Selected Orphan Cancer Drugs in the US and EU, 2009-2024
Table 3.5 Leukaemia Submarket Forecast ($bn), 2012-2023
Table 3.6 Lymphoma Submarket Forecast ($bn), 2012-2023
Table 3.7 Multiple Myeloma Submarket Forecast ($bn), 2012-2023
Table 3.8 Glioma Submarket Forecast ($bn), 2012-2023
Table 4.1 Orphan Cancer Drugs Market: Revenue ($bn) and Market Share (%) by Region, 2012
Table 4.2 Orphan Cancer Drugs Market: Regional Submarket Forecasts ($bn), 2012-2023
Table 4.3 Global Orphan Cancer Drug Market: Regional Submarket Shares (%), 2012-2023
Table 4.4 US Orphan Cancer Drugs: Submarket Forecast ($bn), 2012-2023
Table 4.5 Fee Reductions (%) Available for SMEs Developing Orphan Drugs in the EU, 2013
Table 4.6 EU5 Orphan Cancer Drugs Market: Revenue ($bn) and Market Share (%) by Country, 2012
Table 4.7 EU5 Orphan Cancer Drugs Market: National Submarket Forecasts ($bn), 2012-2023
Table 4.8 Japanese Orphan Cancer Drugs: Submarket Forecast ($bn), 2012-2023
Table 4.9 BRIC Orphan Cancer Drugs Market: National Submarket Forecasts ($bn), 2012-2023
Table 5.1 Top Orphan Cancer Drugs: Revenues ($bn) and Leading Revenue Shares (%), 2012
Table 5.2 Orphan Drug Designations in the US for Rituxan, 2013
Table 5.3 Rituxan: Revenue ($bn), 2008-2012
Table 5.4 Biosimilar Rituximab: Selected Pipeline, 2013
Table 5.5 Rituxan: Revenue Forecast ($bn), 2012-2023
Table 5.6 Orphan Drug Designations in the US for Glivec, 2013
Table 5.7 Glivec: Revenue ($bn), 2008-2012
Table 5.8 Glivec: Revenue Forecast ($bn), 2012-2023
Table 5.9 Revlimid: Revenue ($bn), 2006-2012
Table 5.10 Revlimid: Revenue Forecast ($bn), 2012-2023
Table 5.11 Alimta: Revenue ($bn), 2008-2012
Table 5.12 Alimta: Revenue Forecast ($bn), 2012-2023
Table 5.13 Velcade: Revenue by Company ($bn), 2008-2012
Table 5.14 Velcade: Revenue Forecast ($bn), 2012-2023
Table 5.15 Nexavar: Revenue ($bn), 2008-2012
Table 5.16 Nexavar: Revenue Forecast ($bn), 2012-2023
Table 5.17 Tasigna: Revenue ($bn), 2008-2012
Table 5.18 Tasigna: Revenue Forecast ($bn), 2012-2023
Table 5.19 Temodar: Revenue ($bn), 2010-2012
Table 5.20 Temodar: Revenue Forecast ($bn), 2012-2023
Table 5.21 Vidaza: Revenue by Company ($bn), 2008-2012
Table 5.22 Vidaza: Revenue Forecast ($bn), 2012-2023
Table 5.23 Sprycel: Revenue by Company ($bn), 2008-2012
Table 5.24 Sprycel: Revenue Forecast ($bn), 2012-2023
Table 5.25 Orphan Drug Designations in the US for Afinitor, 2013
Table 5.26 Afinitor: Revenue ($bn), 2009-2012
Table 5.27 Afinitor: Revenue Forecast ($bn), 2012-2023
Table 5.28 Yervoy: Revenue ($bn) 2011-2012
Table 5.29 Yervoy: Revenue Forecast ($bn), 2012-2023
Table 5.30 Kyprolis and Pomalyst: Revenue Forecasts ($bn), 2012-2023
Table 5.31 Bosulif and Iclusig: Revenue Forecasts ($bn), 2012-2023
Table 5.32 Cometriq: Revenue Forecast ($bn), 2012-2023
Table 5.33 Stivarga: Revenue Forecast ($bn), 2012-2023
Table 6.1 Selected Phase II and III Pipeline Orphan Candidates in Development for Leukaemia, 2013
Table 6.2 Ongoing Phase III Trials for Ibrutinib, 2013
Table 6.3 Ongoing Clinical Trials for Midostaurin, 2013
Table 6.4 Selected Phase II and III Pipeline Orphan Candidates in Development for MDS, 2013
Table 6.5 Phase II and III Pipeline Orphan Candidates in Development for Lymphoma, 2013
Table 6.6 Ongoing Phase II and III Trials for Mogamulizumab, 2013
Table 6.7 Selected Phase II and III Pipeline Orphan Candidates in Development for Multiple Myeloma, 2013
Table 6.8 Selected Phase II and III Pipeline Orphan Candidates in Development for Glioma, 2013
Table 7.1 Global Orphan Cancer Drugs Market: Strengths and Weaknesses, 2012-2013
Table 7.2 Global Orphan Cancer Drugs Market: Opportunities and Threats, 2013-2023
Table 7.3 Monthly and Annual Prices ($) in the US for Selected Orphan Cancer Drugs Approved 2011-2013
Table 9.1 Global Orphan Cancer Drugs Market: Revenue ($bn), and Market Share (%) by Sector, 2012, 2017 & 2023
Table 9.2 Global Orphan Cancer Drugs Market: Revenue ($bn), Market Share (%) by Region, 2012, 2017 & 2023
Table 1.1 Currency Exchange Rates
Table 1.2 Common Abbreviations Used in this Report
Table 2.1 EU Prevalence Statistics for Selected Rare Cancer Indications, 2013
Table 3.1 Global Orphan Cancer Drugs Market: Revenue ($bn) and Market Share (%) by Sector, 2012
Table 3.2 Global Orphan Cancer Drugs Market: Overall Market and Revenue Forecasts by Sector ($bn), 2012-2023
Table 3.3 Global Orphan Cancer Drug Market: Submarket Shares (%), 2012-2023
Table 3.4 Patent Expiries for Selected Orphan Cancer Drugs in the US and EU, 2009-2024
Table 3.5 Leukaemia Submarket Forecast ($bn), 2012-2023
Table 3.6 Lymphoma Submarket Forecast ($bn), 2012-2023
Table 3.7 Multiple Myeloma Submarket Forecast ($bn), 2012-2023
Table 3.8 Glioma Submarket Forecast ($bn), 2012-2023
Table 4.1 Orphan Cancer Drugs Market: Revenue ($bn) and Market Share (%) by Region, 2012
Table 4.2 Orphan Cancer Drugs Market: Regional Submarket Forecasts ($bn), 2012-2023
Table 4.3 Global Orphan Cancer Drug Market: Regional Submarket Shares (%), 2012-2023
Table 4.4 US Orphan Cancer Drugs: Submarket Forecast ($bn), 2012-2023
Table 4.5 Fee Reductions (%) Available for SMEs Developing Orphan Drugs in the EU, 2013
Table 4.6 EU5 Orphan Cancer Drugs Market: Revenue ($bn) and Market Share (%) by Country, 2012
Table 4.7 EU5 Orphan Cancer Drugs Market: National Submarket Forecasts ($bn), 2012-2023
Table 4.8 Japanese Orphan Cancer Drugs: Submarket Forecast ($bn), 2012-2023
Table 4.9 BRIC Orphan Cancer Drugs Market: National Submarket Forecasts ($bn), 2012-2023
Table 5.1 Top Orphan Cancer Drugs: Revenues ($bn) and Leading Revenue Shares (%), 2012
Table 5.2 Orphan Drug Designations in the US for Rituxan, 2013
Table 5.3 Rituxan: Revenue ($bn), 2008-2012
Table 5.4 Biosimilar Rituximab: Selected Pipeline, 2013
Table 5.5 Rituxan: Revenue Forecast ($bn), 2012-2023
Table 5.6 Orphan Drug Designations in the US for Glivec, 2013
Table 5.7 Glivec: Revenue ($bn), 2008-2012
Table 5.8 Glivec: Revenue Forecast ($bn), 2012-2023
Table 5.9 Revlimid: Revenue ($bn), 2006-2012
Table 5.10 Revlimid: Revenue Forecast ($bn), 2012-2023
Table 5.11 Alimta: Revenue ($bn), 2008-2012
Table 5.12 Alimta: Revenue Forecast ($bn), 2012-2023
Table 5.13 Velcade: Revenue by Company ($bn), 2008-2012
Table 5.14 Velcade: Revenue Forecast ($bn), 2012-2023
Table 5.15 Nexavar: Revenue ($bn), 2008-2012
Table 5.16 Nexavar: Revenue Forecast ($bn), 2012-2023
Table 5.17 Tasigna: Revenue ($bn), 2008-2012
Table 5.18 Tasigna: Revenue Forecast ($bn), 2012-2023
Table 5.19 Temodar: Revenue ($bn), 2010-2012
Table 5.20 Temodar: Revenue Forecast ($bn), 2012-2023
Table 5.21 Vidaza: Revenue by Company ($bn), 2008-2012
Table 5.22 Vidaza: Revenue Forecast ($bn), 2012-2023
Table 5.23 Sprycel: Revenue by Company ($bn), 2008-2012
Table 5.24 Sprycel: Revenue Forecast ($bn), 2012-2023
Table 5.25 Orphan Drug Designations in the US for Afinitor, 2013
Table 5.26 Afinitor: Revenue ($bn), 2009-2012
Table 5.27 Afinitor: Revenue Forecast ($bn), 2012-2023
Table 5.28 Yervoy: Revenue ($bn) 2011-2012
Table 5.29 Yervoy: Revenue Forecast ($bn), 2012-2023
Table 5.30 Kyprolis and Pomalyst: Revenue Forecasts ($bn), 2012-2023
Table 5.31 Bosulif and Iclusig: Revenue Forecasts ($bn), 2012-2023
Table 5.32 Cometriq: Revenue Forecast ($bn), 2012-2023
Table 5.33 Stivarga: Revenue Forecast ($bn), 2012-2023
Table 6.1 Selected Phase II and III Pipeline Orphan Candidates in Development for Leukaemia, 2013
Table 6.2 Ongoing Phase III Trials for Ibrutinib, 2013
Table 6.3 Ongoing Clinical Trials for Midostaurin, 2013
Table 6.4 Selected Phase II and III Pipeline Orphan Candidates in Development for MDS, 2013
Table 6.5 Phase II and III Pipeline Orphan Candidates in Development for Lymphoma, 2013
Table 6.6 Ongoing Phase II and III Trials for Mogamulizumab, 2013
Table 6.7 Selected Phase II and III Pipeline Orphan Candidates in Development for Multiple Myeloma, 2013
Table 6.8 Selected Phase II and III Pipeline Orphan Candidates in Development for Glioma, 2013
Table 7.1 Global Orphan Cancer Drugs Market: Strengths and Weaknesses, 2012-2013
Table 7.2 Global Orphan Cancer Drugs Market: Opportunities and Threats, 2013-2023
Table 7.3 Monthly and Annual Prices ($) in the US for Selected Orphan Cancer Drugs Approved 2011-2013
Table 9.1 Global Orphan Cancer Drugs Market: Revenue ($bn), and Market Share (%) by Sector, 2012, 2017 & 2023
Table 9.2 Global Orphan Cancer Drugs Market: Revenue ($bn), Market Share (%) by Region, 2012, 2017 & 2023
Velcade Faces Significant Competition in Multiple Myeloma 2013-2023
Velcade begins to lose patent protection in major developed markets in 2017. However, between 2013 and then the drug faces significant competition from new multiple myeloma treatments that will restrain revenue growth. Takeda, for example, is developing a second generation proteasome inhibitor that can be dosed orally, compared with via injection for Velcade. This is likely to be launched in the US and EU in 2016, visiongain believes. Celgene also recently received approvalin the US for its third eneration thalidomide derivative Pomalyst. Onyx Pharmaceuticals has also launched a next generation proteasome inhibitor, Kyprolis, although as with Velcade, this drug must be injected.
Towards the middle of this decade, Velcade will also face competition from monoclonal antibody therapies. Unlike many cancer indications, no monoclonal antibodies have yet been approved for treating multiple myeloma. However, promising pipeline therapies include elotuzumab, in development by Bristol-Myers Squibb and AbbVie. As these drugs reach the market, though, Velcade will be facing generic competition. Actavis has already reportedly submitted an ANDA to the FDA for generic bortezomib. Revenue for Velcade will peak in 2016 at $3.11bn, after which it will decline rapidly to $1.17bn in 2023.
