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Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents such as AMR101 and ACR16 to Provide Treatment Options and Boost the Huntington’s Disease Segment by GBIResearch

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Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents such as AMR101 and ACR16 to Provide Treatment Options and Boost the Huntington’s Disease Segment, which provides insights into the CNS orphan disease therapeutics market until 2017. The report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GBI Research’s team of industry experts. The report provides an in-depth analysis of amyotrophic lateral sclerosis, huntington’s disease and myasthenia gravis. It also provides the share of generics in global CNS orphan disease therapeutics and in each indication market. The report examines the global orphan diseases therapeutics in CNS usage patterns. It includes the geographical distribution of amyotrophic lateral sclerosis, huntington’s disease and myasthenia gravis markets across the US, the top five countries of Europe and in Japan. The report also includes insights into the orphan diseases therapeutics in CNS Research and Development (R&D) product pipeline and explores the competitive landscape, including major players in the CNS orphan disease therapeutics market. Finally, the report includes analysis on Mergers and Acquisitions (M&A) and licensing agreements that took place in the orphan diseases therapeutics in CNS market.

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									      Orphan Diseases Therapeutics in CNS to 2017 - Novel
     Agents such as AMR101 and ACR16 to Provide Treatment
      Options and Boost the Huntington’s Disease Segment
 Reference Code: GBIHC144MR                                                                                             Publication Date: April 2012


                                   GBI Research’s analysis indicates that the global Central Nervous System (CNS) orphan disease
  The global orphan CNS            therapeutics market is a niche market within the pharmaceutical industry. The indications covered
  disease market grew from         in this study are Huntington’s Disease (HD), Amyotrophic Lateral Sclerosis (ALS) and Myasthenia
  $359.6m in 2002 to               Gravis (MG), all of which have very low prevalence and incidence rates. The market is driven by
  $665.5m in 2010 at a             the HD market, and will be further driven by the launch of new molecules in the forecast period.
  CAGR of 8%. It is
  expected to reach $1.2           The Global Market for CNS Disorders is Forecast to Grow Rapidly from 2010 to 2017
  billion by 2017, growing at      The global CNS orphan disease market is set to grow over the forecast period. It grew from
  a CAGR of 17.3%                  $359.6m in 2002 to $665.5m in 2010 at a Compound Annual Growth Rate (CAGR) of 8%. This is
                                   projected to increase to a CAGR of 17.6% over the forecast period, with revenues reaching $2.1
                                   billion in 2017. The market is expected to grow as the pharmaceutical industry recognizes the
                                   attractive opportunities as a result of low competition, high unmet need and government incentives
                                   such as grants and subsidies for the development of drugs for orphan diseases. Less competition
                                   and a high Annual Cost of Treatment (ACT), and the possibility for mergers and licensing
                                   agreements between companies will drive the market in the future

                                    Orphan Diseases Therapeutics in CNS, Global, Revenue Forecasts, ($m), 2002 - 2017

                                                             2,500



                                                             2,000
                                                                               CAGR (2010-2017) : 17.6%
                                              Revenue ($m)




                                                             1,500



                                                             1,000   CAGR (2002-2010) : 8.0%



                                                              500



                                                                0
                                                                            2002                     2010                       2017


                                    Source: GBI Research, Orphanet, NHS Information Centre, FDA, Medscape, EURORDIS, NICE, RX USA, NCBI, UNC
                                    School of Medicine, Archives of Internal Medicine




Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents                                                           GBIHC144MR / Published APR 2012
such as AMR101 and ACR16 to Provide Treatment Options and                                                                                          Page 1
Boost the Huntington’s Disease Segment                                          © GBI Research. This report is a licensed product and is not to be copied,
                                                                                                                reproduced, shared or resold in any form
                                   The Global Market for Orphan Disease CNS Disorders is Growing Due to Increasing
                                   Realization of Available Attractive Opportunities
                                   The global market for CNS orphan disease therapeutics is growing due to government grants and
                                   subsidies for the development of drugs previously considered by pharmaceutical to be unattractive
                                   options. CNS orphan diseases have a low prevalence, but present serious and sometimes fatal
                                   conditions. These incentives range from grants and subsidies during Research and Development
                                   (R&D) to fast track processes to get the drug onto the market. The traditional view of rare diseases
                                   as being unprofitable is being sidelined thanks to the success of drugs such as Rilutek (riluzole) for
                                   ALS. As bigger indications such as HD, ALS and MG, become lucrative areas, other less well
                                   known CNS orphan diseases may also start to receive the same amount of attention from
                                   pharmaceutical companies looking for ways to remain profitable in an increasingly competitive
                                   global climate.
                                   Significant Unmet Need in CNS Orphan Diseases Provides Good Opportunities for
                                   Companies Wishing to Enter the Market
                                   The CNS orphan disease therapeutics market is characterized by high unmet need. HD and ALS
                                   each have only one approved therapy, Xenazine (tetrabenazine) and Rilutek respectively.
                                   Xenazine offers only symptomatic treatment for the chorea associated with HD, while Rilutek
                                   extends the average survival period of ALS sufferers by a few months. Consequently, there is a
                                   significant need for disease-modifying therapeutics that can stop or reverse the effects of the
                                   degeneration involved in these conditions.
                                   In addition, the safety profiles of the existing drugs are low. Both Rilutek and Xenazine are
                                   associated with serious side effects and certain patient groups cannot safely take them at all.
                                   Careful monitoring of patients is required in both diseases. Molecules with disease-modifying
                                   abilities as well as better safety profiles are urgently needed for patients with degenerative and
                                   debilitating conditions.
                                   A company that can develop a disease-modifying product with a good safety profile for the
                                   indications in the market will have a significant opportunity to gain high revenue, as the drug could
                                   command a high price and would have little competition.
                                   Developing Drugs for CNS Orphan Diseases is Challenging and Companies Face Multiple
                                   Difficulties
                                   Despite government incentives, developing CNS orphan disease therapeutics is expensive, time
                                   and resource-heavy, and is therefore a risky endeavor. Recruiting patients for clinical trials is
                                   difficult for a number of reasons. Patient numbers are characteristically low in orphan indications,
                                   and the number of patients willing to take part in clinical trials is even lower. This results in a need
                                   to spread trials across large areas, including across countries. This requires a high degree of
                                   collaboration and is resource-intensive. This results in far more time and money being devoted to
                                   trials for CNS orphan diseases than is the case normally. The number of pipeline products in
                                   development for HD and ALS is low and yet these two diseases are among the largest indications
                                   within the CNS orphan diseases market.
                                   CNS diseases are often poorly understood, as obtaining samples and subjects for testing is
                                   difficult, and the mechanisms that underlie neurological conditions are particularly complicated and
                                   only partially discovered. This hinders the development of new therapies.
                                   Off-Label and Generic Drugs Dominate in Many Indications
                                   Due to the lack of efficacious therapeutics to cure or modify the disease course of CNS orphan
                                   disorders and the low number of targeted, approved drugs for symptomatic treatments, the market
                                   relies on generic drugs to fulfill this need. This brings down the average ACT. When the drugs are
                                   used off-label, this can sometimes mean that reimbursement is not available for patients. In HD,
                                   anti-depressants, anti-psychotics and anti-dyskinetics occupy a large proportion of the market.
                                   They are not specifically approved for the disease.




Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents                                                 GBIHC144MR / Published APR 2012
such as AMR101 and ACR16 to Provide Treatment Options and                                                                                Page 2
Boost the Huntington’s Disease Segment                                © GBI Research. This report is a licensed product and is not to be copied,
                                                                                                      reproduced, shared or resold in any form
                                   1         Table of Contents
                                   1   Table of Contents ........................................................................................................................ 3
                                       1.1    List of Tables..................................................................................................................... 5
                                       1.2    List of Figures ................................................................................................................... 7
                                   2   Orphan Diseases Therapeutics in CNS - Introduction ................................................................. 9
                                       2.1    GBI Research Report Guidance ..................................................................................... 10
                                   3   Orphan Diseases Therapeutics in CNS - Market Overview ....................................................... 11
                                       3.1    Introduction ..................................................................................................................... 11
                                       3.2    Orphan Disease Regulation and Incentives .................................................................... 11
                                       3.3    Global Revenue Forecasts for Orphan Diseases Therapeutics in CNS .......................... 13
                                          3.3.1    Global Revenue and Forecast ................................................................................. 13
                                          3.3.2    Geographical Segmentation .................................................................................... 15
                                          3.3.3    Branded and Generic Market Share ........................................................................ 17
                                          3.3.4    Revenue Share by Indication................................................................................... 18
                                          3.3.5    Market Drivers ......................................................................................................... 20
                                          3.3.6    Market Restraints .................................................................................................... 21
                                          3.3.7    Unmet Needs........................................................................................................... 21
                                          3.3.8    Opportunities ........................................................................................................... 22
                                   4   Orphan Diseases Therapeutics in CNS - Geographical Landscape .......................................... 23
                                       4.1    The US ............................................................................................................................ 23
                                          4.1.1    Revenue .................................................................................................................. 23
                                       4.2    Top Five Countries of Europe ......................................................................................... 25
                                          4.2.1    Revenue .................................................................................................................. 25
                                       4.3    Japan .............................................................................................................................. 26
                                          4.3.1    Revenue .................................................................................................................. 26
                                   5   Orphan Diseases in CNS Disorders - Therapeutic Landscape .................................................. 27
                                       5.1    Amyotrophic Lateral Sclerosis......................................................................................... 27
                                          5.1.1    Disease Overview.................................................................................................... 27
                                          5.1.2    Revenue .................................................................................................................. 27
                                          5.1.3    Average Annual Cost of Treatment ......................................................................... 31
                                          5.1.4    Branded and Generic Market Share ........................................................................ 33
                                          5.1.5    Treatment Algorithm ................................................................................................ 34
                                          5.1.6    Treatment Usage Patterns ...................................................................................... 34
                                          5.1.7    Approved Molecules ................................................................................................ 41
                                          5.1.8    Rilutek ..................................................................................................................... 41
                                          5.1.9    Nuedexta (Dextromethorphan Hydrobromide and Quinidine Sulfate)...................... 42
                                       5.2    Huntington’s Disease ...................................................................................................... 43
                                          5.2.1    Disease Overview.................................................................................................... 43
                                          5.2.2    Total Revenue ......................................................................................................... 44
                                          5.2.3    Average Annual Cost of Treatment (ACT) ............................................................... 47
                                          5.2.4    Branded and Generic Market Share ........................................................................ 48
                                          5.2.5    Treatment Algorithm ................................................................................................ 49
                                          5.2.6    Treatment Usage Patterns ...................................................................................... 50
                                       5.3    Approved Molecules ....................................................................................................... 54
                                          5.3.1    Xenazine (tetrabenazine) ........................................................................................ 54
                                       5.4    Myasthenia Gravis .......................................................................................................... 55
                                          5.4.1    Disease Overview.................................................................................................... 55
                                          5.4.2    Total Revenue ......................................................................................................... 56
                                          5.4.3    Branded and Generic Market Share ........................................................................ 59
                                          5.4.4    Average Annual Cost of Treatment ......................................................................... 60
                                          5.4.5    Treatment Algorithm ................................................................................................ 61
                                          5.4.6    Treatment Usage Patterns ...................................................................................... 62
                                          5.4.7    Approved Molecules ................................................................................................ 66
                                       5.5    Other Indications ............................................................................................................. 67


Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents                                                              GBIHC144MR / Published APR 2012
such as AMR101 and ACR16 to Provide Treatment Options and                                                                                             Page 3
Boost the Huntington’s Disease Segment                                             © GBI Research. This report is a licensed product and is not to be copied,
                                                                                                                   reproduced, shared or resold in any form
                                          5.5.1    Total Revenue ......................................................................................................... 67
                                          5.5.2    Product Profiles ....................................................................................................... 68
                                   6   Global Orphan Diseases in CNS Disorders - Pipeline Analysis ................................................. 71
                                       6.1    Introduction ..................................................................................................................... 71
                                       6.2    Research and Development Pipeline .............................................................................. 72
                                          6.2.1    Overview ................................................................................................................. 72
                                          6.2.2    Pipeline by Clinical Phases of Development ........................................................... 75
                                          6.2.3    Profiles of Promising Drugs in Orphan Diseases in CNS Disorders ........................ 82
                                   7   Global Orphan Diseases in CNS Disorders - Competitive Landscape ...................................... 85
                                       7.1    Major Players .................................................................................................................. 85
                                       7.2    Major Company Profiles .................................................................................................. 85
                                          7.2.1    Biogen Idec.............................................................................................................. 85
                                          7.2.2    Avanir ...................................................................................................................... 86
                                          7.2.3    Neuraltus ................................................................................................................. 87
                                          7.2.4    Cytokinetics ............................................................................................................. 88
                                          7.2.5    Avicena.................................................................................................................... 89
                                          7.2.6    NeuroSearch ........................................................................................................... 90
                                          7.2.7    Trophos ................................................................................................................... 91
                                          7.2.8    Amarin ..................................................................................................................... 92
                                          7.2.9    Siena Biotech .......................................................................................................... 93
                                          7.2.10 Sanofi ...................................................................................................................... 94
                                   8   Global Orphan Diseases in CNS Disorders - Strategic Consolidations ..................................... 95
                                       8.1    Mergers and Acquisitions ................................................................................................ 95
                                          8.1.1    Overview ................................................................................................................. 95
                                          8.1.2    Major Mergers and Acquisitions Deals .................................................................... 97
                                          8.1.3    Segmentation by Indication ..................................................................................... 98
                                          8.1.4    Segmentation by Geography ................................................................................... 99
                                          8.1.5    Segmentation by Deal Value ................................................................................. 100
                                          8.1.6    Segmentation by Year ........................................................................................... 101
                                       8.2    Licensing Agreements .................................................................................................. 102
                                          8.2.1    Overview ............................................................................................................... 102
                                          8.2.2    Major Licensing Deals ........................................................................................... 105
                                          8.2.3    Segmentation by Indication ................................................................................... 107
                                          8.2.4    Segmentation by Geography ................................................................................. 108
                                          8.2.5    Segmentation by Deal Value ................................................................................. 109
                                          8.2.6    Segmentation by Year ........................................................................................... 110
                                       8.3    Co Development Deals ................................................................................................. 111
                                          8.3.1    Segmentation by Geography ................................................................................. 113
                                          8.3.2    Segmentation by Value ......................................................................................... 114
                                          8.3.3    Segmentation by Year ........................................................................................... 115
                                   9   CNS Orphan Diseases - Appendix .......................................................................................... 116
                                       9.1    Market Definition ........................................................................................................... 116
                                       9.2    Abbreviations ................................................................................................................ 116
                                       9.3    Research Methodology ................................................................................................. 117
                                          9.3.1    Coverage ............................................................................................................... 118
                                          9.3.2    Secondary Research ............................................................................................. 118
                                          9.3.3    Primary Research .................................................................................................. 118
                                       9.4    Forecasting ................................................................................................................... 119
                                          9.4.1    Epidemiology-based Forecasting .......................................................................... 119
                                          9.4.2    Expert Panels Validation ....................................................................................... 120
                                       9.5    Contact Us .................................................................................................................... 120
                                       9.6    Disclaimer ..................................................................................................................... 120
                                       9.7    Sources ......................................................................................................................... 120




Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents                                                              GBIHC144MR / Published APR 2012
such as AMR101 and ACR16 to Provide Treatment Options and                                                                                             Page 4
Boost the Huntington’s Disease Segment                                             © GBI Research. This report is a licensed product and is not to be copied,
                                                                                                                   reproduced, shared or resold in any form
                                   1.1    List of Tables
                                   Table 1:    Orphan Diseases Therapeutics in CNS, Global, Revenue ($m), 2002–2010................. 13
                                   Table 2:    Orphan Diseases Therapeutics in CNS, Global, Revenue Forecasts ($m), 2010–2017 13
                                   Table 3:    Orphan Diseases Therapeutics in CNS, Global, Revenue Segmentation by Geography,
                                               ($m), 2002–2010 ............................................................................................................ 15
                                   Table 4:    Orphan Diseases Therapeutics in CNS, Global, Revenue Segmentation by Geography,
                                               ($m), 2010–2017 ............................................................................................................ 16
                                   Table 5:    Orphan Diseases Therapeutics in CNS, Global, Revenues by Indication ($m), 2002-
                                               2010 ............................................................................................................................... 18
                                   Table 6:    Orphan Diseases Therapeutics in CNS, Global, Revenues by Indication ($m), 2010-
                                               2017 ............................................................................................................................... 18
                                   Table 7:    Orphan Diseases Therapeutics in CNS, The US, Revenues, ($m), 2002–2010 ............ 23
                                   Table 8:    Orphan Diseases Therapeutics in CNS, The US, Revenue Forecasts, ($m), 2010–2017
                                               ....................................................................................................................................... 23
                                   Table 9:    Orphan Diseases Therapeutics in CNS, Top Five Countries of Europe, Revenues ($m),
                                               2010–2017 ..................................................................................................................... 25
                                   Table 10:   Orphan Diseases Therapeutics in CNS, Top Five Countries of Europe, Revenue
                                               Forecasts ($m), 2010–2017 ........................................................................................... 25
                                   Table 11:   Orphan Diseases Therapeutics in CNS, Japan, Revenues ($m), 2002–2010 ............... 26
                                   Table 12:   Orphan Diseases Therapeutics in CNS, Japan, Revenue Forecasts ($m), 2010–2017. 26
                                   Table 13:   ALS Therapeutics, Global, Revenues ($m), 2002–2010 ................................................ 28
                                   Table 14:   ALS Therapeutics, Global, Revenue Forecasts ($m), 2010–2017 ................................. 28
                                   Table 15:   ALS Therapeutics, Global, Revenue by Geographical Segmentation ($m), 2002–2010 29
                                   Table 16:   ALS Therapeutics, Global, Revenue by Geographical Segmentation ($m), 2010–2017 30
                                   Table 17:   ALS Therapeutics, Global, Annual Cost of Therapy ($), 2002–2010.............................. 31
                                   Table 18:   ALS Therapeutics, Global, Annual Cost of Therapy ($m), 2010–2017........................... 31
                                   Table 19:   ALS Therapeutics Market, Global, Generic and Branded Revenues ($m), 2010 and 2017
                                               ....................................................................................................................................... 33
                                   Table 20:   ALS Therapeutics Market, Global, Diseased Population (‘000), 2002–2010.................. 34
                                   Table 21:   ALS Therapeutics Market, Global, Diseased Population (‘000), 2010–2017.................. 35
                                   Table 22:   ALS Therapeutics Market, Global, Treatment Seeking Population (‘000), 2002–2010 .. 36
                                   Table 23:   ALS Therapeutics Market, Global, Treatment Seeking Population (‘000), 2010–2017 .. 36
                                   Table 24:   ALS Therapeutics, Global, Diagnosed Population (‘000), 2002–2010 ........................... 38
                                   Table 25:   ALS Therapeutics, Global, Diagnosed Population (‘000), 2010-2017 ............................ 38
                                   Table 26:   ALS Therapeutics, Global, Prescription Population (‘000), 2002–2010.......................... 40
                                   Table 27:   ALS Therapeutics, Global, Prescription Population (‘000), 2010–2017.......................... 40
                                   Table 28:   HD Therapeutics Market, Global, Revenues ($m), 2002–2010 ...................................... 44
                                   Table 29:   HD Therapeutics Market, Global, Revenue Forecasts ($m), 2010–2017 ....................... 44
                                   Table 30:   HD Therapeutics, Global, Revenue Segmentation by Country ($m), 2002–2010 .......... 46
                                   Table 31:   HD Therapeutics, Global, Revenue Forecasts Segmentation by Country ,($m), 2010–
                                               2017 ............................................................................................................................... 46
                                   Table 32:   HD Therapeutics, Global, Annual Cost of Therapy ($), 2002-2010 ................................ 47
                                   Table 33:   HD Therapeutics, Global, Annual Cost of Therapy ($), 2010–2017 ............................... 47
                                   Table 34:   HD Therapeutics Market, Global, Generic and Branded Revenues ($m), 2010 and 2017
                                               ....................................................................................................................................... 48
                                   Table 35:   HD Therapeutics, Global, Diseased Population (‘000), 2002–2010 ............................... 50
                                   Table 36:   HD Therapeutics, Global, Diseased Population (‘000), 2010–2017 ............................... 50
                                   Table 37:   HD Therapeutics, Global, Treatment Seeking Population (‘000), 2002–2010 ................ 51
                                   Table 38:   HD Therapeutics, Global, Treatment Seeking Population (‘000), 2010–2017 ................ 51
                                   Table 39:   HD Therapeutics, Global, Diagnosed Population (‘000), 2002–2010 ............................. 52
                                   Table 40:   HD Therapeutics, Global, Diagnosed Population (‘000), 2010–2017 ............................. 52
                                   Table 41:   HD Therapeutics, Global, Prescription Population (‘000), 2002–2010 ........................... 53
                                   Table 42:   HD Therapeutics, Global, Prescription Population (‘000), 2010–2017 ........................... 53
                                   Table 43:   MG Therapeutics, Global, Revenues ($m), 2002–2010 ................................................. 56
                                   Table 44:   MG Therapeutics, Global, Revenue Forecasts ($m), 2010–2017 .................................. 56
                                   Table 45:   MG Therapeutics, Global, Revenue Segmentation by Geography, ($m), 2002–2010 ... 57
                                   Table 46:   MG Therapeutics, Global, Revenue Forecasts Segmentation by Geography, ($m), 2010-
                                               2017 ............................................................................................................................... 58
                                   Table 47:   MG Therapeutics Market, Global, Branded and Generic Revenues ($m), 2010 and 2017
                                               ....................................................................................................................................... 59


Orphan Diseases Therapeutics in CNS to 2017 - Novel Agents                                                             GBIHC144MR / Published APR 2012
such as AMR101 and ACR16 to Provide Treatment Options and                                                                                            Page 5
Boost the Huntington’s Disease Segment                                            © GBI Research. This report is a licensed product and is not to be copied,
                                                                                                                  reproduced, shared or resold in any form
                                   Table 48:   MG Therapeutics, Global, Annual Cost of Therapy ($m), 2002–2010............................ 60
                                   Table 49:   MG Therapeutics, Global, Annual Cost of Therapy ($m), 2010–2017............................ 60
                                   Table 50:   MG Therapeutics, Global, Diseased Population (‘000), 2002–2010 .............................. 62
                                   Table 51:   MG Therapeutics, Global, Diseased Population (‘000), 2010–2017 .............................. 62
                                   Table 52:   MG Therapeutics, Global, Treatment Seeking Population (‘000), 2002–2010 ............... 63
                                   Table 53:   MG Therapeutics, Global, Treatment Seeking Population (‘000), 2010–2017 ............... 63
                                   Table 54:   MG Therapeutics, Global, Diagnosed Population (‘000), 2002–2010 ..........
								
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