REFERENCE CODE GDHC235CFR PUBLICAT ION DATE M ARCH 2014 PARKINSON S DISEASE - US DRUG FORECAST AND MARKET ANALYSIS TO 2022
Sales for Parkinson s Disease in the US The Parkinson s disease market in the US is expected to grow at a compound annual growth rate (CAGR) of 7.3% from sales of $1.1 billion in 2012 to $2.3 billion in 2022. Major drivers of growth for the US Parkinson s disease market during the forecast period are: Healthcare reform. The figure given below presents the US sales for Parkinson s disease by Therapeutic Class during the forecast period from 2012 2022. Sales for Parkinson s Disease in US by Therapeutic Class, 2012 2022 27% 2012 Total: $1.1bn 0% 27% Launch of Neupro and Duodopa. Major barriers of growth for the US Parkinson s Levodopa disease market during the forecast period are: 26% 20% COMT Inhibitors High out-of-pocket expenses. Poor patient compliance in the US compared with the ROW. 19% 2022 Total: $2.3bn 2% Dopamine Agonists MAO-B Inhibitors A2A Antagonists 50% 18% 11% Source: GlobalData. 2
What Do the Physicians Think? Physicians state that dyskinesia remains a major unmet need and stress the impact that an antidyskinetic medication would have on the treatment of Parkinson s disease. Let s say if we do not consider what is untreatable today [balance, falls, dementia], then the main challenge is probably treating dyskinesia. [EU] KOL, November 2013 Wearing-off wouldn t be a problem, if the patients do not develop severe dyskinesia. Because if you can control dyskinesia, then you can use the drug [at a] high enough [dose] to control any motor fluctuation. So wearing-off itself, it s easier to treat. The problem is most patients with wearing-off, they do have dyskinesia too, and when you try to adjust the dose in order to control wearing-off, then the patient may develop dyskinesia, or a worsening form of dyskinesia. [EU] KOL, November 2013 I think sometimes we re a bit dismissive in saying we don t see the motor complications that we used to see, and I think that s true, because we ve got a range of different drugs. But, some people are really still struggling. Twenty percent of the day they re OFF, [while] twenty percent of the day they re dyskinetic. That s forty percent of the day that s bad for them, and we say, well, it s not as bad as the bad ol days, but it s still pretty bad for them We still don t really have an oral drug that is anti-dyskinetic. [EU] KOL, October 2013 The most challenging [unmet need] Every day I see a few patients for whom treatment is very challenging to me, particularly patients with marked wearing-off, with dyskinesia during ON, it s very difficult to treat with the current medication. If they are eligible for deep brain stimulation, it s okay, but patients over [age] 75, with marked wearing-off, dyskinesia, falling down, and freezing, it s very difficult to treat. And it s very challenging. [OUS] KOL, November 2013 Physicians believe that the introduction of slowrelease levodopa will have a significant impact on the market and be preferred over immediaterelease formulations. 3
Extended-release levodopa will take the place of regular drugs [immediate-release levodopa]. Even in the early phase of the disease, they are better for patients and will reduce the amount of fluctuations in later disease, as they progress. If they [slow-release levodopa therapies] were available, I would prescribe them over the immediate-release formulations [in early stage.] [OUS] KOL, November 2013 If extended release of levodopa are available we may choose such agents as initial therapies, not only in advanced cases but as initial therapy. [OUS] KOL, November 2013 Current therapeutic options are limited to symptomatic control and do not treat the underlying disease. Although there are no latestage therapies that will be launched to meet this need during the forecast period, physicians believe that early pipeline agents hold the promise of becoming one of the most significant advancements for PD in recent history. At this point, it s not [enough] to show that you can have an improvement of one hour of time. It s interesting, and it should be the first step. But we [are] wait[ing] for the next step; we [are] wait[ing] for drugs that have disease-modifying properties. Meaning that if we take [these drugs], we can have a better fate than not having these drugs for six month[s] or one year I m afraid that if a drug could arrive on the market, it will not have a huge impact if it just [demonstrates] symptomatic improvement of one hour of time. [EU] KOL, November 2013 Current treatment options are all symptomatic treatments, therefore many people want to discover disease-modifying treatments for Parkinson s disease, but none have been successful yet. [OUS] KOL, November 2013 4
1 1... 5 1.1 List of Tables... 10 1.2 List of Figures... 14 2 Introduction... 15 2.1 Catalyst... 15 2.2 Related Reports... 15 3 Disease Overview... 18 3.1 Etiology and Pathophysiology... 18 3.1.1 Etiology... 18 3.1.2 Pathophysiology... 21 3.1.3 Prognosis... 23 3.1.4 Quality of Life... 24 3.2 Symptoms... 24 4 Disease Management... 26 4.1 Overview... 26 4.1.1 Diagnosis The UK Brain Bank Criteria... 26 4.1.2 Treatment Guidelines and Leading Prescribed Drugs... 27 4.2 Treatment Synopsis... 28 4.2.1 Dopaminergic Therapy Classes... 29 4.2.2 Treatment of Parkinson s disease by Stage... 31 4.2.3 Other Treatment Options... 34 5
4.3 Parkinson s Disease Assessment Scales... 34 4.3.1 Unified Parkinson s Disease Rating Scale (UPDRS)... 35 4.3.2 Hoehn and Yahr Clinical Staging... 37 4.3.3 Other Clinical Assessments... 38 4.4 Diagnosis and Treatment of Parkinson s Disease by Country... 38 4.4.1 US... 38 5 Competitive Assessment... 41 5.1 Overview... 41 5.2 Strategic Competitor Assessment... 43 5.3 Product Profiles Levodopa Combination Therapy... 44 5.3.1 Sinemet (carbidopa/levodopa)... 45 5.3.2 Duodopa (carbidopa/levodopa intestinal gel)... 49 5.4 Product Profiles COMT Inhibitors... 53 5.4.1 Stalevo/Comtan (entacapone)... 53 5.5 Dopamine Agonists... 58 5.5.1 Neupro (rotigotine transdermal patch)... 59 5.5.2 Requip/Requip XL (ropinirole)... 65 5.5.3 Apokyn (apomorphine)... 68 5.6 Product Profiles MAO-B Inhibitors... 72 5.6.1 Azilect (rasagiline)... 72 5.7 Product Profiles Adenosine 2A Inhibitor... 77 5.7.1 Nouriast (istradefylline)... 78 5.8 Product Profiles Other Therapies... 81 6
6 Opportunity and Unmet Need... 82 6.1 Overview... 82 6.2 Treatment of Motor Complications Dyskinesias and OFF Episodes... 84 6.2.1 Unmet Need... 84 6.2.2 Gap Analysis... 86 6.2.3 Opportunity... 89 6.3 Treatment of Non-Motor Symptoms and Dementia... 89 6.3.1 Unmet Need... 89 6.3.2 Gap Analysis... 90 6.3.3 Opportunity... 92 6.4 Neuroprotective/Disease-Modifying Agents... 92 6.4.1 Unmet Need... 92 6.4.2 Gap Analysis... 93 6.4.3 Opportunity... 96 6.5 Improved Drug Formulations... 97 6.5.1 Unmet Need... 97 6.5.2 Gap Analysis... 97 6.5.3 Opportunity... 98 6.6 Identification of Reliable Biomarkers... 98 6.6.1 Unmet Need... 98 6.6.2 Gap Analysis... 99 6.6.3 Opportunity... 100 6.7 Improved Clinical Trial Design... 100 7
6.7.1 Unmet Need... 100 6.7.2 Gap Analysis... 101 6.7.3 Opportunity... 101 7 Pipeline Assessment... 102 7.1 Overview... 102 7.2 Promising Drugs in Clinical Development... 102 7.2.1 Safinamide... 104 7.2.2 Tozadenant... 111 7.2.3 CVT-301... 114 7.2.4 Rytary/IPX066... 119 7.2.5 Opicapone... 125 7.2.6 Mavoglurant/AFQ056... 129 7.2.7 CD/LD-GR... 132 7.2.8 Other Late-Stage Pipeline Products... 137 8 Current and Future Players... 138 8.1 Overview... 138 8.2 Trends in Corporate Strategy... 139 8.3 Company Profiles... 140 8.3.1 Merck... 140 8.3.2 Roche... 142 8.3.3 AbbVie... 144 8.3.4 UCB... 146 8.3.5 GlaxoSmithKline... 147 8
8.3.6 Novartis... 149 8.3.7 Orion... 152 8.3.8 Newron... 153 8.3.9 Civitas... 155 8.3.10 Impax... 157 8.3.11 Lundbeck... 159 9 Market Outlook... 162 9.1 US... 162 9.1.1 Forecast... 162 9.1.2 Key Events... 166 9.1.3 Drivers and Barriers Global Issues... 166 9.1.4 Drivers and Barriers... 169 10 Appendix... 171 10.1 Bibliography... 171 10.2 Abbreviations... 186 10.3 Methodology... 190 10.4 Forecasting Methodology... 190 10.4.1 Diagnosed Parkinson s Disease Patients... 190 10.4.2 Percent Drug-Treated Patients... 191 10.4.3 Drugs Included in Each Therapeutic Class... 191 10.4.4 Launch and Patent Expiry Dates... 192 10.4.5 General Pricing Assumptions... 193 10.4.6 Compliance Assumptions... 193 9
10.4.7 Individual Drug Assumptions... 194 10.4.8 Generic Erosion... 202 10.4.9 Pricing of Pipeline Agents... 202 10.5 Physicians and Specialists Included in this Study... 204 10.6 About the Authors... 206 10.6.1 Author... 206 10.6.2 Global Head of Healthcare... 207 10.7 About GlobalData... 208 10.8 Disclaimer... 208 1.1 List of Tables Table 1: Symptoms of Parkinson s Disease... 25 Table 2: UK Brain Bank Diagnostic Criteria... 27 Table 3: Diagnosis and Treatment Guidelines for Parkinson s Disease... 28 Table 4: Most Prescribed Drugs for Parkinson s Disease by Class in the Global Markets, 2014... 28 Table 5: Dopaminergic Therapy in Parkinson s Disease... 31 Table 6: UPDRS Clinical Assessment of Disease Severity... 36 Table 7: Parkinson s Disease Assessment Scales Used in Clinical Trials... 38 Table 8: Parkinson s Disease, Country Profile US... 40 Table 9: Treatment of Motor Symptoms in Parkinson s Disease... 42 Table 10: Leading Treatments for Parkinson s Disease, 2014... 44 Table 11: Product Profile Sinemet... 47 Table 12: Sinemet SWOT Analysis, 2014... 49 Table 13: Product Profile Duodopa... 51 10
Table 14: Duodopa SWOT Analysis, 2014... 53 Table 15: Product Profile Stalevo... 55 Table 16: Product Profile Comtan... 56 Table 17: Stalevo/Comtan SWOT Analysis, 2014... 58 Table 18: Product Profile Neupro... 61 Table 19: Neupro SWOT Analysis, 2014... 65 Table 20: Product Profile Requip/Requip XL... 66 Table 21: Requip/Requip XL SWOT Analysis, 2014... 68 Table 22: Product Profile Apokyn... 70 Table 23: Apokyn SWOT Analysis, 2014... 71 Table 24: Product Profile Azilect... 73 Table 25: Azilect SWOT Analysis, 2014... 77 Table 26: Product Profile Nouriast... 79 Table 27: Nouriast SWOT Analysis, 2014... 80 Table 28: Summary of Alternative Parkinson s Disease Therapies... 81 Table 29: Unmet Need and Opportunity in Parkinson s Disease... 83 Table 30: Dyskinesia Pipeline, 2014... 88 Table 31: Dementia Pipeline, 2014... 91 Table 32: Parkinson s Disease-Modifying Therapeutics Pipeline, 2014... 95 Table 33: Product Profile Safinamide... 106 Table 34: Summary of Relevant Clinical Trials for Safinamide... 107 Table 35: Safinamide SWOT Analysis, 2014... 110 Table 36: Product Profile Tozadenant... 112 Table 37: Tozadenant SWOT Analysis, 2014... 114 11
Table 38: Product Profile CVT-301... 116 Table 39: CVT-301 SWOT Analysis, 2014... 119 Table 40: Product Profile Rytary... 121 Table 41: Rytary SWOT Analysis, 2014... 125 Table 42: Product Profile Opicapone... 126 Table 43: Opicapone SWOT Analysis, 2014... 128 Table 44: Product Profile Mavoglurant... 130 Table 45: Mavoglurant SWOT Analysis, 2014... 132 Table 46: Product Profile CD/LD-GR... 133 Table 47: Summary of Relevant Clinical Trials for CD/LD-GR... 134 Table 48: CD/LD-GR SWOT Analysis, 2014... 136 Table 49: Late-Stage Pipeline, 2013... 137 Table 50: Key Companies in the Parkinson s Market, 2014... 138 Table 51: Merck s Parkinson s Disease Portfolio Assessment, 2014... 142 Table 52: Merck s PD SWOT Analysis, 2014... 142 Table 53: Roche s Parkinson s Disease Portfolio Assessment, 2014... 144 Table 54: Roche s PD SWOT Analysis, 2014... 144 Table 55: AbbVie s Parkinson s Disease Portfolio Assessment, 2014... 145 Table 56: AbbVie s PD SWOT Analysis, 2014... 145 Table 57: UCB s Parkinson s Disease Portfolio Assessment, 2014... 146 Table 58: UCB s PD SWOT Analysis, 2014... 147 Table 59: GSK s Parkinson s Disease Portfolio Assessment, 2014... 149 Table 60: GSK s PD SWOT Analysis, 2014... 149 Table 61: Novartis Parkinson s Disease Portfolio Assessment, 2014... 151 12
Table 62: Novartis PD SWOT Analysis, 2014... 151 Table 63: Orion s Parkinson s Disease Portfolio Assessment, 2014... 153 Table 64: Orion s PD SWOT Analysis, 2014... 153 Table 65: Newron s Parkinson s Disease Portfolio Assessment, 2014... 154 Table 66: Newron s PD SWOT Analysis, 2014... 155 Table 67: Civitas Parkinson s Disease Portfolio Assessment, 2014... 156 Table 68: Civitas PD SWOT Analysis, 2014... 157 Table 69: Impax s Parkinson s Disease Portfolio Assessment, 2014... 159 Table 70: Impax s PD SWOT Analysis, 2014... 159 Table 71: Lundbeck s Disease/Therapy Portfolio Assessment, 2014... 161 Table 72: Lundbeck s SWOT Analysis, 2014... 161 Table 73: Sales Forecasts ($m) for Parkinson s Disease in the US, 2012 2022... 164 Table 74: Key Events Impacting Sales for Parkinson s Disease in the US, 2012 2022... 166 Table 75: Parkinson s Disease Market Drivers and Barriers, 2012 2022... 166 Table 76: Parkinson s Disease Market in the US Drivers and Barriers, 2012 2022... 169 Table 77: Key Launch Dates... 192 Table 78: Key Patent Expiries... 193 13
1.2 List of Figures Figure 1: Overview L-dopa Metabolism and Inhibitor Classes... 30 Figure 2: Overview Treatment of Motor Symptoms of Parkinson s Disease... 32 Figure 3: Pharmacokinetics of Levodopa... 85 Figure 4: Parkinson s Disease Phase II III Pipeline, Segmented by Indication, 2014... 103 Figure 5: Competitive Assessment of Late-Stage Pipeline Agents in Parkinson s Disease, 2012 2022... 104 Figure 6: Company Portfolio Gap Analysis in Parkinson s Motor Symptoms, 2012 2022... 139 Figure 7: Sales for Parkinson s Disease in the US by Drug Class, 2012 2022... 165 14
Introduction 2 Introduction 2.1 Catalyst The Parkinson s disease market is expected to grow from $3.6 billion to $5.3 billion over the 10- year forecast period. A major driving force behind this is the increase in the global population and advancements in healthcare that contribute to an aging population at increased risk for Parkinson s disease. The population of Parkinson s disease patients is expected to increase from 3.2 million people in 2012 to 4.3 million in 2022 in the eight major markets covered. The market for Parkinson s disease is expected to grow as it is the second most common neurological disorder, with an increased prevalence in the elderly. Parkinson s disease has had a history of successful drugs that are highly effective; however, unmet needs remain. Advancements in technology and drug delivery systems have driven growth in this market during the forecast period and made it a less risky market than other neurological conditions, while still holding potential for a big payout. While all products to this point have treated the signs and symptoms of Parkinson s disease rather than the underlying condition, the growing understanding of the nervous system holds promise for a breakthrough in the development of disease-modifying agents. Ample opportunity in the Parkinson s disease market remains. As most products have been launched by collaborative efforts of at least two companies, we expect such strategic partnerships to continue during the coming decade in the market for Parkinson s disease. 2.2 Related Reports GlobalData (2013) EpiCast: Parkinson s Disease Epidemiology Forecast to 2022, November 2013, GDHCER043 GlobalData (2012) PharmaPoint: Alzheimer s Disease Global Drug Forecast and Market Analysis to 2022, July 2013 GDHC016PIDR. GlobalData (2014). PharmaPoint: Parkinson s Disease Global Drug Forecast and Market Analysis to 2022, March 2014, GDHC82PIDR GlobalData (2014). Parkinson s Disease 5EU Drug Forecast and Market Analysis to 2022, March 2014, GDHC236CFR 15
Introduction GlobalData (2014). Parkinson s Disease Japan Drug Forecast and Market Analysis to 2022, March 2014, GDHC237CFR GlobalData (2014). Parkinson s Disease Brazil Drug Forecast and Market Analysis to 2022, March 2014, GDHC238CFR GlobalData (2014). Madopar (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC389DFR GlobalData (2014). Sinemet (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC390DFR GlobalData (2014). Duodopa (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC391DFR GlobalData (2014). Stalevo/Comtan (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC392DFR GlobalData (2014). Neupro (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC393DFR GlobalData (2014). Requip/Requip XL (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC394DFR GlobalData (2014). Apokyn (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC395DFR GlobalData (2014). Azilect (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC396DFR GlobalData (2014). Nouriast (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC397DFR GlobalData (2014). Safinamide (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC398DFR GlobalData (2014). Tozadenant (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC399DFR 16
Introduction GlobalData (2014). CVT-301 (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC400DFR GlobalData (2014). Rytary/IPX066 (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC401DFR GlobalData (2014). Opicapone (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC402DFR GlobalData (2014). Mavoglurant/AFQ056 (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC403DFR GlobalData (2014). CD/LD-GR (Parkinson s Disease) Forecast and Market Analysis to 2022, March 2014, GDHC404DFR GlobalData (2014). Parkinson s Disease Current and Future Players, March 2014, GDHC1033FPR 17
Appendix 10.7 About GlobalData GlobalData is a leading global provider of business intelligence in the Healthcare industry. GlobalData provides its clients with up-to-date information and analysis on the latest developments in drug research, disease analysis, and clinical research and development. Our integrated business intelligence solutions include a range of interactive online databases, analytical tools, reports and forecasts. Our analysis is supported by a 24/7 client support and analyst team. GlobalData has offices in New York, Boston, London, India and Singapore. 10.8 Disclaimer All Rights Reserved. No part of this publication may be reproduced, stored in a retrieval system or transmitted in any form by any means, electronic, mechanical, photocopying, recording or otherwise, without the prior permission of the publisher, GlobalData. 208