Pipeline Insight: Orphan Diseases in Neurology Part III: Myasthenia Gravis

Publication Date	November 2008
Publisher	Datamonitor
Product Type	Report
Pages	108
ISBN Number	not applicable
Product Code	DAT13861

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Summary

Introduction

The myasthenia gravis market is set to grow from $44m in 2007 to $239m in 2017 across the seven major pharmaceutical markets in Datamonitor's base scenario forecast. Amarin's pipeline candidate EN101 is forecast to become the market leading brand with sales of $153m in 2017.

Scope

Scenario based analysis and forecasts of the commercial potential of the myasthenia gravis market to 2017.
Analysis of the patient potential, commercial viability, and unmet needs of the myasthenia gravis market.
Indicationspecific sales forecasts of marketed and pipeline drugs to 2017.
Assessment of the treatment algorithms used in this disease.

Highlights

Although myasthenia gravis is an uncommon disease (106,000 patients across the seven major markets), it is more prevalent than Huntington's disease or amyotrophic lateral sclerosis (31,000 and 34,000 patients, respectively), both orphan neurological diseases with larger latestage pipelines. In the majority of patients, symptoms can be well controlled with current treatments. However, very poor sideeffect profiles and inconvenient dosing regimens of these therapies impact on the patient's quality of life, and represent key unmet needs in myasthenia gravis treatment. Amarin's EN101 is forecast to become the market leading brand with sales of $153m in 2017 in Datamonitor's base scenario. In an alternative scenario EN101 proves to be steroid sparing enhancing its commercial potential ($621m in 2017). However, poor bioavailability of RNA therapies enhances the risk of EN101 failing in larger scale trials.

Reasons to Purchase

Access the lucrative potential of this underdeveloped neurological autoimmune disease.
Benchmark key latestage compounds against current leading therapies.
Understand the opportunities in this market based on the comments of interviewed key opinion leaders.

Content

About Datamonitor Healthcare
- About The Cns Pharmaceutical Analysis Team
Chapter 1 Executive Summary
- Scope Of The Analysis
- Datamonitor Insight Into The Myasthenia Gravis Market
- Contributing Experts
- Related Reports
- Key Metrics
Chapter 2 Market Overview
- Definition Of Myasthenia Gravis
- Disease Overview
- Classification Of Myasthenia Gravis Subtypes
- Age Of Onset
- Presence Or Absence Of AntiAcetylcholine Antibodies
- Presence Of MuscleSpecific Receptor Tyrosine Kinase (Musk) Antibodies
- Severity
- Symptoms Of Myasthenia Gravis
- Diagnosis Of Myasthenia Gravis
- AntiAcetylcholine Receptor Antibodies
- Edrophonium Testing
- Electrophysiological Testing
- Etiology Of Myasthenia Gravis
- Prognosis
- Epidemiology Of Myasthenia Gravis
- More Prevalent Than Huntington's Disease Or Amyotrophic Lateral Sclerosis
- Trends In Myasthenia Gravis Epidemiology
- Prevalence Of Myasthenia Gravis
- Few Epidemiology Studies Have Been Conducted Investigating The Prevalence Of Myasthenia Gravis
- Population Size And Prevalence Rate Of Myasthenia Gravis
- Small Patient Population Hinders Research
- Treatment Tree
- Datamonitor's Myasthenia Gravis Treatment Tree
- FirstLine Mestinon
- SecondLine Therapy Steroid Immunosuppression Or Thymectomy
- Third, Fourth And FifthLine Therapy
- ShortTerm Therapies
- Published Treatment Algorithms And Recommendations
- Current Market Value And Trends
- Cost Of Current Treatments
- Unmet Need In Myasthenia Gravis
- More Favorable Dosing Regimens
- Improved SideEffect Profiles
- Polytherapy Adds To SideEffect Burden
- Mestinon (Pyridostigmine)
- Prednisolone And Prednisone
- DrugFree Remission
- Quicker Onset Of Action
- Quicker Diagnosis
- Commercial Viability Of The Myasthenia Gravis Market
- Positive Commercial Attributes Of The Disease
- High Unmet Need
- Chronic Condition
- Well Understood Etiology
- Low Competition
- Defined Patient Groups And Markets
- Low Generic Competition
- Negative Commercial Attributes Of The Disease
- Most Patients Respond To Current Treatments
- Cost Of Care Is Not Particularly High
- Not Typically LifeThreatening
- Patient Population Is Small
- Leading Brand Mestinon
- Seven Major Market Sales For 200407
- Generic Incursion In The Us Drives Down Brand Sales
- Mestinon Sales Constitute Over Half Of The Total Myasthenia Gravis Market
- Forecast To 2017
- Clinical Profile
- SideEffect Profile Is Undesirable
- Three Dosage Formulations Of Mestinon Are Available
- Patients May Take Up To 25 Tablets A Day
- Key Opinion LeaderReported Typical Dosing Regimens
Chapter 3 Future Market Potential
- Future Market
- Base Scenario En101 Only Proving To Be A Symptomatic Treatment
- SteroidSparing Scenario
- Non En101 Scenario
Chapter 4 Pipeline Overview And Dynamics
- Pipeline Drugs Market Potential
- Pipeline Overview
- Classification Of Pipeline Products
Chapter 5 ClinicalStage Drug Analysis & Forecasts
- En101
- Drug Overview
- Forecasts To 2017
- Market Penetration
- Marketing Factors
- En101 Pricing Assumptions Two Scenarios
- Marketing Resources
- Patient Potential
- Satisfaction Of Unmet Needs
- More Favorable Dosing Regimens
- Improved SideEffect Profile
- Quicker Onset Of Action
- Clinical Trial Data
- Interim Analysis Of Phase Iia Trial
- Completed Phase Ib Trial
- Scientific Background
- Rna Therapy
- Oral Delivery Of Rna Therapies Is A Challenge
- Animal Model Suggests That En101 Does Reach Muscles When Administered Orally
- Preclinical Investigations
- Rituxan (Rituximab)
- Drug Overview
- Forecast To 2017
- Marketing Factors
- Cost Of Rituxan/Mabthera In The Seven Major Markets
- Patient Population
- Patient Population For Rituxan Is Very Small
- Satisfaction Of Unmet Needs
- More Favorable Dosing Regimens
- DrugFree Remission
- Clinical Trial Data
- Other Drugs In Clinical Development For Myasthenia Gravis
- Kenketsu VenoglobulinIh (Polyethylene Glycol Treated Human Normal Immunoglobulin)
- Drug Overview
- Eculizumab
- Drug Overview
- Hematopoietic Stem Cell Therapy
- Drug Overview
Bibliography
- Journals
- Websites
Appendix
- Data Definitions, Limitations And Assumptions
- Standard Units
- Japanese Market Data
- Derivation Of Sales Forecasts And Pricing Trends
- Forecast Methodology
- Product Forecasts Base Scenario
- Product Forecasts SteroidSparing Scenario
- Contributing Experts
- About Datamonitor Healthcare
- About The Cns Analysis Team
- Disclaimer
List Of Tables
- Table 1: Patient Populations Of The Three Neurological Diseases Covered In Datamonitor's Orphan Disease Series, 2008
- Table 2: Population Size And Prevalence Rate Of Myasthenia Gravis Across The Seven Major Markets, 2008
- Table 3: Barohn (2008) Treatment Recommendations For Myasthenia Gravis, 2008
- Table 4: Annual Cost Of Therapy For Myasthenia Gravis In The Us, 2007
- Table 5: Neurological Conditions With Similar Symptoms To Myasthenia Gravis
- Table 6: Commercial Viability Of The Myasthenia Gravis Market, 2008
- Table 7: R&D ClinicalStage Pipeline In Myasthenia Gravis, 2008
- Table 8: Methodologies For En101 Forecasts In The Seven Major Markets, 2008
- Table 9: Datamonitor's En101 Pricing At Launch In The Us, Japan And 5eu
- Table 10: Cost Of Symptomatic Neurology Drugs With Orphan Disease Status In The Us, 2007
- Table 11: Cost Of DiseaseModifying Neurology Drugs In The Us, 2007
- Table 12: Cost Of Rituxan/Mabthera In The Us And 5eu For The Treatment Of Myasthenia Gravis, 2008
List Of Figures
- Figure 1: Seven Major Market Myasthenia Gravis Market Size, 20042017
- Figure 2: Myasthenia Gravis Treatment Tree Derived From Datamonitor's Primary And Secondary Research, 2008
- Figure 3: HiltonJones (2007) Treatment Algorithm For Ocular Myasthenia 2007
- Figure 4: HiltonJones (2007) Treatment Algorithm For Generalized Myasthenia, 2007
- Figure 5: Myasthenia GravisSpecific Sales Revenue In The Us, Japan And 5eu, 200407
- Figure 6: Myasthenia GravisSpecific Sales Volume (Ims Standard Units) In The Us, Japan And 5eu, 200407
- Figure 7: Regional Share Of Myasthenia Gravis Sales In The Seven Major Markets, 2007
- Figure 8: Unmet Need In Myasthenia Gravis Treatment, 2008
- Figure 9: Mestinon Myasthenia GravisSpecific Sales ($M), 200407
- Figure 10: Mestinon (Pyridostigmine Hydroxide) And Pyridostigmine Hydroxide Generics By Volume Of Ims Standard Units Sold, 200407
- Figure 11: Pyridostigmine (Mestinon And Generics) Share Of The Myasthenia Gravis Market Across The Seven Major Markets, 200407
- Figure 12: Mestinon (Pyridostigmine) Sales ($M) Forecast In The Seven Major Markets, 20072017
- Figure 13: Myasthenia Gravis Total Market Forecast To 2017 Across The Seven Major Markets; Base Scenario En101 Symptomatic Treatment Only
- Figure 14: Estimated Number Of Myasthenia Gravis Patients Taking Each Treatment In The Us; Base Scenario En101 Symptomatic Treatment Only, 20042017
- Figure 15: Myasthenia Gravis Total Market Forecast To 2017 Across The Seven Major Markets; En101 SteroidSparing Scenario
- Figure 16: Estimated Number Of Myasthenia Gravis Patients Taking Each Treatment In The Us In Datamonitor's En101 Steroid Sparing Scenario, 20042017
- Figure 17: Myasthenia Gravis Total Market Forecast To 2017 Across The Seven Major Markets; No En101 Scenario
- Figure 18: Summary Of Myasthenia Gravis Pipeline Drugs Potential ($M), 20082017
- Figure 19: Number Of Patients Forecast To Receive En101 Across The Seven Major Markets, 201017
- Figure 20: En101 Sales Revenue ($M) Forecast For Myasthenia Gravis Across The Seven Major Markets, 201017
- Figure 21: Number Of Patients Forecast To Receive Rituxan (Rituximab) Across The Seven Major Markets, 20092017
- Figure 22: Rituxan (Rituximab) Sales Revenue ($M) Forecast For Myasthenia Gravis Across The Seven Major Markets, 200917