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Global Genetic Disorders Drugs Market to 2023 - A Rapidly Growing Treatment Landscape Driven by Targeted Complement System Inhibitors and Enzyme Replacement Therapies to Treat PNH and Lysosomal Storage Disease

Global Genetic Disorders Drugs Market to 2023 - A Rapidly Growing Treatment Landscape Driven by Targeted Complement System Inhibitors and Enzyme Replacement Therapies to Treat PNH and Lysosomal Storage Disease

Summary


Genetic disorders are caused by abnormalities in a person’s genome and represent a broad therapy area, with over 200 individual diseases in the pipeline, including cystic fibrosis (CF), Duchenne muscular dystrophy (DMD), lysosomal storage disease (LSD) and paroxysmal nocturnal hemoglobinuria (PNH). Many genetic disorders are progressive, debilitating and/or life threatening, with a severe need of treatment.

In the US, an orphan drug is defined as a drug intended to treat a disease affecting fewer than 200,000 people. Orphan drugs are able to generate high revenue because of developmental incentives such as marketing exclusivity, which allows for longer periods before generics and biosimilars can enter the market. Furthermore, other incentives such as tax credits and accelerated approval lower the developmental costs faced by pharmaceutical companies, reducing the inherent financial risks involved and increasing the likelihood of recouping developmental costs from product sales.

Currently there are 1,390 products in active development for the treatment of genetic disorders, making this the sixth largest therapy area in terms of size. The key indications of LSD, CF and DMD have the highest levels of pipeline activity, with 174, 163 and 109 products in development respectively. While PNH has a relatively small pipeline with just 24 products in development, this indication includes Soliris, which generated revenue of $2.8 billion in 2016 and is anticipated to be the best-selling treatment within genetic disorders throughout the forecast period.Soliris, the best-selling drug within genetic disorders in 2016, is anticipated to remain the best-selling therapy within this cluster throughout the forecast period. The key patents for Soliris extend until 2021 in the US, and while there are Soliris biosimilars in development, they are not expected to enter the market during the forecast period. This lack of alternative treatments – as well as the rarity and life-threatening nature of the treated diseases – allow for high treatment costs and the generation of substantial revenue, despite the low prevalence of the diseases.

The report ""Global Genetic Disorders Drugs Market to 2023 - A Rapidly Growing Treatment Landscape Driven by Targeted Complement System Inhibitors and Enzyme Replacement Therapies to Treat PNH and Lysosomal Storage Disease"" provides an introduction to the genetic disease cluster, and some detailed background information regarding the key indications cystic fibrosis, Duchenne muscular dystrophy, lysosomal storage disease and paroxysmal nocturnal hemoglobinuria. Information is also included regarding symptoms, etiology and pathophysiology, comorbidities and epidemiology.

In depth, this report provides the following -

  • Provides an overview of the genetic disorders marketed products landscape, including product profiles of key marketed products, and revenue projections for each of these drugs.
  • Analyzes the genetic disorders pipeline, detailing, among other parameters, drug distribution by stage of development, molecule type and molecular target, along with an analysis of the key indications cystic fibrosis, Duchenne muscular dystrophy, lysosomal storage disease and paroxysmal nocturnal hemoglobinuria.
  • Presents forecast projections to 2023 on a global level, with forecasts provided for the US, plus aggregate revenue forecasts of the top five EU markets (the UK, France, Germany, Italy and Spain) and Japan. Additionally, revenue forecasts for the major product categories and key players are provided.
  • Assesses the company landscape for the disease cluster, with an analysis of the key players, the types of company involved, and whether the disease cluster comprises a large proportion of each company’s pipeline portfolio and total revenues.
  • Analyses trends in co-development and licensing deals relating to genetic disorder products. Some of the most prominent deals are discussed in detail.
Scope
  • Global revenue from the genetic disorders market is forecast to increase from $19.6 billion in 2016 to $47.7 billion in 2023, at a compound annual growth rate of 13.55%. What is driving this growth?
  • The leading companies in terms of market share are Alexion, Sanofi, Shire and Vertex. Which of these are forecast to experience the largest growth?
  • There are new approvals and late-stage products set to enter the market during the forecast period. Which drugs will achieve blockbuster status?
  • The market has been dominated by complement system inhibitors and enzyme replacement therapies. To what extent will these classes of drugs and others dominate the market over the forecast period?
  • There are 1,390 genetic disorders products in the pipeline. What molecular targets are most abundant in the pipeline and what role will pipeline product approvals play in market growth?
  • Genetic disorders clinical trials have an overall attrition rate of around 93%, what can companies do to maximize their chance of success?
Reasons to buy
  • Understand the current treatment landscape, with portfolios of key marketed products and a focus on historical and forecast sales patterns, and an overview of each drug’s mechanism of action.
  • Analyze the genetic disorders pipeline through a comprehensive review of the pipeline segmented by stage of development, molecule type and molecular target. This review also provides a detailed look at products for the treatment of genetic disorders to provide an insight into the risk associated with attempting to bring pipeline products to market.
  • Predict growth in market size, with in-depth market forecasting from 2016 to 2023. The forecasts will provide an understanding of how epidemiology trends, new drug entries, and patent expirations will influence market value.
  • Identify commercial opportunities in the genetic disorders deals landscape by analyzing trends in licensing and co-development deals.


  • GBI Research Report Guidance
  • Executive Summary
    • High Market Growth, Driven by Orphan Drugs
    • Key Drugs Expected to Retain Market Dominance
    • Large Pipeline and Strong Presence of Highly Specialized Companies
  • Introduction
    • Cystic Fibrosis
      • Duchenne Muscular Dystrophy
      • Lysosomal Storage Disease
      • Paroxysmal Nocturnal Hemoglobinuria
    • Symptoms
      • Table Genetic Disorders, Common Symptoms of Genetic Disorders
      • Table Genetic Disorders, Affected Areas of LSDs
    • Diagnosis
      • Cystic Fibrosis
        • Table Figure 1: Genetic Disorders, CF Testing Algorithm
      • Duchenne Muscular Dystrophy
      • Lysosomal Storage Disease
      • Paroxysmal Nocturnal Hemoglobinuria
    • Etiology and Pathophysiology
      • Cystic Fibrosis
      • Duchenne Muscular Dystrophy
      • Lysosomal Storage Disease
        • Table Genetic Disorders, Classification of Lysosomal Storage Diseases, 2017
      • Paroxysmal Nocturnal Hemoglobinuria
    • Epidemiology Patterns and Forecasts – Prevalence and Patient Segmentation
      • Table Genetic Disorders, Global, Epidemiology of Genetic Disorders, 2017
      • Cystic Fibrosis
        • Table Figure 2: Epidemiology Patterns for Cystic Fibrosis, 2016–2023
      • Duchenne Muscular Dystrophy
        • Table Figure 3: Epidemiology Patterns for Duchenne Muscular Dystrophy, 2016–2023
      • Lysosomal Storage Disease
        • Table Figure 4: Epidemiology Patterns for Lysosomal Storage Disease, 2016–2023
      • Paroxysmal Nocturnal Hemoglobinuria
        • Table Figure 5: Epidemiology Patterns for Paroxysmal Nocturnal Hemoglobinuria, 2016–2023
    • Co-morbidities and Complications
      • Cystic Fibrosis
      • Duchenne Muscular Dystrophy
      • Lysosomal Storage Disease
      • Paroxysmal Nocturnal Hemoglobinuria
    • Prognosis
      • Cystic Fibrosis
        • Table Genetic Disorders, CF Severity Classification, 2017
        • Table Figure 6: Genetic Disorders, Progression of CF
      • Duchenne Muscular Dystrophy
        • Table Stages of Duchenne Muscular Dystrophy, 2017
      • Lysosomal Storage Disease
      • Paroxysmal Nocturnal Hemoglobinuria
    • Treatment
      • Cystic Fibrosis
        • Table Genetic Disorders, Global, CF Treatment Plan, 2017
      • Duchenne Muscular Dystrophy
        • Table Genetic Disorders, Global, DMD Treatment Plan, 2017
        • Table Figure 7: Genetic Disorders, Schema for Initiation of Glucocorticoid Treatment in Duchenne Muscular Dystrophy, 2017
      • Lysosomal Storage Disease
      • Paroxysmal Nocturnal Hemoglobinuria
  • Key Marketed Products
    • Overview
      • Table Figure 8: Genetic Disorders, Global, Key Marketed Products and Approved Indications, 2017
    • Soliris (eculizumab) –Alexion
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Soliris, 2017
      • Table Figure 9: Genetic Disorders Market, Global, Annual Revenue for Soliris ($bn), 2007–2023
    • Orkambi (lumacaftor/ivacaftor) –Vertex
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Orkambi, 2017
      • Table Figure 10: Genetic Disorders Market, Global, Annual Revenue for Orkambi ($bn), 2015–2023
    • Cerezyme (imiglucerase) –Sanofi Genzyme
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Cerezyme, 2017
      • Table Figure 11: Genetic Disorders Market, Global, Annual Revenue for Cerezyme ($bn), 2006–2023
    • Myozyme/Lumizyme (alglucosidase alfa) –Sanofi Genzyme
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Myozyme/Lumizyme, 2017
      • Table Figure 12: Genetic Disorders Market, Global, Annual Revenue for Myozyme/Lumizyme ($bn), 2006–2023
    • Fabrazyme (agalsidase beta) –Sanofi Genzyme
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Fabrazyme, 2017
      • Table Figure 13: Genetic Disorders Market, Global, Annual Revenue for Fabrazyme ($bn), 2006–2023
    • Pulmozyme (dornase alfa) –Roche
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Pulmozyme, 2017
      • Table Figure 14: Genetic Disorders Market, Global, Annual Revenue for Pulmozyme ($bn), 2006–2023
    • Kalydeco (ivacaftor) –Vertex
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Kalydeco, 2017
      • Table Genetic Disorders Market, Global, CFTR Gene Mutations Responsive to Kalydeco, 2017
      • Table Figure 15: Genetic Disorders Market, Global, Annual Revenue for Kalydeco ($bn), 2012–2023
    • Elaprase (idursulfase) –Shire
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Elaprase, 2017
      • Table Figure 16: Genetic Disorders Market, Global, Annual Revenue for Elaprase ($bn), 2006–2023
    • Vimizim (elosulfase alfa) –Biomarin
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Vimizim, 2017
      • Table Figure 17: Genetic Disorders Market, Global, Annual Revenue for Vimizim ($bn), 2014–2023
    • Cerdelga (eliglustat tartrate) –Sanofi Genzyme
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Cerdelga, 2017
      • Table Figure 18: Genetic Disorders Market, Global, Annual Revenue for Cerdelga ($bn), 2014–2023
    • Exondys 51 (eterplirsen) –Sarepta
      • Table Genetic Disorders Market, Global, Approved Genetic Disorders Indications for Exondys 51, 2017
      • Table Figure 19: Genetic Disorders Market, Global, Annual Revenue for Exondys 51 ($bn), 2016–2023
    • Conclusion
  • Pipeline Landscape Assessment
    • Overview
      • Table Figure 20: Genetic Disorders Market, Global, Overall Pharmaceutical Industry Pipeline by Genetic Disorders, 2017
    • Pipeline Development Landscape
      • Table Figure 21: Genetic Disorders Market, Global, Genetic Disorders Pipeline by Stage of Development and Molecule Type, 2017
      • Table Figure 22: Genetic Disorders Market, Global, Pipeline for Key Genetic Disorders Indications by Stage of Development, 2017
    • Molecule Types in the Pipeline
      • Table Figure 23: Genetic Disorders Market, Global, Pipeline for Key Genetic Disorders Indications by Molecule Type, 2017
    • Molecular Targets in the Pipeline
      • Table Figure 24: Genetic Disorders Market, Global, Genetic Disorders Pipeline by Molecular Target, 2017
      • Table Figure 25: Genetic Disorders Market, Global, Pipeline for Key Genetic Disorders Indications by Molecular Target, 2017
    • Clinical Trials Landscape
      • Clinical Trial Failure Rates
        • Table Figure 26: Genetic Disorders Market, Global, Clinical Trial Attrition Rates by Stage of Development (%), 2006–2017
        • Table Figure 27: Genetic Disorders Market, Global, Clinical Trial Attrition Rates by Stage of Development and Indication (%), 2006–2017
        • Table Figure 28: Genetic Disorders Market, Global, Clinical Trial Attrition Rates by Stage of Development and Molecule Type (%), 2006–2017
        • Table Figure 29: Genetic Disorders Market, Global, Clinical Trial Attrition Rates by Stage of Development and Molecular Target (%), 2006–2017
      • Clinical Trial Duration
        • Table Figure 30: Genetic Disorders Market, Global, Clinical Trial Duration by Stage of Development (months), 2006–2017
        • Table Figure 31: Genetic Disorders Market, Global, Clinical Trial Duration by Stage of Development and Indication (months), 2006–2017
        • Table Figure 32: Genetic Disorders Market, Global, Clinical Trial Duration by Stage of Development and Molecule Type (months), 2006–2017
        • Table Figure 33: Genetic Disorders Market, Global, Clinical Trial Duration by Stage of Development and Molecular Target (months), 2006–2017
      • Clinical Trial Size
        • Table Figure 34: Genetic Disorders Market, Global, Clinical Trial Size by Stage of Development (participants), 2006–2017
        • Table Figure 35: Genetic Disorders Market, Global, Clinical Trial Size by Stage of Development and Indication (participants), 2006–2017
        • Table Figure 36: Genetic Disorders Market, Global, Clinical Trial Size by Stage of Development and Molecule Type (participants), 2006–2017
        • Table Figure 37: Genetic Disorders Market, Global, Clinical Trial Size by Stage of Development and Molecular Target (participants), 2006–2017
      • Cumulative Clinical Trial Size
        • Table Figure 38: Genetic Disorders Market, Global, Clinical Program Size by Stage of Development (participants), 2006–2017
        • Table Figure 39: Genetic Disorders Market, Global, Clinical Program Size by Stage of Development and Indication (participants), 2006–2017
        • Table Figure 40: Genetic Disorders Market, Global, Clinical Program Size by Stage of Development and Molecule Type (participants), 2006–2017
        • Table Figure 41: Genetic Disorders Market, Global, Clinical Program Size by Stage of Development and Molecular Target (participants), 2006–2017
    • Assessment of Key Pipeline Products
      • Epidiolex (cannabidiol) –GW Pharmaceuticals
        • Table Figure 42: Genetic Disorders Market, Global, Revenue Forecast for Epidiolex ($m), 2018–2023
      • ALXN-1210 (ravulizumab) –Alexion
        • Table Figure 43: Genetic Disorders Market, Global, Revenue Forecast for ALXN-1210 (ravulizumab) ($m), 2019–2023
      • Lanadelumab – Shire
        • Table Figure 44: Genetic Disorders Market, Global, Revenue Forecast for Lanadelumab ($m), 2018–2023
      • Burosumab – Ultragenyx and Kyowa Hakko Kirin
        • Table Figure 45: Genetic Disorders Market, Global, Revenue Forecast for Burosumab ($m), 2018–2023
      • GBT440 (voxelotor) –Global Blood Therapeutics
        • Table Figure 46: Genetic Disorders Market, Global, Revenue Forecast for GBT440 (voxelotor) ($m), 2020–2023
      • Ivacaftor and Tezacaftor – Vertex
        • Table Figure 47: Genetic Disorders Market, Global, Revenue Forecast for Ivacaftor and Tezacaftor ($m), 2018–2023
  • Multi-scenario Market Forecast to 2023
    • Overall Market Size
      • Table Figure 48: Genetic Disorders Market, Global, Market Size ($bn), 2016–2023
      • Table Genetic Disorders Market, Global, Annual Revenue Forecast for Key Products ($bn), 2016–2023
    • Generic Penetration
      • Table Figure 49: Genetic Disorders Market, Global, Annual Revenue Forecast for Premium and Generic Products ($bn), 2016–2023
      • Table Genetic Disorders Market, Global, Usage of Generics Across Key Indications, 2017
    • Revenue Forecast by Molecular Target
      • Immune/Inflammatory Mediators
        • Table Figure 50: Genetic Disorders Market, Global, Annual Revenue Forecast for Immune/Inflammatory Mediators ($bn), 2016–2023
      • Enzyme Replacement/Modifying Therapies
        • Table Figure 51: Genetic Disorders Market, Global, Annual Revenue Forecast for Enzyme Replacement/Modifying Therapies ($bn), 2016–2023
      • Ion Channel Modulators
        • Table Figure 52: Genetic Disorders Market, Global, Annual Revenue Forecast for Ion Channel Modulators ($bn), 2016–2023
      • G Protein-Coupled Receptor Modulators
        • Table Figure 53: Genetic Disorders Market, Global, Annual Revenue Forecast for G Protein-Coupled Receptor Modulators ($bn), 2016–2023
      • Metabolic Enzyme Mediators
        • Table Figure 54: Genetic Disorders Market, Global, Annual Revenue Forecast for Metabolic Enzyme Mediators ($bn), 2016–2023
  • Company Analysis and Positioning
    • Table Figure 55: Genetic Disorders Therapeutics Market, Global, Company Analysis Matrix, 2016–2023
    • Table Figure 56: Genetic Disorders Market, Global, Cluster by Growth and Market Share, 2016–2023
    • Revenue and Market Share Analysis by Company
      • Table Genetic Disorders Market, Global, Forecast Revenue by Company, 2016–2023
      • Table Figure 57: Genetic Disorders Market, Global, Forecast Market Share by Company (%), 2016–2023
      • Table Figure 58: Genetic Disorders Market, Global, Companies by Compound Annual Growth Rate (%), 2016–2023
      • Table Figure 59: Genetic Disorders Market, Global, Revenues by Product Type, 2016–2023
      • Alexion – How will Alexion overcome the threat posed by the emergence of Soliris biosimilars?
        • Table Figure 60: Genetic Disorders Market, Global, Alexion Annual Revenue Forecast ($bn), 2016–2023
      • Sanofi – How will revenue be affected by the expiry of key patents?
        • Table Figure 61: Genetic Disorders Market, Global, Sanofi Annual Revenue Forecast ($bn), 2016–2023
      • Vertex – Will Vertex continue to focus heavily on the treatment of CF?
        • Table Figure 62: Genetic Disorders Market, Global, Vertex Annual Revenue Forecast ($bn), 2016–2023
      • Shire – How will late-stage pipeline drug Lanadelumab affect Shire's revenue?
        • Table Figure 63: Genetic Disorders Market, Global, Shire Annual Revenue Forecast ($bn), 2016–2023
      • Amgen – Will Amgen develop more products for genetic diseases following the success of Repatha?
        • Table Figure 64: Genetic Disorders Market, Global, Amgen Annual Revenue Forecast ($bn), 2016–2023
      • GW Pharmaceuticals – How will Epidiolex drive sales for GW?
        • Table Figure 65: Genetic Disorders Market, Global, GW Pharmaceuticals Annual Revenue Forecast ($bn), 2016–2023
      • Pfizer – How will Pfizer's small genetic disorders pipeline affect longterm sales?
        • Table Figure 66: Genetic Disorders Market, Global, Pfizer Annual Revenue Forecast ($bn), 2016–2023
      • BioMarin – To what extent will BioMarin continue to focus exclusively on the treatment of genetic disorders?
        • Table Figure 67: Genetic Disorders Market, Global, BioMarin Annual Revenue Forecast ($bn), 2016–2023
      • Sarepta – Will Sarepta emerge as a key player in the genetic disorders landscape?
        • Table Figure 68: Genetic Disorders Market, Global, Sarepta Annual Revenue Forecast ($bn), 2016–2023
    • Company Landscape
      • Table Figure 69: Genetic Disorders Market, Global, Companies by Type, 2017
    • Marketed and Pipeline Portfolio Analysis
      • Table Figure 70: Genetic Disorders Market, Global, Pipeline, High-Activity and Late-Stage Developers by Level of Genetic Disorders Specialization, 2017
      • Table Figure 71: Genetic Disorders Market, Global, Proportion of Total Company Revenue Attributed to Genetic Disorders, 2016–2023
  • Strategic Consolidations
    • Licensing Deals
      • Deals by Region, Year and Value
        • Table Figure 72: Genetic Disorders Market, Global, Licensing Deals by Region, Year and Value, 2006–2017
        • Table Figure 73: Genetic Disorders Market, Global, Licensing Deals by Indication, 2006–2017
      • Deals by Stage of Development and Value
        • Table Figure 74: Genetic Disorders Market, Global, Licensing Deals by Stage of Development and Value, 2006–2016
      • Deals by Molecule Type, Molecular Target and Value
        • Table Figure 75: Genetic Disorders Market, Global, Licensing Deals by Molecule Type and Molecular Target, 2006–2017
      • Table for Licensing Deals with Disclosed Values
        • Table Genetic Disorders Market, Global, Licensing Deals, 2006–2017
    • Co-development Deals
      • Deals by Region, Year and Value
        • Table Figure 76: Genetic Disorders Market, Global, Co-development Deals by Region, Year and Value, 2006–2017
        • Table Figure 77: Genetic Disorders Market, Global, Co-development Deals by Indication, 2006–2017
      • Deals by Stage of Development and Value
        • Table Figure 78: Genetic Disorders Market, Global, Co-development Deals by Stage of Development and Value, 2006–2017
      • Deals by Molecule Type, Molecular Target and Value
        • Table Figure 79: Genetic Disorders Market, Global, Co-development Deals by Molecule Type and Molecular Target, 2006–2017
      • Table for Co-development Deals with Disclosed Values
        • Table Genetic Disorders Market Therapeutics Market, Global, Co-development Deals, 2006–2017
  • Appendix
    • References
    • Figures of All Clinical Stage Pipeline Products
      • Table Figure 80: Genetic Disorders, Global, Table of all Clinical Stage Pipeline Products, 2017
    • Abbreviations
    • Disease List
    • Methodology
      • Coverage
      • Secondary Research
      • Market Size and Revenue Forecasts
      • Pipeline Analysis
      • Competitive Landscape
    • Contact Us
    • Disclaimer

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