神经肌肉疾病治疗药的全球市场 - 2022年～2029年

本报告提供全球神经肌肉疾病治疗药市场相关调查，提供市场概要，以及各类型，各疾病类型，各终端用户，各地区趋势，竞争情形，及加入此市场的主要企业简介等资讯。

目录

第1章 神经肌肉疾病治疗药市场调查手法和范围

第2章 神经肌肉疾病治疗药市场-市场定义和概要

第3章 神经肌肉疾病治疗药市场-摘要整理

• 各类型市场明细
• 各疾病类型市场明细
• 各终端用户市场明细
• 各地区市场明细

第4章 神经肌肉疾病治疗药市场-市场动态

• 影响市场的要素
  • 促进因素
  • 阻碍因素
  • 市场机会
  • 影响分析

第5章 神经肌肉疾病治疗药市场-产业分析

• 波特的五力分析
• 供应链分析
• 价格分析
• 法规分析
• 未满足需求

第6章 神经肌肉疾病治疗药市场-COVID-19分析

第7章 神经肌肉疾病治疗药市场-各类型

• 简介
• 市场规模分析，及与前一年同期比较成长分析(%)、各类型
• 市场魅力指数，各类型
  • 药物
  • 治疗
  • 其他

第8章 神经肌肉疾病治疗药市场-各疾病类型

• 简介
• 市场规模分析，及与前一年同期比较成长分析(%)、各疾病类型
• 市场魅力指数，各疾病类型
  • 脊髓性肌萎缩症(SMA)
  • 肌肉萎缩症
  • 肌病
  • 发炎性肌病
  • 其他

第9章 神经肌肉疾病治疗药市场-各终端用户

• 简介
  • 市场规模分析，及与前一年同期比较成长分析(%)、各终端用户
  • 市场魅力指数，各终端用户
• 医院
  • 简介
  • 市场规模分析，100万美元(2020年～2029年)，及与前一年同期比较成长分析(%)(2021年～2029年)
• 诊所
• 其他

第10章 神经肌肉疾病治疗药市场-各地区

• 简介
  • 市场规模分析，100万美元(2020年～2029年)，及与前一年同期比较成长分析(%)(2021年～2029年)，各地区
  • 市场魅力指数，各地区
• 北美
• 欧洲
• 南美
• 亚太地区
• 中东·非洲

第11章 神经肌肉疾病治疗药市场-竞争情形

• 主要的发展与策略
• 企业占有率分析
• 产品的基准

第12章 神经肌肉疾病治疗药市场-企业简介

• Sarepta Therapeutics Inc.
• Biogen Inc.
• Brainstorm Cell Therapeutics Inc.
• F. Hoffmann-La Roche Ltd.
• Novartis AG
• Pfizer Inc.
• PTC Therapeutics Inc.
• NMD Pharma
• Sanofi SA
• BioMarin

第13章 神经肌肉疾病治疗药市场-DataM

Market Overview

The global neuromuscular disease therapeutics market size was valued at US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).

Neuromuscular disorders affect the peripheral nervous system, including all motor and sensory nerves that connect the brain and spinal cord to the rest of the body. The most common symptom of these disorders is progressive muscle weakness.

Market Dynamics

The factors influencing the global neuromuscular disease therapeutics market are the increasing research and development and the growing prevalence of neuromuscular disease.

The increasing research and development is expected to drive the market growth in the forecast period

Neuromuscular diseases (NMDs) are a broad category of disorders that cause progressive motor function impairment. Motor neurons (MNs), skeletal muscle (SkM), or their synaptic connection, the neuromuscular junction, are the most common NMDs (NMJ). MNs [e.g., amyotrophic lateral sclerosis (ALS) caused by direct loss or retrograde degeneration of MNs], muscle [e.g., Duchenne muscular dystrophy (DMD) or myotonic dystrophy (DM)], NMJs [e.g., myasthenia gravis (MG) or congenital myasthenic syndromes (CMS)], or a combination of these [e.g. (e.g., Pompe disease). Regardless of its source, the structural and functional deficit in a targeted tissue will resonate throughout the motor unit, resulting in multiple shared symptoms among different NMDs.

Methods to differentiate hiPSCs into neuronal cells have advanced rapidly in the last decade, thanks to an increased understanding of early neural development and the commitment of neuroprogenitor cells to highly specialized neural subtypes, including MNs. MNs are found throughout the CNS and are divided into upper MNs (UMNs) and lower MNs (LMNs), which are developmentally and genetically distinct despite their shared nomenclature. Different NMDs can target UMNs and LMNs, making their distinction important when modelling NMDs. The cerebral cortex's pre-motor and primary motor regions are where UMNs originate. As a result, many recent breakthroughs in neuromuscular disease therapy development have been made. There are nearly 200 products in the pipeline for neuromuscular disorders, with a roughly 50:50 ratio of preclinical to clinical development. Most target amyotrophic lateral sclerosis (ALS) and Duchenne's muscular dystrophy (DMD). Recent technological advancements have aided the development of several different types of therapies. In 2018, small molecules (targeting receptor modulation, epigenetic reprogramming, redox metabolism, and other mechanisms), gene therapy, and antisense oligonucleotides (ASOs) accounted for nearly half of the nearly 200 potential therapies for neuromuscular diseases under development (Aitken et al., 2018). The US Food and Drug Administration (FDA) recently approved ASO and gene therapy.

The side effects associated with neuromuscular disease therapeutics are expected to hamper the market growth

Myasthenia gravis (MG) is a neuromuscular junction disorder that causes varying degrees of weakness in the body's skeletal muscles. Although there is currently no cure for MG, traditional therapies such as acetylcholinesterase inhibitors and immunomodulating therapies (corticosteroids, azathioprine, and mycophenolate mofetil) enable most patients to live productive lives with an average life expectancy. These therapies, however, are associated with side effects such as nausea, vomiting, gastrointestinal (GI) upset, increased infection risk, weight gain, and liver damage, which can deter patients from using them. Poor adherence is also a result of unsatisfactory treatment outcomes with traditional MG therapies, with 23 percent of patients reporting poor treatment compliance. Despite this, patient-reported information on the exact nature, duration, and severity of these side effects are scarce.

COVID-19 Impact Analysis

The COVID-19 pandemic has prompted a rapid reorganization of hospital settings and patient service provision to deal with emerging but unmet medical needs. The prevention strategies, in particular, have an impact on the management of patients with NMD. Immune-mediated pathogenesis is linked to some types of NMD. Patients with NMD who receive immunomodulatory therapy (IMT) are more likely to contract COVID-19 infections that are more severe. In order to guide patients and clinicians during the COVID-19 pandemic, a consensus statement on IMT management has recently emerged. Patient compliance and caregiver support, dose reduction of certain IMTs, or switching to alternative agents for high-risk NMD patients can all be considered depending on the pandemic burden of the region. NMD experts should be consulted before temporarily suspending, reducing, or changing IMT, and patients should not proceed without first consulting them. Hence, covid-19 is expected to have impacted the global neuromuscular disease therapeutics market due to delays in appointments and fear of contraction of covid.

Segment Analysis

Spinal Muscular Atrophy (SMA) segment is expected to dominate the market growth in the forecast period

Spinal muscular atrophy (SMA) is a genetic disorder characterized by muscle wasting (atrophy) and weakness (skeletal muscles). It is caused by the loss of motor neurons, which are specialized nerve cells that control muscle movement. When comparing muscles close to the center of the body (proximal) to muscles further away from the center of the body (distal), the weakness is more severe in the proximal muscles (distal). Muscle weakness usually worsens as people get older. Changes in the same genes cause many different types of spinal muscular atrophy. The types differ in terms of onset age and severity of muscle weakness, but there is some overlap. Other types of spinal muscular atrophy and related motor neuron diseases are caused by mutations in other genes, such as spinal muscular atrophy with progressive myoclonic epilepsy, spinal muscular atrophy with lower extremity predominance, and X-linked infantile spinal muscular atrophy, and spinal muscular atrophy with respiratory distress type 1. Around the world, 1 in 8,000 to 10,000 people suffer from spinal muscular atrophy. The most common spinal muscular atrophy type is type I, which accounts for roughly half of all cases.

Moreover, on 29th July 2021, Roche launched Evrysdi, a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children aged 2 months and older, in India, at a maximum retail price of around Rs 6 lakh per bottle. The US FDA first approved Evrysdi in August 2020, and it is now available in India just 11 months later. Hence, the increasing FDA approvals and growing prevalence of spinal muscular atrophy (SMA) are expected to boost the market growth.

Geographical Analysis

North America region is expected to hold the largest market share in the global neuromuscular disease therapeutics market

The increasing prevalence of neuromuscular diseases and increasing FDA approvals in this region is expected to boost the market growth. For instance, neuromuscular diseases (NMDs) are a broad category of disorders that affect an estimated 250,000 people in the United States.

Moreover, on 17th December 2021, Vyvgart (efgartigimod) is approved by the US Food and Drug Administration for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody.

Competitive Landscape

The global neuromuscular disease therapeutics market is moderately competitive with mergers, collaborations, and product launches. Some of the key players in the market are Biogen Inc., Brainstorm Cell Therapeutics Inc., F. Hoffmann-La Roche Ltd., Novartis AG, Pfizer Inc., PTC Therapeutics Inc., Sanofi SA, Sarepta Therapeutics Inc., NMD Pharma, BioMarin

Sarepta Therapeutics Inc.

Overview: Sarepta Therapeutics, Inc. is a medical research and drug development company based in Cambridge, Massachusetts, with headquarters and research facilities. The company was founded in 1980.

Product Portfolio: LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4) is a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins. SRP-9003 is being developed for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4). SRP-9003 is a gene therapy construct that transduces skeletal and cardiac muscle and delivers a gene that codes for the full-length beta-SG protein, the sole cause of the disease's progressive degeneration and shortened lifespan.

Key Development: On 2nd February 2022, Sarepta Therapeutics partnered with GenEdit to develop gene-editing therapies for neuromuscular diseases through a research partnership and option agreement. The alliance will use GenEdit's NanoGalaxy platform and Sarepta's gene-editing technology to develop the therapies.

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Target Audience

Service Providers/ Buyers

Industry Investors/Investment Bankers

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Manufacturers

Table of Contents

1. Neuromuscular Disease Therapeutics Market Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Neuromuscular Disease Therapeutics Market- Market Definition and Overview

3. Neuromuscular Disease Therapeutics Market- Executive Summary

• 3.1. Market Snippet By Type
• 3.2. Market Snippet By Disease Type
• 3.3. Market Snippet By End User
• 3.4. Market Snippet By Region

4. Neuromuscular Disease Therapeutics Market-Market Dynamics

• 4.1. Market Impacting Factors
  • 4.1.1. Drivers:
    • 4.1.1.1. The increasing research and development
  • 4.1.2. Restraints:
    • 4.1.2.1. The side effects associated with neuromuscular disease therapeutics
  • 4.1.3. Opportunity
  • 4.1.4. Impact Analysis

5. Neuromuscular Disease Therapeutics Market- Industry Analysis

• 5.1. Porter's Five Forces Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Regulatory Analysis
• 5.5. Unmet Needs

6. Neuromuscular Disease Therapeutics Market- COVID-19 Analysis

• 6.1. Analysis of Covid-19 on the Market
  • 6.1.1. Before COVID-19 Market Scenario
  • 6.1.2. Present COVID-19 Market Scenario
  • 6.1.3. After COVID-19 or Future Scenario
• 6.2. Pricing Dynamics Amid Covid-19
• 6.3. Demand-Supply Spectrum
• 6.4. Government Initiatives Related to the Market During Pandemic
• 6.5. Manufacturers Strategic Initiatives
• 6.6. Conclusion

7. Neuromuscular Disease Therapeutics Market- By Type

• 7.1. Introduction
• 7.2. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
• 7.3. Market Attractiveness Index, By Type
  • 7.3.1. Drugs
    • 7.3.1.1.1. Introduction
    • 7.3.1.1.2. Market Size Analysis, US$ Mn, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 7.3.2. Therapies
  • 7.3.3. Others

8. Neuromuscular Disease Therapeutics Market-By Disease Type

• 8.1. Introduction
• 8.2. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
• 8.3. Market Attractiveness Index, By Disease Type
  • 8.3.1. Spinal Muscular Atrophy (SMA)
    • 8.3.1.1.1. Introduction
    • 8.3.1.1.2. Market Size Analysis, US$ Mn, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
  • 8.3.2. Muscular dystrophy
  • 8.3.3. Myopathy
  • 8.3.4. Inflammatory Myopathies
  • 8.3.5. Others

9. Neuromuscular Disease Therapeutics Market- By End User

• 9.1. Introduction
  • 9.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
  • 9.1.2. Market Attractiveness Index, By End User
• 9.2. Hospitals
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
• 9.3. Clinics
• 9.4. Other

10. Neuromuscular Disease Therapeutics Market- By Region

• 10.1. Introduction
  • 10.1.1. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029, By Region
  • 10.1.2. Market Attractiveness Index, By Region
• 10.2. North America
  • 10.2.1. Introduction
  • 10.2.2. Key Region-Specific Dynamics
  • 10.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 10.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 10.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
  • 10.2.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 10.2.6.1. U.S.
    • 10.2.6.2. Canada
    • 10.2.6.3. Mexico
• 10.3. Europe
  • 10.3.1. Introduction
  • 10.3.2. Key Region-Specific Dynamics
  • 10.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 10.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 10.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
  • 10.3.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 10.3.6.1. Germany
    • 10.3.6.2. U.K.
    • 10.3.6.3. France
    • 10.3.6.4. Italy
    • 10.3.6.5. Spain
    • 10.3.6.6. Rest of Europe
• 10.4. South America
  • 10.4.1. Introduction
  • 10.4.2. Key Region-Specific Dynamics
  • 10.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 10.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 10.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
  • 10.4.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 10.4.6.1. Brazil
    • 10.4.6.2. Argentina
    • 10.4.6.3. Rest of South America
• 10.5. Asia Pacific
  • 10.5.1. Introduction
  • 10.5.2. Key Region-Specific Dynamics
  • 10.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 10.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 10.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
  • 10.5.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 10.5.6.1. China
    • 10.5.6.2. India
    • 10.5.6.3. Japan
    • 10.5.6.4. Australia
    • 10.5.6.5. Rest of Asia Pacific
• 10.6. Middle East and Africa
  • 10.6.1. Introduction
  • 10.6.2. Key Region-Specific Dynamics
  • 10.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 10.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Disease Type
  • 10.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User

11. Neuromuscular Disease Therapeutics Market- Competitive Landscape

• 11.1. Key Developments and Strategies
• 11.2. Company Share Analysis
• 11.3. Product Benchmarking

12. Neuromuscular Disease Therapeutics Market- Company Profiles

• 12.1. Sarepta Therapeutics Inc. *
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Key Highlights
  • 12.1.4. Financial Overview
• 12.2. Biogen Inc.
• 12.3. Brainstorm Cell Therapeutics Inc.
• 12.4. F. Hoffmann-La Roche Ltd.
• 12.5. Novartis AG
• 12.6. Pfizer Inc.
• 12.7. PTC Therapeutics Inc.
• 12.8. NMD Pharma
• 12.9. Sanofi SA
• 12.10. BioMarin

LIST NOT EXHAUSTIVE

13. Neuromuscular Disease Therapeutics Market- DataM

• 13.1. Appendix
• 13.2. About Us and Services
• 13.3. Contact Us