肢帯型筋ジストロフィー治療の世界市場-2023年～2030年

概要

世界の肢帯型筋ジストロフィー治療市場は、そのダイナミクスに影響を与える様々な要因によって、長年にわたって著しい成長と変貌を遂げてきました。肢帯型筋ジストロフィーとして知られる一連の遺伝性疾患は、筋肉の減少と衰弱（筋ジストロフィー）を引き起こします。この病気（常染色体劣性遺伝）を発症するには、多くの場合、両親ともに欠陥のある（動作不能な）遺伝子を受け継がなければなりません。

特に新興国における政府投資と研究開発は、肢帯型筋ジストロフィー1型および2型に対する遺伝子治療や副腎皮質ステロイドなどの新規治療に対する先端技術の利用を引き続き促進し、世界の肢帯型筋ジストロフィー治療市場を押し上げると思われます。

市場促進要因：市場抑制要因

共同研究および薬事承認の増加

先進国は、高額の投資、所得水準、インフラ整備に牽引され、医療分野の改善において急速な成長を遂げています。肢帯型筋ジストロフィー治療に対する需要の高まりは、研究の活発化に起因しています。さまざまな国との共同研究、買収、技術進歩に伴うライセンス契約は、市場の成長を促進する重要な要因です。

2023年7月12日、遺伝子治療分野における生物生産を強化するための世界初の効果的なデジタルモデルを作成するため、欧州の基幹システム向け人工知能開発企業であるタレスと、希少遺伝性疾患の遺伝子治療開発と研究の革新的リーダーであるジェネソンが、それぞれの専門知識を結集します。遺伝子治療の開発は、非常に難しく、複雑で、高価で、時間のかかる手順です。製造コストと時間の削減は重要な課題です。

さらに、2023年2月16日、肢帯型筋ジストロフィー（LGMD）治療薬AB-1003（別名LION-101）が欧州委員会（EC）から希少疾病用医薬品（オーファンドラッグ）の指定を受けたと、バイエルAGの完全子会社で独立経営のアスクレピオス・バイオファーマシューティカル社が発表しました。

さらに、肢帯型筋ジストロフィーに対する新薬やソリューションの利用が増加しています。筋ジストロフィー患者の増加、革新的な製品開発のための研究とともに複数の薬剤の利用が増加していることは、肢帯型筋ジストロフィー治療市場の成長を促進する主な要因です。

治療に伴う副作用

遺伝子治療はまだ比較的新しい医療形態ですが、幅広い疾患を治療できる可能性があります。ある種のがん、アレルギー反応、注射を伴う場合の臓器や組織の損傷などは、潜在的な危険性の一例です。遺伝子治療薬は、最近の開発により、より安全性が高まっています。初期の調査では、毒性、炎症、悪性腫瘍など、遺伝子治療に伴う健康被害が非常に大きいことが指摘されていました。

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • 共同研究の増加と規制当局の承認
    • 技術進歩と開発の高まり
  • 抑制要因
    • 治療に伴う副作用
  • 機会
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• サプライチェーン分析
• 価格分析
• 規制分析
• SWOT分析
• 特許分析
• ロシア・ウクライナ戦争の影響分析
• DMIの見解

第6章 COVID-19分析

第7章 タイプ別

• 肢帯型筋ジストロフィー1型
• 肢帯型筋ジストロフィー2型

第8章 治療法別

• 薬物療法
  • コルチコステロイド
    • プレドニン
    • デフラザコート（エムフラザ）
  • 低分子薬
    • リビトール（BBP-418）
• 遺伝子治療
• その他

第9章 エンドユーザー別

• 病院
• 外来手術センター
• その他

第10章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • イタリア
  • スペイン
  • その他欧州
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋
  • 中国
  • インド
  • 日本
  • オーストラリア
  • その他アジア太平洋
• 中東・アフリカ

第11章 競合情勢

• 競合シナリオ
• 市況/シェア分析
• M&A分析

第12章 企業プロファイル

• Jubilant Cadista Pharmaceuticals Inc.
  • 会社概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な動向
• Sarepta Therapeutics, Inc.
• ML Bio Solutions
• Genethon
• Asklepios BioPharmaceutical, Inc.
• Atamyo Therapeutics
• Vita Therapeutics, Inc.
• Edgewise Therapeutics
• BridgeBio Inc.
• Hansa Biopharma

第13章 付録

Overview

The global limb girdle muscular dystrophy treatment market has witnessed significant growth and transformations over the years, with various factors influencing its dynamics. A vast set of hereditary illnesses known as limb-girdle muscular dystrophies cause muscle loss and weakening (muscular dystrophy). To develop the illness (autosomal recessive inheritance), often both parents have to pass on the defective (inoperative) gene.

Government investments and research, particularly in developing economies, will continue to drive utilization of advanced technology for novel treatments such as gene therapy or corticosteroids for limb-girdle muscular dystrophy type 1 and type 2 and boost the global limb girdle muscular dystrophy treatment market.

Market Dynamics: Drivers & Restraints

Growing collaborations and regulatory approvals

Developed economies have been witnessing rapid growth in betterment of their healthcare sectors, driven by high investments, income levels, and infrastructure development. Several countries have experienced substantial demand for limb girdle muscular dystrophy treatment owing to the rising research. Various collaborations, acquisitions and license agreement with technological advancements among different countries will be a crucial factor driving the growth of the market.

On July 12, 2023, in order to create the first of its kind effective digital models for enhancing bioproduction produces in the area of gene therapy, Thales, a European developer in artificial intelligence for mission-critical systems, and Genethon, an innovator and leader in genetic therapy development and research for rare genetic diseases, are bringing together their expertise. Gene therapy treatment development is a very difficult, intricate, expensive, and time-consuming procedure. The reduction of production costs and time represents a significant challenge.

Additionally, on February 16, 2023, the limb-girdle muscular dystrophy (LGMD) medicine AB-1003 (also known as LION-101) has been given orphan drug designation by the European Commission (EC), according to Asklepios BioPharmaceutical, Inc., a fully owned and independently run subsidiary of Bayer AG.

Furthermore, the rising utilization of novel medicines and solutions for limb girdle muscular dystrophy. Rising muscular dystrophy cases and increasing utilization of several drugs along with research for innovative product development will be a major factor driving the growth of the limb girdle muscular dystrophy treatment market.

Side effects associated with the therapy

Although they are still relatively new forms of medicine, genetic treatments offer the potential to treat a wide range of illnesses. Certain cancers, allergic reactions, or organ or tissue damage if an injection is involved are examples of potential dangers. Genetic medicines are currently more safer owing to recent developments. Early research indicated very substantial health hazards associated with gene therapy, including toxicity, inflammation, and malignancy.

Segment Analysis

The global limb girdle muscular dystrophy treatment market is segmented based on type, treatment, end user and region.

Limb-girdle muscular dystrophy type 2 type segment accounted for approximately 56% of market share

Calpainopathy, also known as limb-girdle muscular dystrophy form 2A, is brought on by CAPN3 gene mutations. About 30% of cases of limb-girdle muscular dystrophy are Type 2A, which is also the most prevalent type. Dysferlinopathy is also known as limb-girdle muscular dystrophy form 2B and is brought on by DYSF gene mutations.

Initial symptoms of LGMD2A include frequently weakness and wastage (loss of muscular mass) in the shoulder, hip, and thigh muscles. Leg involvement is apparent earlier shoulder and arm involvement, and this weakness typically affects each side of the body equally.

On September 19, 2023, ATA-200, a gene therapy for type 2C/R5 limb-girdle muscular dystrophy (LGMD2C/R5) that is related to -sarcoglycan (SGCG), was the subject of a Clinical Trial Application (CTA) filed in Europe by Atamyo Therapeutics, a biotechnology company specializing in the creation of new-generation gene treatments targeting neuromuscular diseases. The business also said that ATA-200 has been approved for non-dilutive funding from France 2030, a public program run by Bpifrance, in the amount of up to €8 million ($8.6 million), to support their clinical trial and industrial development projects.

Geographical Penetration

North America accounted for around 39% of market share in 2022

Due to the rising need for limb girdle muscular dystrophy treatment in healthcare, manufacturers in North America have chances of increasing their operations. There are many producers and suppliers in North America and owing to the quick economic growth of the region, industrial production has expanded, driving the demand for limb girdle muscular dystrophy treatment.

Increasing expenditure on healthcare, advancement of technologies and different types of medications for several diseases, along with increase in biopharmaceutical or biotechnology business establishment across the region are also contributing to the growth of limb girdle muscular dystrophy treatment market share of this region. The market in this area is growing as people become more aware of various novel treatments. The aforementioned elements attest to North America's hegemonic position in the world.

North America continues to be a key player in the global limb girdle muscular dystrophy treatment market, with United States leading the way. Government initiatives promoting infrastructure development and investment, and a focus on rising advancements have fueled the demand for limb girdle muscle medications in the United States have been proactive in executing several initiatives or surgical procedures, stimulating these medications demand.

COVID-19 Impact Analysis

The outbreak of the COVID-19 pandemic in late 2019 created unprecedented challenges for industries worldwide, including the global limb girdle muscular dystrophy treatment market. As countries grappled with lockdowns, supply chain disruptions and reduced economic activity, the pharmaceutical sector, with a significant consumer of corticosteroid or small molecule, was significantly impacted. The widespread lockdowns and restrictions imposed by the pandemic, which began in early 2020, had an effect on a number of initiatives all around the world.

Major medical and biotechnology industries came to a standstill and shifted their attention towards the management of COVID-19, leading to a slump in demand for limb girdle muscular dystrophy treatment. Now several research studies have been initiated and companies have again started trial for their products efficiency. Overall, the impact of the pandemic on the global limb girdle muscular dystrophy treatment market is expected to be relatively moderate, with the market continuing to grow steadily due to the ongoing need and research for novel treatments.

Key Developments

• On August 3, 2023, the initial participant in the Phase 1 / Phase 2 LION-CS101 research study for people having limb-girdle muscular dystrophy subtype 2I/R9 (LGMD2I/R9) was recently dosed, according to Asklepios BioPharmaceutical, Inc., a genetic therapy company that is entirely owned and independently run as a subsidiary of Bayer AG. A brand-new FKRP replacement gene therapy under research is called AB-1003 (also referred to as LION-101).
• On June 22, 2023, the FDA accelerated the authorization of delandistrogene moxeparvovec-rokl (ELEVIDYS), an adeno-associated virus-based gene therapy, which is intended for the management of ambulatory children and adolescents with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene, according to Sarepta Therapeutics, Inc., the pioneer in precision genetic therapy for rare diseases. Based on the levels of ELEVIDYS micro-dystrophin seen in ELEVIDYS-treated patients, this indication was given accelerated approval.

Competitive Landscape

The major global players in the market include: Jubilant Cadista Pharmaceuticals Inc., Sarepta Therapeutics, Inc., ML Bio Solutions, Genethon, Asklepios BioPharmaceutical, Inc., Atamyo Therapeutics, Vita Therapeutics, Inc., Edgewise Therapeutics, BridgeBio Inc. and Hansa Biopharma.

Why Purchase the Report?

• To visualize the global limb girdle muscular dystrophy treatment market segmentation based on type, treatment, end user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of limb girdle muscular dystrophy treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global limb girdle muscular dystrophy treatment market report would provide approximately 61 tables, 57 figures and 186 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

Table of Contents

1. Methodology and Scope

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

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Type
• 3.2. Snippet by Treatment
• 3.3. Snippet by End User
• 3.4. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Growing collaborations and regulatory approvals
    • 4.1.1.2. Rising technological advancements and developments
  • 4.1.2. Restraints
    • 4.1.2.1. Side effects associated with the therapy
  • 4.1.3. Opportunity
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Force Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Regulatory Analysis
• 5.5. SWOT Analysis
• 5.6. Patent Analysis
• 5.7. Russia-Ukraine War Impact Analysis
• 5.8. DMI Opinion

6. COVID-19 Analysis

• 6.1. Analysis of COVID-19
  • 6.1.1. Scenario Before COVID
  • 6.1.2. Scenario During COVID
  • 6.1.3. Scenario Post COVID
• 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. By Type

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 7.1.2. Market Attractiveness Index, By Type
• 7.2. Limb-girdle Muscular Dystrophy Type 1*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Limb-girdle Muscular Dystrophy Type 2

8. By Treatment

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 8.1.2. Market Attractiveness Index, By Treatment
• 8.2. Medications *
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.2.3. Corticosteroids
    • 8.2.3.1. Prednisone
    • 8.2.3.2. Deflazacort (Emflaza)
  • 8.2.4. Small Molecule
    • 8.2.4.1. Ribitol (BBP-418)
• 8.3. Gene Therapy
• 8.4. Others

9. 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 and Y-o-Y Growth Analysis (%)
• 9.3. Ambulatory Surgical Centers
• 9.4. Others

10. By Region

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), 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 Treatment
  • 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 Treatment
  • 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. UK
    • 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 Treatment
  • 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 Treatment
  • 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 Treatment
  • 10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User

11. Competitive Landscape

• 11.1. Competitive Scenario
• 11.2. Market Positioning/Share Analysis
• 11.3. Mergers and Acquisitions Analysis

12. Company Profiles

• 12.1. Jubilant Cadista Pharmaceuticals Inc.*
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. Sarepta Therapeutics, Inc.
• 12.3. ML Bio Solutions
• 12.4. Genethon
• 12.5. Asklepios BioPharmaceutical, Inc.
• 12.6. Atamyo Therapeutics
• 12.7. Vita Therapeutics, Inc.
• 12.8. Edgewise Therapeutics
• 12.9. BridgeBio Inc.
• 12.10. Hansa Biopharma

LIST NOT EXHAUSTIVE

13. Appendix

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