デュシェンヌ型筋ジストロフィー（DMD）治療の世界市場規模：治療タイプ別、治療環境別、地域別、範囲および予測

デュシェンヌ型筋ジストロフィー（DMD）治療市場規模と予測

デュシェンヌ型筋ジストロフィー（DMD）治療市場規模は、2024年に7億2,279万米ドルと評価され、2031年には19億524万米ドルに達すると予測され、2024年から2031年までのCAGRは12.88%で成長すると予測されます。デュシェンヌ型筋ジストロフィー（DMD）治療は、症状の管理、疾患の進行抑制、生活の質の向上を主な目的としています。プレドニゾンやデフラザコートのような副腎皮質ステロイドは、副作用の可能性はあるもの、筋力や機能の維持を助けるために一般的に処方されます。理学療法と個人に合わせた運動プログラムは、可動性の維持と拘縮の予防に重要な役割を果たします。

コルチコステロイドに加え、イデベノンやエテプリルセンなどの薬剤がDMDに関連する特定の課題に対処できる可能性があります。遺伝子治療やエクソンスキッピング薬などの有望な新しい治療法は、DMDの遺伝的基盤を標的とし、長期的な改善につながる可能性があります。

CRISPR-Cas9のような遺伝子編集技術は、DMDの原因となる遺伝子異常を修正する可能性を秘めています。一方、エクソンスキッピング療法は、RNAスプライシングを修正することによってジストロフィンの産生を回復させ、筋肉の変性を遅らせることを目的としています。

さらに、幹細胞治療と筋肉再生技術を含む再生医療アプローチは、損傷した筋肉組織を治癒し、置き換える可能性を提供します。

DMD治療の将来は有望であり、患者の予後を改善し、生活の質を向上させることを目指し、これらの革新的な治療に焦点を当てた研究や臨床研究が進行中です。

デュシェンヌ型筋ジストロフィー（DMD）治療の世界市場力学

世界のデュシェンヌ型筋ジストロフィー（DMD）治療市場を形成している主な市場力学は以下の通りである：

主な市場促進要因

DMDの有病率と診断の増加：

DMD患者数の増加と診断技術の向上が治療需要を牽引しています。筋ジストロフィー協会によると、DMDは世界の男性出生数3,500～5,000人に1人の割合で発症しています。2014年にNeuroepidemiology誌に発表された研究では、米国におけるDMDの有病率は5～24歳の男性1万人あたり1.38人と推定されています。

遺伝子治療と個別化医療の進歩：

遺伝子治療と個別化治療における画期的な進歩により、DMD治療に新たな道が開かれつつあります。米国食品医薬品局（FDA）は近年、DMDに対するいくつかの遺伝子標的療法を承認しています。例えば2016年、FDAはDMD患者の治療薬として初めて承認されたExondys 51（エテプリルセン）を承認しました。それ以来、さらに2つのエクソンスキッピング療法が承認されている：2019年にはVyondys 53（golodirsen）、2020年にはViltepso（viltolarsen）が承認されています。

研究資金と臨床試験の増加：

研究開発への多額の投資と、進行中の多数の臨床試験。米国国立衛生研究所（NIH）のRePORTERデータベースによると、デュシェンヌ型筋ジストロフィー研究に対するNIHの助成金は、2015年の約3,300万米ドルから2020年には8,000万米ドル以上に増加します。さらに、2021年現在、ClinicalTrials.govには、DMDに関連する200以上の研究が掲載されており、この分野での研究への取り組みが重要であることを示しています。

主な課題

高額な治療費と限られたアクセシビリティ：

DMD治療、特に遺伝子治療には高額な費用がかかるため、患者へのアクセシビリティが低下し、市場の成長が制限される可能性があります。2021年にNeurology誌に発表された研究によると、米国におけるDMD患者の年間治療費は、病期にもよるが5万～10万米ドルです。新しい遺伝子治療の場合、そのコストはさらに高くなる可能性があります。例えば、脊髄性筋萎縮症（もう一つの神経筋疾患）の遺伝子治療薬であるゾルゲンスマは、患者一人当たり100万米ドルであり、世界で最も高価な薬剤の一つです。

厳しい規制要件と長い承認プロセス：

DMD治療、特に遺伝子治療は複雑であるため、臨床試験や規制プロセスに時間とコストがかかります。タフツ医薬品開発研究センターの2018年の報告書によると、DMD治療薬を含む希少疾患治療薬の臨床試験開始から薬事承認までの平均期間は8.1年です。

限られた患者数と臨床試験募集の課題：

DMDは希少疾患であるため、臨床試験に十分な患者をリクルートすることが難しく、研究開発が遅れる可能性があります。Orphanet Journal of Rare Diseasesによると、DMDは全世界の男性出生数の約3,500～5,000人に1人が罹患しています。これは、推定患者数が世界で約28万4,000人であることを意味します。

主要動向

遺伝子治療と遺伝子編集アプローチへの注目の高まり：

遺伝子治療はDMDの有望な治療選択肢として浮上しており、複数の候補が臨床試験段階にあります。Gene Therapy Clinical Trials Worldwideデータベースによると、2021年現在、DMDを対象とした遺伝子治療の臨床試験が20件以上進行中または計画中です。米国食品医薬品局（FDA）は、DMDに対する複数の遺伝子治療候補にファスト・トラックおよび希少小児疾患の指定を与えており、このアプローチの重要性が高まっていることを強調しています。

併用療法とマルチモーダル治療アプローチの増加：

DMD病態の複数の側面を同時に標的とする併用療法の研究がますます進んでいます。2020年にJournal of Neuromuscular Diseases誌に発表された研究では、DMDを対象とした53の臨床試験をレビューしています。そのうち13％が併用療法を研究しており、マルチモーダル治療への傾向が強まっていることを示しています。

バイオマーカー開発と個別化医療の進歩：

早期診断、予後改善、個別化治療戦略を可能にするため、DMDのバイオマーカー開発の傾向が高まっています。米国国立衛生研究所（NIH）は、DMDのバイオマーカー研究に多大な投資を行っています。NIH RePORTERデータベースによると、DMDバイオマーカー研究への資金は2015年の約500万米ドルから2020年には1,500万米ドル以上に増加します。

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目次

第1章 デュシェンヌ型筋ジストロフィー（DMD）治療の世界市場：イントロダクション

• 市場概要
• 調査範囲
• 前提条件

第2章 エグゼクティブサマリー

第3章 VERIFIED MARKET RESEARCHの調査手法

• データマイニング
• バリデーション
• 一次資料
• データソース一覧

第4章 世界のデュシェンヌ型筋ジストロフィー（DMD）治療市場の展望

• 概要
• 市場力学
  • 促進要因
  • 抑制要因
  • 機会
• ポーターのファイブフォースモデル
• バリューチェーン分析

第5章 デュシェンヌ型筋ジストロフィー（DMD）治療の世界市場：治療タイプ別

• 概要
• ステロイド療法
• エクソンスキッピング療法
• 遺伝子治療
• 分子ベース療法

第6章 デュシェンヌ型筋ジストロフィー（DMD）治療の世界市場：治療環境別

• 概要
• 病院
• 専門クリニック
• 在宅医療

第7章 デュシェンヌ型筋ジストロフィー（DMD）治療の世界市場：地域別

• 概要
• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • その他欧州
• アジア太平洋
  • 中国
  • 日本
  • インド
  • その他アジア太平洋地域
• 世界のその他の地域
  • ラテンアメリカ
  • 中東アフリカ

第8章 世界のデュシェンヌ型筋ジストロフィー（DMD）治療市場：競合情勢

• 概要
• 各社の市場ランキング
• 主な発展戦略

第9章 企業プロファイル

• Sarepta Therapeutics, Inc
• Santhera Pharmaceuticals
• PTC Therapeutics
• FibroGen, Inc
• ITALFARMACO S.p.A.
• Nobelpharma Co. Ltd
• NS Pharma Inc.
• Pfizer Inc.

第10章 主な発展

• 製品上市／開発
• 合併と買収
• 事業拡大
• パートナーシップと提携

第11章 付録

• 関連調査

Duchenne Muscular Dystrophy (DMD) Treatment Market Size And Forecast

The Duchenne Muscular Dystrophy (DMD) Treatment Market size was valued at USD 722.79 Million in 2024 and is projected to reach USD 1905.24 Million by 2031 , growing at a CAGR of 12.88% from 2024 to 2031. Duchenne muscular dystrophy (DMD) treatment primarily aims to manage symptoms, slow disease progression, and enhance the quality of life, as there is currently no cure for this condition. Corticosteroids like prednisone and deflazacort are commonly prescribed to help maintain muscle strength and function, despite potential side effects. Physical therapy and tailored exercise programs play a crucial role in preserving mobility and preventing contractures.

In addition to corticosteroids, medications such as idebenone and eteplirsen may address specific challenges associated with DMD. Promising emerging therapies, including gene therapy and exon-skipping medications, target the genetic basis of DMD and could potentially lead to long-term improvements.

Gene editing techniques, such as CRISPR-Cas9, hold the potential to correct the genetic abnormalities responsible for DMD while exon-skipping therapies aim to restore dystrophin production by modifying RNA splicing, thereby slowing muscle degeneration.

Furthermore, regenerative medicine approaches that involve stem cell therapies and muscle regeneration techniques offer the possibility of healing and replacing damaged muscle tissue.

The future of DMD treatment looks promising, with ongoing research and clinical studies focused on these innovative therapies, aiming to enhance patient outcomes and improve their quality of life.

Global Duchenne Muscular Dystrophy (DMD) Treatment Market Dynamics

The key market dynamics that are shaping the global duchenne muscular dystrophy (DMD) treatment market include:

Key Market Drivers

Increasing Prevalence and Diagnosis Of DMD:

The rising number of DMD cases, coupled with improved diagnostic techniques, is driving the demand for treatments. According to the Muscular Dystrophy Association, DMD affects approximately 1 in every 3,500 to 5,000 male births worldwide. A study published in the journal Neuroepidemiology in 2014 estimated the prevalence of DMD in the United States to be 1.38 per 10,000 males aged 5-24 years.

Advancements in Gene Therapy and Personalized Medicine:

Breakthroughs in gene therapy and personalized treatment approaches are opening new avenues for DMD treatment. The U.S. Food and Drug Administration (FDA) has approved several gene-targeted therapies for DMD in recent years. For instance, in 2016, the FDA approved Exondys 51 (eteplirsen), the first drug approved to treat patients with DMD. Since then, two more exon-skipping therapies have been approved: Vyondys 53 (golodirsen) in 2019 and Viltepso (viltolarsen) in 2020.

Increased Research Funding and Clinical Trials:

Substantial investments in research and development, along with numerous ongoing clinical trials. According to the National Institutes of Health (NIH) RePORTER database, NIH funding for Duchenne Muscular Dystrophy research increased from approximately $33 million in 2015 to over $80 million in 2020. Additionally, as of 2021, ClinicalTrials.gov listed over 200 studies related to DMD, indicating significant ongoing research efforts in this field.

Key Challenges

High Cost of Treatment and Limited Accessibility:

The expensive nature of DMD treatments, particularly gene therapies, can limit market growth by reducing accessibility for patients. According to a study published in the journal Neurology in 2021, the annual cost of care for patients with DMD in the United States ranges from $50,000 to $100,000, depending on the stage of the disease. For newer gene therapies, the costs can be even higher. For instance, Zolgensma, a gene therapy for spinal muscular atrophy (another neuromuscular disorder), is priced at USD1 Million per patient, making it one of the world's most expensive drugs.

Stringent Regulatory Requirements and Long Approval Processes:

The complex nature of DMD treatments, especially gene therapies, leads to lengthy and costly clinical trials and regulatory processes. A report by the Tufts Center for the Study of Drug Development in 2018 found that the average time from the start of clinical testing to regulatory approval for rare disease drugs, including those for DMD, is 8.1 years.

Limited Patient Population and Challenges in Clinical Trial Recruitment:

The rarity of DMD makes it difficult to recruit sufficient patients for clinical trials, potentially slowing down research and development efforts. According to the Orphanet Journal of Rare Diseases, DMD affects approximately 1 in 3,500 to 5,000 male births worldwide. This translates to an estimated patient population of about 284,000 individuals globally.

Key Trends

Increasing Focus on Gene Therapy and Gene Editing Approaches:

Gene therapy is emerging as a promising treatment option for DMD, with several candidates in clinical trials. According to the Gene Therapy Clinical Trials Worldwide database, as of 2021, there were over 20 ongoing or planned gene therapy clinical trials for DMD. The U.S. Food and Drug Administration (FDA) has granted Fast Track and Rare Pediatric Disease designations to multiple gene therapy candidates for DMD, highlighting the growing importance of this approach.

Rise In Combination Therapies and Multi-Modal Treatment Approaches:

Researchers are increasingly exploring combination therapies that target multiple aspects of DMD pathology simultaneously. A study published in the Journal of Neuromuscular Diseases in 2020 reviewed 53 clinical trials for DMD. Of these, 13% were investigating combination therapies, indicating a growing trend towards multi-modal treatment approaches.

Advancement in Biomarker Development and Personalized Medicine:

There's a growing trend towards developing biomarkers for DMD to enable earlier diagnosis, better prognosis, and personalized treatment strategies. The National Institutes of Health (NIH) has invested significantly in biomarker research for DMD. According to the NIH RePORTER database, funding for DMD biomarker research increased from approximately USD 5 Million in 2015 to over $15 million in 2020.

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Global Duchenne Muscular Dystrophy (DMD) Treatment Market Regional Analysis

Here is a more detailed regional analysis of the global duchenne muscular dystrophy (DMD) treatment market:

North America

North America substantially dominates the global Duchenne Muscular Dystrophy treatment market owing to the high prevalence of DMD.

The relatively high incidence rate of DMD in North America contributes to the region's market dominance. According to the Centers for Disease Control and Prevention (CDC), DMD affects approximately 1 in every 3,500 to 5,000 male births in the United States.

Substantial funding for DMD research has accelerated the development of new treatments. The National Institutes of Health (NIH) reported awarding over $76 million in research grants for muscular dystrophy in fiscal year 2020, with a significant portion dedicated to DMD research.

The U.S. Food and Drug Administration (FDA) has implemented policies to expedite the approval process for DMD treatments. As of 2023, the FDA has approved four exon-skipping therapies for DMD, demonstrating the impact of these regulatory initiatives on bringing new treatments to market.

In North America, healthcare reimbursement policies and insurance coverage significantly enhance the accessibility of Duchenne muscular dystrophy (DMD) treatments for eligible patients.

Government-funded healthcare programs, private insurance plans, and patient assistance initiatives collectively help alleviate the financial burden associated with DMD treatment costs for patients and their families.

This favorable reimbursement landscape incentivizes pharmaceutical companies to invest in the research, development, and commercialization of DMD therapies, facilitating broader access to life-changing treatments.

Asia Pacific

Asia Pacific is anticipated to witness the fastest growth in the global Duchenne Muscular Dystrophy treatment market during the forecast period owing to the large patient population and increasing diagnosis rates.

The Asia Pacific region has a significant DMD patient population due to its large overall population. In India alone, it's estimated that there are about 5 out of every 100,000 male births affected by DMD, according to a study published in the Annals of the Indian Academy of Neurology.

Improved diagnostic capabilities in countries like China and Japan have led to earlier and more accurate DMD diagnoses.

Economic growth in many Asia Pacific countries has led to increased healthcare spending. For instance, China's healthcare expenditure grew from 5.03% of GDP in 2010 to 6.57% in 2020, according to World Bank data. This growth has supported the development of specialized care facilities for rare diseases like DMD.

Expanding clinical research and treatment options There's been a significant increase in DMD-related clinical trials in the Asia Pacific region. According to the U.S. National Library of Medicine's ClinicalTrials.gov database, as of 2023, there were over 50 ongoing or completed clinical trials for DMD treatments in countries like China, Japan, and South Korea combined. This surge in research activity is driving innovation and expanding treatment options for DMD patients in the region.

Global Duchenne Muscular Dystrophy (DMD) Treatment Market: Segmentation Analysis

The Global Duchenne Muscular Dystrophy (DMD) Treatment Market is segmented based on Treatment Type, Treatment Setting, And Geography.

Duchenne Muscular Dystrophy (DMD) Treatment Market, By Treatment Type

• Steroid Therapy0
• Exon Skipping Therapy
• Gene Therapy
• Molecular-based Therapy

Based on the Treatment Type, the Global Duchenne Muscular Dystrophy Treatment Market is bifurcated into Steroid Therapy, Exon Skipping Therapy, Gene Therapy, and Molecular-based Therapy. Steroid therapy leads the Global Duchenne Muscular Dystrophy (DMD) Treatment Market, primarily due to its widespread use and established effectiveness in slowing disease progression. Corticosteroids, such as prednisone and deflazacort, are recognized as the standard of care for DMD, helping to preserve muscle function and delay the progression of the disease.

Duchenne Muscular Dystrophy (DMD) Treatment Market, By Treatment Setting

• Hospitals
• Specialty Clinics
• Homecare

Based on the Treatment Setting, the Global Duchenne Muscular Dystrophy (DMD) Treatment Market is bifurcated into Hospitals, Specialty Clinics, and Homecare. The Hospital segment dominates the Global Duchenne Muscular Dystrophy (DMD) Treatment Market owing to its ability to provide comprehensive care that encompasses diagnosis, treatment, rehabilitation, and support services. Hospitals typically have access to advanced therapies, including gene therapy and stem cell therapy, which are essential for managing DMD effectively.

Duchenne Muscular Dystrophy (DMD) Treatment Market, By Geography

• North America
• Europe
• Asia Pacific
• Rest of the World

Based on Geography, the Global Duchenne Muscular Dystrophy (DMD) Treatment Market is classified into North America, Europe, Asia Pacific, and the Rest of the World. North America substantially dominates the global Duchenne Muscular Dystrophy (DMD) treatment market owing to the high prevalence of DMD. The relatively high incidence rate of DMD in North America contributes to the region's market dominance. According to the Centers for Disease Control and Prevention (CDC), DMD affects approximately 1 in every 3,500 to 5,000 male births in the United States. Substantial funding for DMD research has accelerated the development of new treatments. The National Institutes of Health (NIH) reported awarding over $76 million in research grants for muscular dystrophy in fiscal year 2020, with a significant portion dedicated to DMD research.

Key Players

• The "Global Duchenne Muscular Dystrophy (DMD) Treatment Market" study report will provide valuable insight with an emphasis on the global market. The major players in the market are
• Sarepta Therapeutics, Inc., Santhera Pharmaceuticals, PTC Therapeutics, FibroGen, Inc., ITALFARMACO S.p.A., Nobelpharma Co. Ltd, NS Pharma, Inc., Pfizer Inc.
• This section offers in-depth analysis through a company overview, position analysis, the regional and industrial footprint of the company, and the ACE matrix for insightful competitive analysis. The section also provides an exhaustive analysis of the financial performances of mentioned players in the given market.

Our market analysis also entails a section solely dedicated to such major players wherein our analysts provide an insight into the financial statements of all the major players, along with product benchmarking and SWOT analysis. The competitive landscape section also includes key development strategies, market share, and market ranking analysis of the above-mentioned players globally.

Global Duchenne Muscular Dystrophy (DMD) Treatment Market Key Developments

• In June 2023, Sarepta Therapeutics received accelerated FDA approval for ELEVIDYS(TM) (delandistrogene moxeparvovec-rokl), the first gene therapy for DMD. This ground-breaking medication has been approved for ambulatory patients aged 4-5 years who have a verified DMD mutation.
• In September 2023, PTC Therapeutics reported positive findings from their Phase 3 MOVE study of vatiquinone for mitochondrial dysfunction and DMD-related fatigue. The company intends to submit a New Drug Application to the FDA within the first half of 2024.
• In December 2023, Solid Biosciences announced the collaboration with AavantiBio to build a new firm focused on developing genetic therapies for rare diseases such as DMD. The United company wants to speed up the development of gene treatments for muscular dystrophy.
• In February 2024, Pfizer discontinued its Phase 3 research of fordadistrogene movaparvovec, a miniature dystrophin gene DMD therapy is not recommended due to safety concerns. These rulings change the competitive landscape for gene treatments in DMD treatment.
• In April 2024, Edgewise Therapeutics begins a Phase 2 trial for EDG-5506, a revolutionary muscle-preserving DMD treatment. The medicine is intended to protect skeletal and heart muscle in DMD patients, potentially reducing disease development.

TABLE OF CONTENTS

1 INTRODUCTION OF GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET

• 1.1 Overview of the Market
• 1.2 Scope of Report
• 1.3 Assumptions

2 EXECUTIVE SUMMARY

3 RESEARCH METHODOLOGY OF VERIFIED MARKET RESEARCH

• 3.1 Data Mining
• 3.2 Validation
• 3.3 Primary Interviews
• 3.4 List of Data Sources

4 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET OUTLOOK

• 4.1 Overview
• 4.2 Market Dynamics
  • 4.2.1 Drivers
  • 4.2.2 Restraints
  • 4.2.3 Opportunities
• 4.3 Porters Five Force Model
• 4.4 Value Chain Analysis

5 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY TREATMENT TYPE

• 5.1 Overview
• 5.2 Steroid Therapy
• 5.3 Exon Skipping Therapy
• 5.4 Gene Therapy
• 5.5 Molecular-based Therapy

6 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY TREATMENT SETTING

• 6.1 Overview
• 6.2 Hospitals
• 6.3 Specialty Clinics
• 6.4 Homecare

7 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY GEOGRAPHY

• 7.1 Overview
• 7.2 North America
  • 7.2.1 U.S.
  • 7.2.2 Canada
  • 7.2.3 Mexico
• 7.3 Europe
  • 7.3.1 Germany
  • 7.3.2 U.K.
  • 7.3.3 France
  • 7.3.4 Rest of Europe
• 7.4 Asia Pacific
  • 7.4.1 China
  • 7.4.2 Japan
  • 7.4.3 India
  • 7.4.4 Rest of Asia Pacific
• 7.5 Rest of the World
  • 7.5.1 Latin America
  • 7.5.2 Middle East And Africa

8 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET COMPETITIVE LANDSCAPE

• 8.1 Overview
• 8.2 Company Market Ranking
• 8.3 Key Development Strategies

9 COMPANY PROFILES

• 9.1 Sarepta Therapeutics, Inc
  • 9.1.1 Overview
  • 9.1.2 Financial Performance
  • 9.1.3 Product Outlook
  • 9.1.4 Key Developments
• 9.2 Santhera Pharmaceuticals
  • 9.2.1 Overview
  • 9.2.2 Financial Performance
  • 9.2.3 Product Outlook
  • 9.2.4 Key Developments
• 9.3 PTC Therapeutics
  • 9.3.1 Overview
  • 9.3.2 Financial Performance
  • 9.3.3 Product Outlook
  • 9.3.4 Key Developments
• 9.4 FibroGen, Inc
  • 9.4.1 Overview
  • 9.4.2 Financial Performance
  • 9.4.3 Product Outlook
  • 9.4.4 Key Developments
• 9.5 ITALFARMACO S.p.A.
  • 9.5.1 Overview
  • 9.5.2 Financial Performance
  • 9.5.3 Product Outlook
  • 9.5.4 Key Developments
• 9.6 Nobelpharma Co. Ltd
  • 9.6.1 Overview
  • 9.6.2 Financial Performance
  • 9.6.3 Product Outlook
  • 9.6.4 Key Developments
• 9.7 NS Pharma Inc.
  • 9.7.1 Overview
  • 9.7.2 Financial Performance
  • 9.7.3 Product Outlook
  • 9.7.4 Key Developments
• 9.8 Pfizer Inc.
  • 9.8.1 Overview
  • 9.8.2 Financial Performance
  • 9.8.3 Product Outlook
  • 9.8.4 Key Developments

10 KEY DEVELOPMENTS

• 10.1 Product Launches/Developments
• 10.2 Mergers and Acquisitions
• 10.3 Business Expansions
• 10.4 Partnerships and Collaborations

11 Appendix

• 11.1 Related Research