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表紙：アルファマンノシドーシス治療の世界市場：2023年～2030年
市場調査レポート
商品コード
1382520

アルファマンノシドーシス治療の世界市場：2023年～2030年

Global Alpha-mannosidosis Treatment Market - 2023-2030
出版日: | 発行: DataM Intelligence | ページ情報: 英文 187 Pages | 納期: 約2営業日

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価格
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本日の銀行送金レート: 1USD=157.14円
アルファマンノシドーシス治療の世界市場：2023年～2030年
出版日: 2023年11月17日
発行: DataM Intelligence
ページ情報: 英文 187 Pages
納期: 約2営業日
ご注意事項 :
本レポートは最新情報反映のため適宜更新し、内容構成変更を行う場合があります。ご検討の際はお問い合わせください。
  • 全表示
  • 概要
  • 目次
概要

概要

アルファマンノシドーシス治療は、体内の細胞内で特定の複合糖質を適切に分解できないことを特徴とする稀な遺伝子疾患です。中枢神経系を含む多くの身体器官やシステムが、糖の蓄積によって影響を受ける。この病気の影響は人によって大きく異なります。アルファマンノシドーシス治療は、アルファマンノシダーゼ酵素の生成をコードするMAN2B1遺伝子の変化によって発症します。

アルファマンノシドーシス治療の重症度は様々です。発達の遅れ、知的課題、言語障害、難聴、骨の変形などが典型的な徴候です。アルファマンノシドーシス治療では、アルファマンノシダーゼ酵素を産生するMAN2B1遺伝子が変異しています。現在、アルファマンノシドーシス治療の治療法はなく、症状管理と支持療法が中心です。

市場力学：促進要因と阻害要因

政府機関による承認の増加

欧州医薬品庁（EMA）や米国食品医薬品局（FDA）などの規制機関は、希少疾患の治療法開発を促進するための取り組みやインセンティブを提供しています。これらのイニシアチブは、市場の拡大を支援すると同時に、業界における綿密な研究開発を促進します。

例えば、Chiesi Farmaceutici S.p.A.の一部門であるChiesi Global Rare Diseasesによると、米国食品医薬品局（FDA）は2023年2月、成人および小児のアルファマンノシドーシス治療（AM）治療薬としてLamzede（velmanase alfa-tycv）を承認しました。マンノシダーゼという酵素の欠乏により、AMは極めてまれな進行性のリソソーム貯蔵障害となります。

遺伝学や分子生物学を含む医学の発展により、この疾患のメカニズムは解明されつつあります。このような情報から、標的を絞った医薬品が開発されるかもしれないです。製薬会社、バイオテクノロジー企業、研究機関によって、希少疾患の治療法の開発に向けて投資が行われています。革新的な治療法を市場に投入するために、資金と投資は調査方法を加速させ、臨床試験を迅速に行うことができます。

市場力学：市場抑制要因

アルファマンノシドーシス治療の管理・治療を抑制する要因はいくつかあります。例えば、有望な治療法が開発されたとしても、特にヘルスケア資源が限られている地域や経済的に恵まれない地域においては、その治療法へのアクセスを広く確保することが困難な場合があり、希少疾患研究への資金援助が限られていることが、この治療に関わる妨げとなる可能性があります。アルファマンノシドーシス治療の診断は、その希少性と症状のばらつきのために困難であり、誤診されたり診断されなかったりして、適切な医療管理の遅れにつながる可能性があります。市場規模が小さいため、製薬会社は希少疾患の治療法への投資意欲が低いかもしれないです。

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
      • 政府機関による認可の増加
  • 抑制要因
      • 希少疾患研究への資金提供の制限
    • 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
  • パイプライン分析
  • アンメットニーズ
  • DMI意見

第6章 COVID-19分析

第7章 適応症別

  • I型
  • II型
  • III型

第8章 治療法別

  • 骨髄移植（BMT）
  • 酵素補充療法（ERT）
  • 遺伝子治療
  • その他

第9章 エンドユーザー別

  • 病院
  • 専門クリニック
  • 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
  • アジア太平洋地域
    • 中国
    • インド
    • 日本
    • オーストラリア
    • その他アジア太平洋地域
  • 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

  • CHIESI Farmaceutici S.p.A.
    • 会社概要
    • 製品ポートフォリオと説明
    • 財務概要
    • 主な発展
  • Zymenex

第13章 付録

目次
Product Code: PH7430

Overview

Alpha-mannosidosis is a rare genetic condition characterized by an inability to properly break down certain groups of complex sugars in the body's cells. Numerous bodily organs and systems, including the central nervous system, are impacted by sugar buildup. The disease's effects might vary greatly from person to person. Alpha-mannosidosis is brought on by changes in the MAN2B1 gene, which codes for the creation of the alpha-mannosidase enzyme.

Individuals with alpha-mannosidosis may have varying degrees of severity. Developmental delays, intellectual challenges, speech problems, hearing loss, and bone deformities are typical signs. The MAN2B1 gene, which gives instructions for producing the alpha-mannosidase enzyme, is mutated in alpha-mannosidosis. There is currently no treatment for alpha-mannosidosis; instead, the focus is on symptom management and supportive care.

Market Dynamics: Drivers and Restraints

The rise of approvals by government agencies

Regulatory organisations, such the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA), provide initiatives and incentives to promote the development of therapies for rare diseases. These initiatives promote in-depth research and development in the industry while helping market expansion.

For instance, in February 2023, U.S. Food and Drug Administration (FDA) has approved Lamzede (velmanase alfa-tycv) for the treatment of alpha-mannosidosis (AM) in adults and children, according to Chiesi Global Rare Diseases, a division of Chiesi Farmaceutici S.p.A. A lack of the enzyme -mannosidase results in AM, an extremely rare, progressive lysosomal storage disorder.

The mechanisms of the disease have become well-defined because to ongoing developments in medical science, including genetics and molecular biology. The development of targeted medicines may result from such information. Investments are made towards the creation of treatments for rare medical conditions by pharmaceutical companies, biotech companies, and research organisations. In order to bring innovative therapies to market, funding and investment can speed up the research process and make clinical trials quicker.

Market Dynamics: Restraint

There are several factors that can restrain the management and treatment of alpha-mannosidosis. For instance, even if promising treatments are developed, it may be difficult to ensure widespread access to them, especially in areas with limited healthcare resources or in economically disadvantaged communities, and limited funding for rare diseases research can hinder this are involved. Diagnosing alpha-mannosidosis can be challenging due to its rarity and the variability in symptoms, and it may be misdiagnosed or undiagnosed, leading to delays in appropriate medical management. Due to the small market size, pharmaceutical companies may be less motivated to invest in treatments for rare diseases.

Segment Analysis

The global alpha-mannosidosis marketis segmented based on indication, treatment, end user and region.

The Enzyme Replacement Therapy (ERT) segment from the type segment accounted for approximately 39.07% of Alpha-mannosidosis share

Certain lysosomal storage disorders, such as Alpha-mannosidosis, are treated medically with enzyme replacement therapy (ERT). A lack of the enzyme alpha-mannosidase results in the rare hereditary condition known as alpha-mannosidosis. The patient is subsequently given the purified and altered enzyme via routine intravenous infusions, frequently in a clinical environment like a hospital or specialised treatment facility. Complex carbohydrates must be broken down by this enzyme in the lysosomes of cells.

It's crucial to stay in mind that ERT is frequently a lifetime treatment. Regular infusions are required to keep the body's enzyme levels stable and to keep the breakdown of stored materials going. Healthcare workers regularly monitor patients receiving ERT. Depending on the patient's response and specific requirements, the dosage and frequency of the infusions may need to be changed.

Geographical Analysis

North America accounted for approximately 34.04% of the market share in 2022

The largest market share for alpha mannosidosis was held by North America, whose dominance was primarily explained by the greater volume of clinical trials carried out there. Additionally, the region has witnessed a rise in the development of orphan medications. Many orphan drugs are receiving FDA and other regulatory body approvals as a result of such government policies.

Additionally, among the regions, the mannosidosis market in Europe is predicted to grow at the quickest rate. The numerous government attempts to raise public knowledge of rare genetic illnesses and associated therapies have been responsible for the market expansion in Europe.

COVID-19 Impact Analysis

Over only a short time, the spread of new coronavirus had a rather detrimental effect on the global alpha mannosidosis market. During the pandemic era, the healthcare, pharmaceutical, and other associated industries experienced intense pressure. The number of COVID-19 patients increased to the point where hospitals and clinics were overrun. These facilities' resources were almost entirely devoted to combating the pandemic. This led to the neglect of individuals with other, less serious medical illnesses, such as genetic diseases like alpha mannosidosis.

In addition, a number of governmental and private healthcare organisations prioritised funding the pandemic crisis. This resulted in less money being available for illnesses other than COVID-19. Healthcare service providers are anticipated to concentrate on non-Covid patients with other health conditions as the number of COVID-19 cases declines. In turn, it is projected that this will accelerate market expansion during the post-pandemic period.

Market Segmentation

By Indication

  • Type I
  • Type II
  • Type III

By Treatment

  • Bone Marrow Transplant (BMT)
  • Enzyme Replacement Therapy (ERT)
  • Gene Therapy
  • Others

By End User

  • Hospitals
  • Specialty Clinics
  • Others

By Region

  • North America
    • U.S.
    • Canada
    • Mexico
  • Europe
    • Germany
    • UK
    • France
    • Italy
    • Spain
    • Rest of Europe
  • South America
    • Brazil
    • Argentina
    • Rest of South America
  • Asia-Pacific
    • China
    • India
    • Japan
    • Australia
    • Rest of Asia-Pacific
  • Middle East and Africa

Competitive Landscape

major global players in the market include: CHIESI Farmaceutici S.p.A. and Zymenex among others.

Why Purchase the Report?

  • To visualize the global alpha-mannosidosis market segmentation based on indication, treatment, end user and region as well as understandkey commercial assets and players.
  • Identify commercial opportunities by analyzing trends and co-development.
  • Excel data sheet with numerous data points of alpha-mannosidosis 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 Alpha-mannosidosis market report would provide approximately 39 tables, 50 figures and 187 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 Indication
  • 3.2. Snippet by Treatment
  • 3.3. Snippet by End User
  • 3.4. Snippet by Region

4. Market Dynamics

  • 4.1. Impacting Factors
    • 4.1.1. Drivers
      • 4.1.1.1. The rise of approvals by government agencies
  • 4.2. Restraints
      • 4.2.1.1. Limited funding for rare diseases research
    • 4.2.2. Opportunity
    • 4.2.3. 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. Pipeline Analysis
  • 5.6. Unmet Needs
  • 5.7. 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 Indication

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
    • 7.1.2. Market Attractiveness Index, By Indication
  • 7.2. Type I *
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 7.3. Type II
  • 7.4. Type III

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. Bone Marrow Transplant (BMT) *
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.3. Enzyme Replacement Therapy (ERT)
  • 8.4. Gene Therapy
  • 8.5. 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. Specialty Clinics
  • 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 Indication
    • 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 Indication
    • 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 Indication
    • 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 Indication
    • 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 Indication
    • 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 Group

11. Competitive Landscape

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

12. Company Profiles

  • 12.1. CHIESI Farmaceutici S.p.A. *
    • 12.1.1. Company Overview
    • 12.1.2. Product Portfolio and Description
    • 12.1.3. Financial Overview
    • 12.1.4. Key Developments
  • 12.2. Zymenex

LIST NOT EXHAUSTIVE

13. Appendix

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