フコシドーシス治療の世界市場-2023年～2030年

概要

フコシドーシス治療市場動向は、技術的進歩や調査研究の高まりにより、調査研究が活発化していることを示しています。フコシドーシスの治療のためにいくつかの治療法が研究されています。骨髄移植もフコシドーシスの治療に利用されています。

さらに、良好な結果をもたらす治療法の研究開発の高まりによるフコシドーシス治療のパイプラインの増加、市場におけるフコシドーシス治療症例数の増加、研究開発の増加がフコシドーシス治療市場規模を押し上げています。

フコシドーシス治療市場は、この分野の進歩の高まりにより、北米地域からの治療需要が伸びています。Merck KGaA、JCR Pharmaceuticals Co.Ltd.、Orchard Therapeutics plc、Merck KGaAのような重要な競合企業が市場で積極的に活動しているため、市場は今後成長するでしょう。

ダイナミクス

研究者による研究開発の進展がフコシドーシス治療薬市場の成長を促す

現在、フコシドーシス・アリソソーム病の治療法を見つけるために、研究者らによる数多くの研究開発が行われています。フコシダーゼ欠損症のイヌでは、造血幹細胞移植（HSCT）が有望な結果を示しました。

少数のフコシドーシス患者が造血幹細胞移植を受け、いくつかの症例では症状が安定しました。フコシドーシス造血幹細胞移植は当初、無症状の生後7ヵ月の乳児に試みられ、症状のある兄姉がフコシドーシスであることが判明しました。この治療法が有効であることがわかると、慎重に選ばれた患者にのみ、控えめに適用されるようになっています。

フコシドーシスでは、症状が完全に進行してからの移植よりも、早期の移植の方が成功率が高いことが証明されました。移植成功後、白血球、血漿、脳脊髄液中の-Lフコシダーゼ濃度は徐々に上昇し、MRI検査では髄鞘の改善が認められました。このような前向きな結果を伴う研究は、今後数年間の市場の高成長をもたらすと思われます。

活発化するパイプラインと技術進歩が市場成長の好機を生み出す

現在、複数の大手企業がフコシドーシスの治療のための新規療法を開発しており、製品はパイプラインにあります。これらの企業は、パートナーシップ、研究開発、共同研究、強力なパイプラインなどの戦略的イニシアチブを展開し、市場の成長を高めています。

JCRファーマ株式会社JCRは、フコシドーシス患者を対象とした新規治療薬候補である血液脳関門（BBB）透過性-L-フコシダーゼJR-471の開発に、JCRのBBB透過特許技術であるJ-Brain Cargoを活用することを選択したと発表しました。フコシドーシスは、常染色体劣性遺伝するライソゾーム貯蔵障害の一種であり、新たな適応症です。

糖タンパク質代謝酵素（-フコシダーゼ）の変異による機能不全の結果、糖鎖や糖タンパク質が全身に蓄積します。JR-471の静脈内注射は、JCRが疾患モデルマウスを用いて行った予備調査では、脳への送達と脳内の疾患原因成分の減少を示しました。このような要因が市場開拓の機会となっています。

造血幹細胞療法に伴う合併症が市場成長を妨げる

骨髄移植後、合併症は突然起こることもあれば徐々に起こることもあります。幹細胞移植の移植元、年齢、ベースラインのパフォーマンス状態、準備レジメンの種類、強度などはすべて、これらの副作用に影響を与える可能性があります。慢性的な副作用には、水痘・帯状疱疹ウイルス再活性化、カプセル化細菌感染、慢性GVHDなどがあります。

貧血、好中球減少、血小板減少を伴う骨髄抑制、粘膜炎、洞閉塞症候群、急性移植片対宿主病、グラム陽性またはグラム陰性細菌感染、ヘルペスウイルス科感染、アスペルギルスやカンジダによる真菌感染などは、最初の90日以内に起こりうる急性合併症のほんの一部です。

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
    • 研究者による研究開発の成長
    • フコシドーシス治療薬開発のための共同研究の増加
  • 抑制要因
    • 造血幹細胞治療に伴う合併症
  • 機会
    • 活発化するパイプラインと技術進歩
  • 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

第6章 COVID-19分析

第7章 タイプ別

• フコシドーシスI型
• フコシドーシスII型

第8章 治療法別

• 抗生物質療法
• 補液療法
• 造血幹細胞移植療法
• 酵素補充療法

第9章 エンドユーザー別

• 病院
• 外来手術センター
• 専門クリニック

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
  • スペイン
  • イタリア
  • 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

• Merck KGaA
  • 企業概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な動向
• JCR Pharmaceuticals Co., Ltd.
• Orchard Therapeutics plc
• Protalix BioTherapeutics, Inc.
• Magenta Therapeutics
• Evotec SE
• Jasper Therapeutics, Inc.
• Hemogenyx Pharmaceuticals plc
• Garuda Therapeutics
• STEMPEUTICS RESEARCH PVT LTD.

第13章 付録

Overview

The fucosidosis treatment market trends show rising research owing to the rising technological advancements and research studies. Several therapies are being researched for the treatment of fucosidosis. Bone marrow transplant can also be utilized for the management of fucosidosis.

Furthermore, rising pipeline for fucosidosis treatment due to the rising research and development of therapies with positive outcomes and increase in the number of fucosidosis treatment cases in the market, and an increase in the research and development is driving up the fucosidosis treatment market size.

The market is experiencing a growth in demand for treatment from North American areas as a result of the rising advancements in this field. With significant competitors like Merck KGaA, JCR Pharmaceuticals Co., Ltd., Orchard Therapeutics plc, and Merck KGaA actively operating in the market, the market will grow in upcoming time.

Dynamics

Growing Research and Development by the Researchers Drive the Growth of the Fucosidosis Treatment Market

Numerous research and developments are currently taking place by the researchers for finding treatment of fucosidosis alysosomal disease. In canines with fucosidase deficiency, hematopoietic stem cell transplantation (HSCT) exhibited promising results; -L-fucosidase enzyme activity regulated in plasma, leucocytes, and brain and visceral organs.

A small number of individuals with fucosidosis had HSCT, and in some cases, the symptoms stabilized. The procedure was initially tried on an asymptomatic 7-month-old infant who was identified as having fucosidosis following the identification of the symptomatic elder sibling. When it was discovered to be a successful treatment, it was applied sparingly and only on carefully chosen patients.

In fucosidosis, earlier transplant proved to be more successful than transplant following symptoms have fully developed. Following a successful transplant, -L fucosidase levels in white blood cells, plasma, and cerebrospinal fluid gradually increased, and an MRI scan showed improved myelination. Such kind of researches with positive outcomes will result in high growth of the market in coming years.

Rising Robust Pipeline and Technological Advancements Create a Lucrative Opportunities for Growth of the Market

Currently several major players are developing novel therapies for the treatment of fucosidosis, the products are in the pipeline. These companies are developing strategic initiatives such as partnerships, research and development, collaborations or robust pipeline for increasing the growth of the market.

JCR Pharmaceuticals Co., Ltd. Announced its choice to utilize J-Brain Cargo, JCR's patented BBB penetrating technology, to the development of a JR-471, a novel therapeutic candidate, blood-brain barrier (BBB)-penetrating -L-fucosidase for the treatment of patients with fucosidosis. Fucosidosis is a type of lysosomal storage disorder which is inherited in an autosomal recessive way and is the new target indication.

Glycans and glycoproteins build up throughout the body as a result of a glycoprotein-metabolizing enzyme (-fucosidase) malfunctioning due to mutations. The intravenously injected JR-471 showed brain delivery and a decrease in the disease-causing components in the brain in preliminary research conducted by JCR utilizing disease-model mice. These kind of factors are responsible for developing opportunities for the growth of the market.

Complications Associated with Hematopoietic Stem Cell Therapy will Hamper the Growth of the Market

Following a bone marrow transplant, complications might be sudden or gradual. The source of the stem cell transplant, age, baseline performance condition, type of preparative regimen, and intensity can all have an impact on these adverse effects. Chronic side effects include varicella-zoster virus reactivation, encapsulated bacterial infection, and chronic GVHD.

Myelosuppression with anemia, neutropenia, or thrombocytopenia, mucositis, sinusoidal obstruction syndrome, acute graft versus host disease, gram-positive or gram-negative bacterial infections, herpesviridae infections, as well as fungal infections with Aspergillus or Candida are just a few of the acute complications that can happen within the first 90 days.

Segment Analysis

The global fucosidosis treatment market is segmented based on type, treatment and end user.

Owing to High Incidence, the Fucosidosis Type II from Type Segment Accounted for Approximately 62% of the Fucosidosis Treatment Market Share

The fucosidosis type II category from type held the largest market share in 2022 and is expected to maintain its dominance over the forecast period due to a more number of cases of fucosidosis worldwide. Type II is linked to a longer survival time, angiokeratoma corporis diffusum development, and milder psychomotor decline and neurologic symptoms.

Fucosidosis type II sufferers may have coarsened facial features, developmental delays, speech and/or language impairments, hypotonia, slow development, hepatosplenomegaly, vertebral defects, cardiomegaly, minor retinal vascular alterations, epileptic attacks, angiokeratomas, telangiectasias, defects in central regulation of body temperature, and a slowly progressing neurodegenerative condition with a developing encephalopathy, the upper motor neuron dysfunction with hyperreflexia.

Delay in bone age, spondyloepiphyseal dysplasia, dysostosis multiplex, hip dysplasia, as well as diaphyseal expansion of long bones are further abnormalities. Hypomyelination may be detected by brain MRI. White matter in the cerebellum, periventricular region of the brain, lobar region, and subcortical region can be seen on T2-weighted imaging as areas of signal hyperintensity. These all abnormalities are associated with type II fucosidosis.

Geographical Penetration

North America Accounted for Approximately 34.2% of the Market Share in 2022, Owing to the Strong Presence of Major Players and Increasing Healthcare Infrastructure Investment

Due to the rising need for fucosidosis 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 fucosidosis treatment.

Increasing expenditure on healthcare and rising research studies, advancement of technologies for treatment of lysosomal diseases, and increase in pharmaceutical and biotechnology business establishment across the region are also contributing to the growth of fucosidosis treatment market share of this region.

The key healthcare organizations' and businesses' joint research initiatives are also expected to contribute to the rising demand, as will new product development, which constantly looks to improve present options. The market in this area is growing as people become more aware of various treatments, such as bone marrow transplants, for management purposes. The aforementioned elements further attest to North America's hegemonic position in the world.

Competitive Landscape

The major global players in the fucosidosis treatment market include Merck KGaA, JCR Pharmaceuticals Co., Ltd., Orchard Therapeutics plc, Protalix BioTherapeutics, Inc., Magenta Therapeutics, Evotec SE, Jasper Therapeutics, Inc., Hemogenyx Pharmaceuticals plc, Garuda Therapeutics and STEMPEUTICS RESEARCH PVT LTD. Among others.

COVID-19 Impact Analysis

Russia Ukraine Conflict Analysis

Due to the low prevalence as well as absence of major market participants in this region, it is predicted that the Russia-Ukraine conflict will not have a significant effect on the worldwide fucosidosis treatment market. The growth of the global fucosidosis treatment market, however, is anticipated to be relatively unaffected by the import and export of raw materials over the forecast period.

By Type

• Fucosidosis Type I
• Fucosidosis Type II

By Treatment

• Antibiotic Therapy
• Fluid Replacement Therapy
• Hematopoietic-Stem-Cell Transplantation
• Enzyme Replacement Therapy

By End User

• Hospitals
• Ambulatory Surgical Centers
• Specialty Clinics

By Region

• North America
  • U.S.
  • Canada
  • Mexico

• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • 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

Key Developments

• On October 31, 2023, a memorandum of understanding on the granting of special negotiation rights for the global marketing outside of Japan of four fresh drug candidates being produced for ultra-rare lysosomal storage diseases (LSDs) by JCR was executed, Their boards of directors resolved, according to an announcement from JCR Pharmaceuticals Co., Ltd and MEDIPAL HOLDINGS CORPORATION. Additionally, the two businesses have reached an agreement to license for a therapeutic candidate for the management of fucosidosis in line with the execution of the memorandum.
• On February 7, 2023, Garuda Therapeutics disclosed a $62 million Series B funding led by Northpond Ventures, Cormorant Asset Management, OrbiMed Advisors, and Aisling Capital, with involvement from Mass General Brigham Ventures, Sectoral Asset Management, Alexandria Venture Investments, along with other prestigious investors and individuals. The company develops off-the-shelf, robust cellular therapies based on blood stem cell. The company has received $134 million in investment in all.

DMI Opinion:

The global fucosidosis treatment market is going to see moderate growth in upcoming years owing to rising usage of enzyme replacement therapy for fucosidosis treatment. Several researches are taking place worldwide for stem cell transplant therapy development. Numerous advances are taking place leading to the growth of market such as fluid replacement therapy. According to DMI the fucosidosis treatment market will see a decent growth with several novel therapies in the market.

Why Purchase the Report?

• To visualize the global fucosidosis 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 fucosidosis 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 fucosidosis treatment market report would provide approximately 53 tables, 54 figures and 195 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 Research and Development by the Researchers
    • 4.1.1.2. Rising Collaborations for Developing Fucosidosis Treatment
  • 4.1.2. Restraints
    • 4.1.2.1. Complications Associated with Hematopoietic Stem Cell Therapy
  • 4.1.3. Opportunity
    • 4.1.3.1. Rising Robust Pipeline and Technological Advancements
  • 4.1.4. Impact Analysis

5. Industry Analysis

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

6. COVID-19 Analysis

• 6.1. Analysis of COVID-19
  • 6.1.1. Scenario Before COVID-19
  • 6.1.2. Scenario During COVID-19
  • 6.1.3. Scenario Post COVID-19
• 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. Fucosidosis Type I*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Fucosidosis Type II

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. Antibiotic Therapy *
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Fluid Replacement Therapy
• 8.4. Hematopoietic-Stem-Cell Transplantation
• 8.5. Enzyme Replacement Therapy

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. Specialty Clinics

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. U.K.
    • 10.3.6.3. France
    • 10.3.6.4. Spain
    • 10.3.6.5. Italy
    • 10.3.6.6. Rest of Europe
• 10.4. South America
  • 10.4.1. Introduction
  • 10.4.2. Key Region-Specific Dynamics
  • 12.4.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 12.4.2. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 12.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 12.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 12.4.4.1. Brazil
    • 12.4.4.2. Argentina
    • 12.4.4.3. 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. Merck KGaA *
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. JCR Pharmaceuticals Co., Ltd.
• 12.3. Orchard Therapeutics plc
• 12.4. Protalix BioTherapeutics, Inc.
• 12.5. Magenta Therapeutics
• 12.6. Evotec SE
• 12.7. Jasper Therapeutics, Inc.
• 12.8. Hemogenyx Pharmaceuticals plc
• 12.9. Garuda Therapeutics
• 12.10. STEMPEUTICS RESEARCH PVT LTD.

LIST NOT EXHAUSTIVE

13. Appendix

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