アルファサラセミアの世界市場-2023年～2030年

市場概要

世界のアルファサラセミア市場は、2022年に11億米ドルに達し、2023-2030年の予測期間中にCAGR 7.4%で成長し、2030年には19億米ドルに達すると予測されています。アルファサラセミア市場の動向は、技術的進歩の高まりに起因する調査の上昇を示しています。ヘモグロビンの生成を担う4つのアルファグロビン遺伝子のうち1つ以上が損傷または欠損すると、アルファサラセミアとなります。

さらに、アルファサラセミアに対する鉄キレート剤の採用が増加し、治療が可能になり、市場におけるアルファサラセミア無症候性の症例数が増加し、研究開発が増加していることが、アルファサラセミアの市場規模を押し上げています。

同市場は、この分野における進歩の高まりの結果、北米地域からの治療に対する需要が伸びています。アギオス・ファーマシューティカルズ社、アクティス社、ノバルティス・ファーマシューティカルズ社、太郎製薬株式会社などの競合他社が積極的に参入しています。

市場力学

主要企業による技術進歩と治験の増加がアルファサラセミア市場の成長を牽引

現在、複数の治験が実施され、良好な結果が得られています。2022年11月15日、American Society of Hematologyに掲載された研究結果サラセミア赤血球（RBC）のアデノシン三リン酸（ATP）レベルは、その高いエネルギー要求量を満たすには不十分です。赤血球のATP産生を担う解糖の最終段階を制御する重要な酵素であるピルビン酸キナーゼ（PK）は、薬剤mitapivatによってアロステリックに活性化されます。

先に、輸血依存性または非輸血依存性サラセミア（NTDT）患者を対象としたミタピバットの非盲検第2相試験（NCT03692052）の主な結果では、4～12週（Wk）を含む1回の評価において、80.0％の患者でヘモグロビン（Hb）がベースラインから1.0g/dL上昇する反応が認められました。溶血マーカーも改善し、Hb値は上昇を続け、72週目には平均Hb値が1.7g/dL（0.5）まで上昇しました。前述のような主張の結果、予測期間を通じて市場は主導権を握ると予測されます。

研究開発における新興国市場の協力の高まりが市場開拓の機会を生み出す

長年の研究の結果、現在では多くの大手メーカーが新規製品を開発し、治療目的で発売しており、これがアルファサラセミア市場の拡大を促進しています。いくつかの治療法がアルファサラセミアの管理のために規制当局によって承認されており、主要メーカーはサラセミアを含む希少疾患のための製品開発のために協力しています。

例えば、2023年1月8日、商業段階のバイオ製薬会社であるAmryt Pharma Plcは、希少疾患の新規治療薬の買収、創出、販売に取り組んでおり、研究中心のヘルスケアおよびバイオ製薬グループであるChiesi Farmaceutici S.p.A.は、ChiesiがAmrytを買収することで両社が正式に合意したと発表しました。

アルファサラセミアに対する適切な治療法の欠如が市場の成長を妨げる

現在、アルファサラセミアに対する信頼できる治療法は市場に存在しません。アルファサラセミアはキレーションや輸血では治癒しません。幹細胞移植は、特定のアルファサラセミア患者を治癒させることができます。幹細胞移植の技術は重大であり、危険と隣り合わせです。

アルファサラセミア患者を支援するために、医療関係者や研究者によって他の治療法も開発されています。サラセミアの唯一の治療法は幹細胞移植です。重度のサラセミア患者のうち、適合するドナーが見つかり、手術の良い候補者となれるのはごく一部です。

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
    • 主要企業による技術進歩と治験の増加
    • アルファサラセミアの有病率の上昇
  • 抑制要因
    • アルファサラセミアに対する適切な治療法の欠如
  • 機会
    • 研究開発のための主要企業による共同研究の増加
  • 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

• アルファサラセミアの無症候性キャリア
• アルファサラセミアの保因者
• ヘモグロビンH病
• 大型アルファサラセミア

第8章 治療法別

• 葉酸サプリメント
• 鉄キレート剤
  • デフェロキサミン（デスフェラル）
  • デフェラシロクス
    • エクスジェイド
    • ジャデヌ
  • デフェリプロンまたはL1（フェリプロックス）
• 輸血
• 幹細胞移植

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
  • 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

• Agios Pharmaceuticals, Inc.
  • 企業概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な動向
• Actis
• Novartis Pharmaceuticals Corporation
• Global Calcium PVT LTD
• CHIESI Farmaceutici S.p.A.
• Taro Pharmaceutical Industries Ltd.
• Amgen Inc.
• Sun Pharmaceutical Industries Ltd.
• FRESENIUS SE & Co. KGaA
• Acceleron Pharma

第13章 付録

Market Overview

The Global Alpha Thalassemia Market reached US$ 1.1 billion in 2022 and is expected to reach US$ 1.9 billion by 2030 growing with a CAGR of 7.4% during the forecast period 2023-2030. The alpha thalassemia market trends show rising research owing to the rising technological advancements. When one or more of the four alpha-globin genes-which are responsible for producing hemoglobin-are damaged or absent, alpha thalassemia is the result.

Furthermore, rising adoption of iron chelators for alpha thalassemia due to the rising availability of treatment and increase in the number of alpha thalassemia silent carrier cases in the market, and an increase in the research and development is driving up the alpha thalassemia market size.

The market is experiencing a growth in demand for treatments from North American areas as a result of the rising advancements in this field. With significant competitors like Agios Pharmaceuticals, Inc., Actis, Novartis Pharmaceuticals Corporation, and Taro Pharmaceutical Industries Ltd. actively operating in the market.

Market Dynamics

Growing Advancements and Clinical Trials by Key Players Drive the Growth of the Alpha Thalassemia Market

Several clinical trials are taking place currently and resulting in positive outcomes. On November 15, 2022, a study published in American Society of Hematology Adenosine triphosphate (ATP) levels in thalassemic red blood cells (RBCs) are insufficient to meet their elevated energy requirements. Pyruvate kinase (PK), a crucial enzyme that controls the final stage of glycolysis, which is responsible for producing ATP in RBCs, is activated allosterically by the drug mitapivat.

Earlier, the main findings of an open-label stage 2 study (NCT03692052) of mitapivat in patients with - or -non-transfusion-dependent thalassemia (NTDT) showed that 80.0% of the patients had a reaction in hemoglobin (Hb) of 1.0 g/dL rise from baseline at 1 assessments in between 4-12 weeks (Wk), inclusive. Hemolysis markers also improved, and Hb levels continued to rise, with a mean Hb rising to 1.7 g/dL (0.5) at Wk 72. The market is predicted to take the lead throughout the forecast period as a consequence of the aforementioned claims.

Rising Collaboration by Key Players for Research and Development Creates Opportunities for the Growth of the Market

As a result of years of study, a number of major manufacturers are now developing novel products and releasing them for therapeutic purposes, which is fostering the expansion of the alpha thalassemia market. Several treatments have been approved by the regulatory authorities for management of alpha thalassemia and major players are collaborating for development of products for rare diseases including thalassemia.

For instance, on January 8, 2023, a commercial-stage biopharmaceutical company, Amryt Pharma Plc, committed to acquiring, creating, and marketing novel treatments for rare diseases, and Chiesi Farmaceutici S.p.A., a research-focused healthcare and biopharmaceuticals group, announced that the two companies have come to terms to an official agreement pursuant to which Chiesi will purchase Amryt.

Unavailability of Proper Treatment for Alpha Thalassemia will Hamper the Growth of the Market

There is not a reliable treatment for alpha thalassemia currently on the market. Alpha thalassemia cannot be cured by chelation or blood transfusions. A stem cell transplant can cure certain alpha thalassemia major patients. The technique of a stem cell transplant is serious and fraught with danger.

Other therapies are being developed by medical professionals and researchers to assist those who have alpha thalassemia. The only treatment for thalassemia is a stem cell transplant. Only a small percentage of those with severe thalassemia can locate a donor match and be a good candidate for the operation.

For more details on this report - Request for Sample

Segment Analysis

The global alpha thalassemia market is segmented based type, treatment, end user and region.

Owing to High Utilization, the Iron Chelators from Treatment Segment Accounted for Approximately 40.1% of the Alpha Thalassemia Market Share

The iron chelators category from treatment held the largest market share in 2022 and is expected to maintain its dominance over the forecast period due to a growth in alpha thalassemia types worldwide. Iron overload in thalassemias is primarily brought on by two mechanisms: blood transfusions and increased iron absorption as a result of inefficient erythropoiesis. Atypical dietary iron absorption causes severe thalassemia patients who are not receiving blood transfusions to have an annual rise in body iron burden of 2 to 5 g.

Blood transfusion recipients are more likely to develop iron excess. Iron from all the transfusions can accumulate in the body over time since red blood cells have a lot of iron in them. The accumulation of iron can make it difficult for several organs to function effectively, including the heart, liver, and brain. People having thalassemia may require chelation therapy, in which case doctors provide a medication - either a pill or an injection under the skin - to remove surplus iron as it builds up in the organs, to prevent iron overload.

Due to the widespread use of iron chelation therapy in medical centers during the past few decades, the survival rates of patients with iron excess have significantly increased. The three iron chelators that the United States Food and Drug Administration has approved for clinical use are deferoxamine, deferiprone, and deferasirox. It is generally recognized that these iron chelators can all be used to treat iron overload in a variety of clinical circumstances.

Geographical Penetration

North America Accounted for Approximately 44.7% 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 alpha thalassemia 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 iron chelators for treatment.

Increasing expenditure on healthcare and rising adoption among patients, advancement of technologies for tests or treatment, and increase in pharmaceutical business establishment across the region are also contributing to the growth of alpha thalassemia market share of this region.

It is also anticipated that the main healthcare organizations and enterprises collaborative research projects as well as new product development, which constantly seeks to improve available alternatives, will contribute to the expanding demand. Individuals are becoming more aware of various types of tests for alpha thalaessemia and their treatment that are being utilized for management purposes, leading to the expansion of the market in this region. The above-mentioned factors further proves the dominance of North America on a global scale.

Competitive Landscape

The major global players in the alpha thalassemia market include Agios Pharmaceuticals, Inc., Actis, Novartis Pharmaceuticals Corporation, Global Calcium PVT LTD, CHIESI Farmaceutici S.p.A., Taro Pharmaceutical Industries Ltd., Amgen Inc., Sun Pharmaceutical Industries Ltd., FRESENIUS SE & Co. KGaA and Acceleron Pharma 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 alpha thalassemia market. The growth of the global alpha thalassemia market however, is anticipated to be relatively unaffected by the import and export of raw materials over the forecast period.

By Type

• Alpha Thalassemia Silent Carrier
• Alpha Thalassemia Carrier
• Hemoglobin H Disease
• Alpha Thalassemia Major

By Treatment

• Folic Acid Supplement
• Iron Chelators

Deferoxamine (Desferal)

Deferasirox

• Exjade
• Jadenu

Deferiprone or L1 (Ferriprox)

• Blood Transfusion
• Stem Cell Transplant

By End User

• Hospitals
• Ambulatory Surgical Centers
• Others

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 March 31, 2023, FERRIPROX MR deferiprone extended-release tablets, 1000 mg, have been approved by Health Canada for the treatment of individuals with transfusional iron overload resulting from thalassemia syndromes if current chelation therapy is insufficient, or sickle cell disease (SCD), or other anemias, according to Chiesi Global Rare Diseases, a subsidiary of the Chiesi Group created to deliver novel treatments and solutions to individuals affected by rare diseases.
• On August 11, 2022, data from the core time of the open-label, stage 2 investigation regarding PYRUKYND (mitapivat) in individuals having non-transfusion-dependent - or -thalassemia was released on August 11, 2022, in The Lancet, according to a release from Agios Pharmaceuticals, Inc., an innovator in the field of cellular metabolism and treatments for genetically defined diseases. The 2021 Annual Congress of the European Hematology Association (EHA) featured an earlier presentation of study data. PYRUKYND is an experimental, oral, first-in-class small molecule wild-type allosteric activator and other PK enzyme mutations.
• On April 23, 2021, through its affiliate Novadoz, the Indian company MSN Labs has introduced four recently-approved generic drugs into the US market: albendazole, droxidopa, deferasirox, and pregabalin. For the upcoming ten years, the corporation claims to have a "deep pipeline" of launches planned.

Why Purchase the Report?

• To visualize the global alpha thalassemia 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 alpha thalassemia 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 thalassemia 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 Advancements and Clinical Trials by Key Players
    • 4.1.1.2. Rising Alpha Thalassemia Disease Prevalence
  • 4.1.2. Restraints
    • 4.1.2.1. Unavailability of Proper Treatment for Alpha Thalassemia
  • 4.1.3. Opportunity
    • 4.1.3.1. Rising Collaboration by Key Players for Research and Development
  • 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. Alpha Thalassemia Silent Carrier *
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Alpha Thalassemia Carrier
• 7.4. Hemoglobin H Disease
• 7.5. Alpha Thalassemia Major

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. Folic Acid Supplement *
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Iron Chelators
  • 8.3.1. Deferoxamine (Desferal)
  • 8.3.2. Deferasirox
    • 8.3.2.1. Exjade
    • 8.3.2.2. Jadenu
  • 8.3.3. Deferiprone or L1 (Ferriprox)
• 8.4. Blood Transfusion
• 8.5. Stem Cell Transplant

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. 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. Agios 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. Actis
• 12.3. Novartis Pharmaceuticals Corporation
• 12.4. Global Calcium PVT LTD
• 12.5. CHIESI Farmaceutici S.p.A.
• 12.6. Taro Pharmaceutical Industries Ltd.
• 12.7. Amgen Inc.
• 12.8. Sun Pharmaceutical Industries Ltd.
• 12.9. FRESENIUS SE & Co. KGaA
• 12.10. Acceleron Pharma

LIST NOT EXHAUSTIVE

13. Appendix

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