首頁 > 市場調查報告書 > 製藥

孤兒藥

市場調查報告書

商品編碼

1738518

全球鐮狀細胞疾病治療市場規模（按類型、藥物類別、最終用戶、區域範圍和預測）

Global Sickle Cell Disease Treatment Market Size By Type (Bone Marrow Transplant, Blood Transfusion), By Drug Class (Hydroxyurea, L-glutamine, Voxelotor), By End-User (Hospitals, Specialty Clinics), By Geographic Scope And Forecast

出版日期: 2025年05月12日 | 出版商:

Verified Market Research | 英文 202 Pages | 商品交期: 2-3個工作天內

價格

簡介目錄

鐮狀細胞疾病治療市場規模與預測

2024 年鐮狀細胞疾病治療市場規模價值 33 億美元，預計到 2032 年將達到 103 億美元，在 2026-2032 年預測期內的複合年成長率為 16.85%。

鐮狀細胞疾病 (SCD) 是一種遺傳性血液疾病，其特徵是血紅蛋白異常，導致紅血球呈鐮狀，阻塞血管並引起疼痛、感染疾病和器官損傷。
鐮狀細胞性猝死 (SCD) 的治療包括骨髓移植、輸血和藥物治療，以控制症狀和預防併發症。骨髓移植是唯一的治療方法，但由於捐贈者相容性問題，其可用性有限，因此藥物治療對大多數患者來說是必不可少的。
隨著基因治療和根治性治療方法的進步為長期疾病管理和改善患者預後帶來希望，鐮狀細胞疾病治療的未來看起來更加光明。

全球鐮狀細胞疾病治療市場動態

影響全球鐮狀細胞疾病治療市場的關鍵市場動態是：

關鍵市場促進因素

基因治療的進展：基因治療的進展：基因治療的發展透過針對致病基因突變，為治療鐮狀細胞疾病帶來了巨大的希望。 CRISPR 和其他鐮狀編輯技術的進步有望徹底改變治療格局，為患者提供治癒選擇。
鐮狀細胞病患疾病率上升：鐮狀細胞疾病患者數量正在增加，尤其是在撒哈拉以南非洲、中東和印度的部分地區，這刺激了對有效治療方法的需求。旨在篩檢和早期診斷該疾病的公共衛生措施正在推動市場成長。
政府和非政府組織 (NGO) 的計劃：各國政府和非政府組織不斷加強，提供治療機會、提高公眾意識並資助新治療方法的研究，這些措施正在支持治療市場的發展。旨在擴大發展中地區骨髓登記和藥物治療可近性的計畫也為發展做出了貢獻。

主要問題

治療費用高：骨髓移植和基因治療等治療費用昂貴，限制了低收入社區患者獲得治療的機會。此外，SCD 的長期治療需要定期輸血和藥物治療，這進一步增加了患者和醫療保健系統的經濟負擔。
骨髓移植機會有限：骨髓移植有可能治癒SCD，但找到合適的捐贈者是一項艱鉅的挑戰。患者和捐贈者之間需要基因匹配，這限制了骨髓移植的可近性，尤其是在醫療基礎設施有限的地區。
開發中地區缺乏認知和醫療保健服務：在低收入地區，許多患者缺乏全面的醫療保健服務，導致診斷和治療困難。農村地區對SCD的認知度低，導致診斷延誤，發病率和死亡率居高不下。

主要趨勢

基因治療和治療方法：基因治療的研究和開發正在為SCD的根治性治療鋪平道路。目前，旨在糾正SCD致病基因的臨床試驗正在進行中，這為依賴終身治療的患者帶來了新的希望。
引入新藥療法：SCD 的藥物研發正在取得進展，Voxelotor 和 L-谷氨醯胺等新療法已核准，可減少疼痛發作的頻率並改善血紅蛋白水平。這些治療方法旨在改善 SCD 患者的生活品質。
全球衛生計劃：各國政府、非政府組織和醫療保健組織正在加強全球衛生工作，以改善SCD治療的可近性，尤其是在疾病負擔較高的非洲和印度。這些舉措旨在提高公眾意識、篩檢和治療方法。

Sickle Cell Disease (SCD) is an inherited blood disorder characterized by abnormal hemoglobin, resulting in red blood cells adopting a sickle-like shape. This leads to blockages in blood vessels causing pain, infections, and organ damage.
Treatments for SCD include bone marrow transplants, blood transfusions, and drug therapies aimed at managing the symptoms and preventing complications. While bone marrow transplants are the only potential cure, their availability is limited due to donor compatibility issues making drug treatments essential for most patients.
The future of SCD treatment is evolving with advancements in gene therapies and curative treatments offering hope for long-term disease management and improved patient outcomes.

Global Sickle Cell Disease Treatment Market Dynamics

The key market dynamics that are shaping the global sickle cell disease treatment market include:

Key Market Drivers:

Advancements in Gene Therapy: The development of gene therapies offers significant potential for treating SCD by targeting the genetic mutations that cause the disease. Breakthroughs in CRISPR and other gene-editing technologies are anticipated to revolutionize the treatment landscape providing curative options for patients.
Rising Prevalence of Sickle Cell Disease: The growing number of SCD cases, particularly in sub-Saharan Africa, the Middle East, and parts of India is increasing the demand for effective treatment solutions. Public health initiatives aimed at screening and diagnosing the disease early are boosting market growth.
Government and Non-Governmental Organization (NGO) Initiatives: Increased efforts by governments and NGOs to provide access to care, improve awareness, and fund research for new therapies are supporting the treatment market. Programs aimed at increasing bone marrow registries and access to drug therapies in developing regions are also contributing to market expansion.

Key Challenges:

High Costs of Treatment: Treatments such as bone marrow transplants and gene therapies are expensive limiting their accessibility to patients in lower-income regions. Additionally, long-term management of SCD requires regular blood transfusions and drug therapies, further increasing the economic burden on patients and healthcare systems.
Limited Access to Bone Marrow Transplants: While bone marrow transplants offer the potential for curing SCD, finding compatible donors remains a significant challenge. The need for genetic matches between patients and donors restricts the availability of transplants, particularly in regions with limited healthcare infrastructure.
Lack of Awareness and Healthcare Access in Developing Regions: In low-income regions, many patients lack access to comprehensive healthcare services, making diagnosis and treatment difficult. Limited awareness of SCD in rural areas results in delayed diagnosis leading to higher morbidity and mortality rates.

Key Trends:

Gene Therapy and Curative Approaches: Research and development in gene therapies are paving the way for potentially curative treatments for SCD. Clinical trials are ongoing to determine the efficacy of these therapies in modifying or correcting the faulty gene responsible for SCD offering new hope for patients who currently rely on lifelong treatments.
Introduction of New Drug Therapies: Drug development for SCD is progressing, with new therapies such as voxelotor and L-glutamine gaining approval for reducing the frequency of painful crises and improving hemoglobin levels. These therapies are aimed at improving the quality of life for SCD patients.
Global Health Initiatives: Governments, NGOs, and healthcare organizations are increasing their focus on global health initiatives to ensure better access to SCD treatments, particularly in Africa and India, where the disease burden is high. These initiatives aim to improve awareness, screening, and treatment availability.

Global Sickle Cell Disease Treatment Market Regional Analysis

Here is a more detailed regional analysis of the global sickle cell disease treatment market

North America:

North America dominates the global sickle cell disease treatment market, with the United States holding the largest market share. This is driven by a well-established healthcare infrastructure, advancements in gene therapies, and increased funding for research and development. The U.S. government, through the National Institutes of Health (NIH), has initiated various programs to support SCD treatment research and improve patient care. Additionally, the availability of advanced treatments such as gene therapy and new drug approvals is contributing to market growth.
Organizations such as the National Institutes of Health (NIH) and the Centers for Disease Control and Prevention (CDC) are actively involved in research and awareness campaigns to address the issues presented by SCD. There is also extensive collaboration between pharmaceutical corporations and research institutions to produce more effective medicines such as voxelotor and crizanlizumab which have just received FDA approval. With higher awareness, improved diagnoses, and increased treatment options, the North American market is positioned to expand further meeting the patient population's unmet requirements and enhancing the overall quality of life for those living with sickle cell disease.

Asia Pacific:

The Asia-Pacific region is witnessing significant growth in the SCD treatment market driven by increasing awareness of the disease and improving healthcare infrastructure. Countries like India and Pakistan have high incidences of SCD, with public health programs being implemented to improve screening and diagnosis. Government initiatives aimed at increasing access to bone marrow transplants and affordable drug therapies are expected to fuel market growth. Additionally, rising investments in healthcare and partnerships with global health organizations are improving the availability of advanced treatment options in the region.
Advances in treatment choices and technologies are changing the SCD treatment landscape in Asia Pacific. Traditional treatments, such as hydroxyurea, blood transfusions, and pain management medicines are being supplemented with new therapies, such as gene therapy and developing biologics with the potential to greatly enhance quality of life. Clinical trials for novel medicines are gaining traction, as pharmaceutical companies engage in R&D to bring viable cures to market. Furthermore, public-private partnerships and collaborations encourage the delivery of vital pharmaceuticals and access to comprehensive care.

Global Sickle Cell Disease Treatment Market: Segmentation Analysis

The Global Sickle Cell Disease Treatment Market is segmented based on Type, Drug Class, End-User, and Geography.

Sickle Cell Disease Treatment Market, By Type

Bone Marrow Transplant
Blood Transfusion

Based on Type, the Global Sickle Cell Disease Treatment Market is bifurcated into Bone Marrow Transplant and Blood Transfusion. The bone marrow transplant segment is dominant due to potential curative options for sickle cell disease (SCD), though it is limited by the availability of compatible donors. Blood Transfusion is a widely used treatment method, particularly for managing acute symptoms and preventing complications such as stroke and organ damage in SCD patients.

Sickle Cell Disease Treatment Market, By Drug Class

Hydroxyurea
L-glutamine
Voxelotor

Based on Drug Class, the Global Sickle Cell Disease Treatment Market is segmented into Hydroxyurea, L-glutamine, and Voxelotor. Hydroxyurea is the most commonly used medication for SCD, significantly reducing the frequency of painful episodes and the need for blood transfusions. Voxelotor and L-glutamine are newer drug therapies aimed at improving hemoglobin levels and reducing the severity of disease symptoms providing additional treatment options for patients.

Sickle Cell Disease Treatment Market, By End-User

Hospitals
Specialty Clinics

Based on the End-User, the Global Sickle Cell Disease Treatment Market is bifurcated into Hospitals and Specialty Clinics. Hospitals dominate the market as they provide comprehensive treatment options including blood transfusions, bone marrow transplants, and access to specialized medications. Specialty Clinics focus on long-term care management for SCD patients offering personalized treatment plans and outpatient care for ongoing monitoring.

Key Players

The Global Sickle Cell Disease Treatment Market study report provides valuable insights with an emphasis on the global market. The major players in the market include Global Blood Therapeutics, Inc., Novartis AG, Pfizer Inc., Emmaus Life Sciences, Inc., Bluebird Bio, Inc., Sangamo Therapeutics, Inc., and CRISPR Therapeutics AG.

Our market analysis also includes a section solely dedicated to these major players, offering insights into their financial statements, product benchmarking, and SWOT analysis. The competitive landscape section covers key development strategies, market share, and ranking analysis of the above-mentioned players globally.

Global Sickle Cell Disease Treatment Market Key Developments

In March 2024, Global Blood Therapeutics received FDA approval for its drug voxelotor, which is designed to improve hemoglobin levels and reduce complications in SCD patients.
In August 2024, Novartis announced positive results from its clinical trials of gene therapy for SCD, showing promising results in reducing the need for blood transfusions.
In October 2024, Bluebird Bio announced advancements in its gene-editing therapy using CRISPR technology, with early trials showing potential for curative treatment in SCD patients.

1 INTRODUCTION OF GLOBAL SICKLE CELL DISEASE 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 SICKLE CELL DISEASE 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 SICKLE CELL DISEASE TREATMENT MARKET, BY TYPE

5.1 Overview
5.2 Bone Marrow Transplant
5.3 Blood Transfusion

6 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET, BY DRUG CLASS

6.1 Overview
6.2 Hydroxyurea
6.3 L-glutamine
6.4 Voxelotor

7 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET, BY END-USER

7.1 Overview
7.2 Hospitals
7.3 Specialty Clinics

8 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET, BY GEOGRAPHY

8.1 Overview
8.2 North America
- 8.2.1 U.S.
- 8.2.2 Canada
- 8.2.3 Mexico
8.3 Europe
- 8.3.1 Germany
- 8.3.2 France
- 8.3.3 Spain
- 8.3.4 Italy
- 8.3.5 U.K.
- 8.3.6 Rest of Europe
8.4 Asia Pacific
- 8.4.1 China
- 8.4.2 Japan
- 8.4.3 India
- 8.4.4 South Korea
- 8.4.5 Australia
- 8.4.6 Rest of Asia Pacific
8.5 Rest of the World
- 8.5.1 Latin America
- 8.5.2 Middle East and Africa

9 GLOBAL SICKLE CELL DISEASE TREATMENT MARKET COMPETITIVE LANDSCAPE

9.1 Overview
9.2 Company Market Ranking
9.3 Key Development Strategies

10 COMPANY PROFILES

10.1 Global Blood Therapeutics, Inc.
- 10.1.1 Overview
- 10.1.2 Financial Performance
- 10.1.3 Product Outlook
- 10.1.4 Key Developments
10.2 Novartis AG
- 10.2.1 Overview
- 10.2.2 Financial Performance
- 10.2.3 Product Outlook
- 10.2.4 Key Developments
10.3 Pfizer Inc.
- 10.3.1 Overview
- 10.3.2 Financial Performance
- 10.3.3 Product Outlook
- 10.3.4 Key Developments
10.4 Emmaus Life Sciences, Inc.
- 10.4.1 Overview
- 10.4.2 Financial Performance
- 10.4.3 Product Outlook
- 10.4.4 Key Developments
10.5 Bluebird Bio, Inc.
- 10.5.1 Overview
- 10.5.2 Financial Performance
- 10.5.3 Product Outlook
- 10.5.4 Key Developments
10.6 Sangamo Therapeutics, Inc.
- 10.6.1 Overview
- 10.6.2 Financial Performance
- 10.6.3 Product Outlook
- 10.6.4 Key Developments
10.7 CRISPR Therapeutics AG.
- 10.7.1 Overview
- 10.7.2 Financial Performance
- 10.7.3 Product Outlook
- 10.7.4 Key Developments