鎌状赤血球症治療の世界市場 - 2025年～2033年

世界の鎌状赤血球症治療市場は2023年に27億3,000万米ドルに達し、2032年には70億4,000万米ドルに達すると予測され、予測期間2024年～2033年のCAGRは11.3％で成長します。

鎌状赤血球症（SCD）は、ヘモグロビン産生異常を特徴とする遺伝性血液疾患群です。これらの異常なヘモグロビン細胞は、通常の円盤状とは異なり鎌状であるため、鎌状赤血球症と呼ばれます。これらの細胞は通常の機能を果たせず、しばしば細い血管に蓄積して血流を阻害し、血管閉塞性クリーゼと呼ばれる痛みを伴うエピソード、臓器障害、脳卒中を引き起こします。

鎌状赤血球症（SCD）にはいくつかのタイプがあり、遺伝子変異と遺伝のタイプによって分類されます。ヘモグロビンSS（HbSS）、ヘモグロビンSC（HbSC）、ヘモグロビン（HbS）β-サラセミアなどがあります。鎌状赤血球症はアフリカ系、ヒスパニック系アメリカ人、地中海系などに多くみられます。

鎌状赤血球症の治療法には、輸血、骨髄移植、ヒドロキシ尿素による薬物療法、生物学的製剤などがあります。鎌状赤血球症治療市場は、研究開発活動の活発化、新規製品の承認、有病人口の増加などが主な要因です。

市場力学：

促進要因と抑制要因

鎌状赤血球症治療市場を牽引するのは、新薬開発活動の活発化と規制当局の承認です。

鎌状赤血球症は、有病者数が増加している現在の世界の負担疾患の一つです。患者数が増加しているにもかかわらず、治療の選択肢はわずかな薬剤と高価な輸血や骨髄移植に限られています。アンメットニーズの高さに対処するため、多くのイノベーターや市場企業が厳しい研究開発活動に投資し、患者のために先進的な治療法を発表しています。これらの先進療法には、遺伝子編集療法、ピルビン酸キナーゼ阻害剤、赤血球造血刺激因子製剤などが含まれます。

例えば、2023年12月、米国食品医薬品局（FDA）は、血管閉塞性クリーゼを再発する12歳以上の患者における鎌状赤血球症の治療薬としてCASGEVY（exagamglogene autotemcel[exa-cel]）を承認しました。CASGEVYは、バーテックス・ファーマシューティカルズ・インコーポレーテッドとCRISPRセラピューティクスが共同開発したCRISPR/Cas9ベースの遺伝子編集療法です。承認後、バーテックス社は2024年半ばに最初の患者にCASGEVYを投与しました。

さらに同日、米国FDAはブルーバード・バイオ社のLYFGENIA（lovotibeglogene autotemcel）を同じ適応症で承認しました。ブルーバード社はLYFGENIA投与患者50人の細胞採取を2025年3月31日までに、70人の細胞採取を2025年6月30日までに完了することを目指しています。

一方、エマウス・メディカル社がエナドリという商品名で販売しているL-グルタミン経口粉末は、鎌状赤血球症の症状治療薬として世界中で承認されつつあります。例えば、エナドリは2023年5月にバーレーン国家保健規制当局から、次いで2023年7月にオマーン保健省から、2024年2月にはプエルトリコで販売承認を取得しています。

製品のリコールや流通の不一致は、鎌状赤血球症治療市場の成長を大きく妨げる可能性があります。

鎌状赤血球症の治療選択肢は限られており、製品流通の齟齬や製品回収が市場成長の妨げになる可能性があります。治療法が承認されると、アンメットニーズの高い疾患では採用率が高くなります。メーカーは、こうした患者を治療するための医薬品を定期的に供給しなければならないです。何らかの混乱は、採用率の低下や市場全体の阻害につながる可能性があります。例えば、2025年9月、ファイザー社は鎌状赤血球症の治療を目的としたOXBRYTA（voxelotor）を世界市場から自主的に販売中止しました。ファイザー社はオクスブライタの全ロットを回収し、総合的なベネフィットがリスクを上回らないと評価した後、すべての臨床試験を中止しました。DataMの予測では、Oxbrytaの販売中止により、鎌状赤血球症治療の市場は2025年初頭に混乱すると予想されています。

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
    • 鎌状赤血球症の罹患率の上昇
    • 新薬開発活動と規制当局の承認の増加
  • 抑制要因
    • 製品リコールと流通の不一致
  • 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
• パイプライン分析
• 疫学
• 特許分析
• 規制分析

第6章 疾患タイプ別

• ヘモグロビンSS（HbSS）
• ヘモグロビンSC（HbSC）
• ヘモグロビン（HbS）ベータサラセミア
• その他

第7章 治療の種類別

• 薬物療法
• 遺伝子治療
• 輸血
• 骨髄移植

第8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
  • その他欧州地域
• 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
  • 韓国
  • その他アジア太平洋地域
• 中東・アフリカ

第9章 競合情勢

• 競合シナリオ
• 市況・シェア分析
• M&A分析

第10章 企業プロファイル

• Novartis AG.
  • 会社概要
  • 製品ポートフォリオ
    • 製品説明
    • 製品の主要業績評価指標（KPI）
    • 過去および予測の製品販売
    • 製品販売量
  • 財務概要
    • 会社の収益
    • 地域別収益分配
    • 収益予測
  • 主な発展
    • 合併と買収
    • 主な製品開発活動
    • 規制当局の承認等
  • SWOT分析
• Vertex Pharmaceuticals Incorporated.
• bluebird bio, Inc.
• Emmaus Medical, Inc,
• Bristol-Myers Squibb Company.
• CHIESI FARMACEUTICI S.p.A.
• Teva Pharmaceutical Industries Ltd.
• Similar data will be provided for each market player.
• Agios Pharmaceuticals, Inc.
  • パイプライン製品の説明
  • 製品の主要業績評価指標（KPI）
  • 主な活動
  • 市場参入のタイムライン
  • 製品普及率
  • 売上予測と予測
• Beam Therapeutics.
• Editas Medicine
• Novo Nordisk A/S
• Pfizer Inc.
• 各市場参入企業に対しても同様のデータが提供されます。

第11章 付録

The global sickle cell disease treatment market reached US$2.73 billion in 2023 and is expected to reach US$7.04 billion by 2032, growing at a CAGR of 11.3% during the forecast period 2024-2032.

Sickle cell disease (SCD) is a group of inherited blood disorders characterized by abnormalities in hemoglobin production. These abnormal hemoglobin cells are sickle-shaped unlike the regular disc-shaped hence the name sickle cell disease. These cells cannot perform regular functions and often accumulate in the small blood vessels obstructing blood flow, leading to painful episodes called vaso-occlusive crises, organ damage, and stroke.

There are several types of sickle cell disease (SCD), classified based on the type of gene mutation and inheritance. These include hemoglobin SS (HbSS), hemoglobin SC (HbSC), and hemoglobin (HbS) beta-thalassemia. Sickle cell disease is most common among populations who are of African descent, Hispanic Americans, Mediterranean descent, etc.

The treatment options for sickle cell disease include blood transfusions, bone marrow transplantation, pharmacotherapy with hydroxyurea, and some biologic drugs. The market for sickle cell disease treatment is majorly driven by the rising R&D activities novel product approvals, rising prevalent population.

Market Dynamics: Drivers & Restraints

The rising novel drug development activities and regulatory approvals drive the sickle cell disease treatment market.

Sickle cell disease is one of the current global burdens of diseases with a rising number of prevalent cases. Despite the higher number of cases, the treatment options are limited to a handful of drugs and expensive blood transfusions and bone marrow transplantations. To tackle the high unmet demands, many innovators and market players are investing in rigorous R&D activities and are launching advanced therapies for patients. These advanced therapies include gene editing therapies, pyruvate kinase inhibitors, erythropoiesis-stimulating agents, etc.

For instance, in December 2023, the U.S. Food and Drug Administration (FDA) approved CASGEVY (exagamglogene autotemcel [exa-cel]) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises. CASGEVY is a CRISPR/Cas9-based gene editing therapy co-developed by Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics. Post its approval, Vertex administered CASGEVY to the first patient in the mid of 2024.

Moreover, on the same day, the U.S. FDA approved bluebird bio, Inc.'s LYFGENIA (lovotibeglogene autotemcel) for the same indication. bluebird aims to complete cell collections from 50 LYFGENIA-administered patients by March 31, 2025, and from 70 patients by June 30, 2025.

On the other hand, L-glutamine oral powder sold under the brand name Enadri, manufactured by Emmaus Medical, Inc, is being approved across the globe for the treatment of symptoms of sickle cell disease. For instance, in May 2023, Enadri received marketing approval from the Bahrain National Health Regulatory Authority, followed by approval from the Oman Ministry of Health in July 2023, and in Purto Rico in February 2024.

Product recalls and discrepancies in distribution can significantly hinder Sickle cell disease treatment market growth.

The treatment options for sickle cell disease are limited, and any discrepancies in the product distribution, or product recalls may hinder the market growth. Once a therapy has been approved, the adoption rate is high in conditions with high unmet needs. Manufacturers must ensure a regular supply of medicine to treat these patients. Any disruption can lead to decreased adoption and hindrance of the overall market. For instance, in September 2025, Pfizer Inc. voluntarily discontinued OXBRYTA (voxelotor) from the worldwide markets which was aimed at treating sickle cell disease. Pfizer has recalled all the lots of Oxbryta and also discontinued all the clinical trials after assessing that the overall benefits do not outweigh the risks. As per DataM estimates the market for sickle cell disease treatment is expected to be disrupted in early 2025, due to Oxbryta discontinuation.

Segment Analysis

The global sickle cell disease treatment market is segmented based on disease type, treatment type, and region.

Gene therapy in the treatment type segment is expected to grow with the highest CAGR in the forecasted period.

Gene therapy is a revolutionary treatment option for sickle cell disease and is considered the ultimate cure. They act on the root cause modifying the genes either by gene addition, editing, or removal. The pathophysiology of sickle cell disease lies in the inheritance of mutated genes from parents, and in such conditions, gene therapy comes to the rescue.

Currently, two gene therapies have been approved for use in sickle cell disease and they are notably Casgevy and Lyfgenia. Casgevy (exagamglogene autotemcel) co-developed by Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics is a CRISPR/Cas9-technology-based cellular gene therapy consisting of autologous CD34+ Hematopoietic stem cells (HSc) edited at the erythroid-specific enhancer region of the BCL11A gene to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin (HbF) protein production. Casgevy is prepared from the patient's hematopoietic stem cells and administered as an HSc transplant.

Whereas LYFGENIA (lovotibeglogene autotemcel) developed by Bluebird Bio, Inc. is also prepared from patient's hematopoietic stem cells enriched with CD34+ cells and transduced with BB305 LVV encoding BA-T87Q-globin. This gene therapy is intended for one-time administration to add functional copies of modified genes into a patient's hematopoietic stem cells.

These gene therapies are backed by favorable scientific evidence that demonstrates the safety and efficacy profile. The manufacturers of these two gene therapies are actively involved in regulatory submissions across the globe and are further conducting clinical trials in child populations below 12 years of age.

The adoption rate for these gene therapies is expected to pick up in 2025 and is expected to experience significant growth in the forecast period. Moreover, there are two more potential gene therapies in the pipeline being developed by Beam Therapeutics., and Editas Medicine, which is anticipated to make a market entry in this decade and contribute to the overall segment growth.

Geographical Analysis

North America is estimated to hold the highest market share throughout the forecast period, owing to factors like the rising prevalence of cases of sickle cell disease, increasing research and development activities by market players, strong presence of market players, and higher revenue generation from approved therapies.

For instance, according to the American Society of Hematology and the Centers for Disease Control and Prevention, nearly 100,000 people are living with sickle cell disease in the U.S. which affects 1 in every 365 Black or African American births.

This higher prevalence coupled with the availability of advanced therapies like Casgevy, Lyfgenia, Adekveo, Reblozyl, and Endari represents the high treatment rate among the U.S. patient population. Moreover, these therapies/drugs are sold at higher prices by manufacturers in the U.S. which leads to higher revenue generation. For instance, in 2023, Bristol-Myers Squibb Company., the manufacturer of Rbelozyl generated nearly 80% of the total US$1 billion revenue from the U.S.

All these factors signify the dominance of North America in the global sickle cell disease treatment market.

Competitive Landscape

The major global players in the Sickle cell disease treatment market are Novartis AG., Vertex Pharmaceuticals Incorporated., bluebird bio, Inc., Emmaus Medical, Inc, Bristol-Myers Squibb Company., and CHIESI FARMACEUTICI S.p.A., and Teva Pharmaceutical Industries Ltd. among others.

Emerging Players

The emerging players in the Sickle cell disease treatment market include Agios Pharmaceuticals, Inc., Beam Therapeutics., Editas Medicine, Novo Nordisk A/S, and Pfizer Inc. among others.

Key Developments

• In December 2024, Agios Pharmaceuticals, Inc. has announced that the European Commission (EC) is positive for designating mitapivat as an orphan medicinal product (OMP) for the treatment of sickle cell disease. The drug is an oral, small-molecule PK activator that has met primary endpoints in phase 3 clinical trials (ENERGIZE and ENERGIZE-T) of transfusion-dependent alpha- or beta-thalassemia, post which Agios has filed regulatory applications for mitapivat (PYRUKIND) in the U.S., EU, KSA, and UAE.
• In December 2024, Editas Medicine, Inc. presented the updated results of the RUBY clinical trial evaluating Reni-cel in patients with severe sickle cell disease. The study concluded that Reni-cel is well tolerated and has continued to demonstrate a safety profile. 27 out of 28 patients were free of vaso-occlusive events (VOEs), and early normalization of total hemoglobin was observed in patients.

Why Purchase the Report?

• Pipeline & Innovations: Reviews ongoing clinical trials, and product pipelines, and forecasts upcoming pharmaceutical advancements.
• Disease Type Performance & Market Positioning: Analyzes product performance, market positioning, and growth potential to optimize strategies.
• Real-world Evidence: Integrates patient feedback and data into product development for improved outcomes.
• Physician Preferences & Health System Impact: Examines healthcare provider behaviors and the impact of health system mergers on adoption strategies.
• Market Updates & Industry Changes: Covers recent regulatory changes, new policies, and emerging technologies.
• Competitive Strategies: Analyzes competitor strategies, market share, and emerging players.
• Pricing & Market Access: Reviews pricing models, reimbursement trends, and market access strategies.
• Market Entry & Expansion: Identifies optimal strategies for entering new markets and partnerships.
• Regional Growth & Investment: Highlights high-growth regions and investment opportunities.
• Supply Chain Optimization: Assesses supply chain risks and distribution strategies for efficient product delivery.
• Sustainability & Regulatory Impact: Focuses on eco-friendly practices and evolving regulations in healthcare.
• Post-market Surveillance: Uses post-market data to enhance product safety and access.
• Pharmacoeconomics & Value-Based Pricing: Analyzes the shift to value-based pricing and data-driven decision-making in R&D.

The global Sickle cell disease treatment market report would provide approximately 45 tables, 41 figures, and 210 pages.

Target Audience 2023

• Manufacturers: Pharmaceutical, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
• Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
• Technology & Innovation: R&D Professionals, Clinical Trial Managers, Pharmacovigilance Experts.
• Investors: Healthcare Investors, Venture Fund Investors, Pharma Marketing & Sales.
• Consulting & Advisory: Healthcare Consultants, Industry Associations, Analysts.
• Supply Chain: Distribution and Supply Chain Managers.
• Consumers & Advocacy: Patients, Advocacy Groups, Insurance Companies.
• Academic & Research: Academic Institutions.

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 Disease Type
• 3.2. Snippet by Treatment Type
• 3.3. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Rising prevalence of sickle cell disease
    • 4.1.1.2. Rising novel drug development activities and regulatory approvals
  • 4.1.2. Restraints
    • 4.1.2.1. Product recalls and discrepancies in distribution
  • 4.1.3. Opportunities
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Force Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Pipeline Analysis
• 5.5. Epidemiology
• 5.6. Patent Analysis
• 5.7. Regulatory Analysis

6. By Disease Type

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
  • 6.1.2. Market Attractiveness Index, By Disease Type
• 6.2. Hemoglobin SS (HbSS)*
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. Hemoglobin SC (HbSC)
• 6.4. Hemoglobin (HbS) Beta Thalassemia
• 6.5. Others

7. By Treatment Type

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 7.1.2. Market Attractiveness Index, By Treatment Type
• 7.2. Pharmacotherapy*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Gene Therapy
• 7.4. Blood Transfusions
• 7.5. Bone Marrow Transplantation

8. By Region

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 8.1.2. Market Attractiveness Index, By Region
• 8.2. North America
  • 8.2.1. Introduction
  • 8.2.2. Key Region-Specific Dynamics
  • 8.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
  • 8.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.2.5.1. The U.S.
    • 8.2.5.2. Canada
    • 8.2.5.3. Mexico
• 8.3. Europe
  • 8.3.1. Introduction
  • 8.3.2. Key Region-Specific Dynamics
  • 8.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
  • 8.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.3.5.1. Germany
    • 8.3.5.2. U.K.
    • 8.3.5.3. France
    • 8.3.5.4. Spain
    • 8.3.5.5. Italy
    • 8.3.5.6. Rest of Europe
• 8.4. South America
  • 8.4.1. Introduction
  • 8.4.2. Key Region-Specific Dynamics
  • 8.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
  • 8.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.4.5.1. Brazil
    • 8.4.5.2. Argentina
    • 8.4.5.3. Rest of South America
• 8.5. Asia-Pacific
  • 8.5.1. Introduction
  • 8.5.2. Key Region-Specific Dynamics
  • 8.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
  • 8.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 8.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.5.5.1. China
    • 8.5.5.2. India
    • 8.5.5.3. Japan
    • 8.5.5.4. South Korea
    • 8.5.5.5. Rest of Asia-Pacific
• 8.6. Middle East and Africa
  • 8.6.1. Introduction
  • 8.6.2. Key Region-Specific Dynamics
  • 8.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
  • 8.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type

9. Competitive Landscape

• 9.1. Competitive Scenario
• 9.2. Market Positioning/Share Analysis
• 9.3. Mergers and Acquisitions Analysis

10. Company Profiles

Key Market Players

• 10.1. Novartis AG.*
  • 10.1.1. Company Overview
  • 10.1.2. Product Portfolio
    • 10.1.2.1. Product Description
    • 10.1.2.2. Product Key Performance Indicators (KPIs)
    • 10.1.2.3. Historic and Forecasted Product Sales
    • 10.1.2.4. Product Sales Volume
  • 10.1.3. Financial Overview
    • 10.1.3.1. Company Revenue's
    • 10.1.3.2. Geographical Revenue Shares
    • 10.1.3.3. Revenue Forecasts
  • 10.1.4. Key Developments
    • 10.1.4.1. Mergers & Acquisitions
    • 10.1.4.2. Key Product Development Activities
    • 10.1.4.3. Regulatory Approvals etc.
  • 10.1.5. SWOT Analysis
• 10.2. Vertex Pharmaceuticals Incorporated.
• 10.3. bluebird bio, Inc.
• 10.4. Emmaus Medical, Inc,
• 10.5. Bristol-Myers Squibb Company.
• 10.6. CHIESI FARMACEUTICI S.p.A.
• 10.7. Teva Pharmaceutical Industries Ltd.
• Similar data will be provided for each market player.

Emerging Market Players

• 10.8. Agios Pharmaceuticals, Inc.*
  • 10.8.1. Pipeline Products Description
  • 10.8.2. Product Key Performance Indicators (KPIs)
  • 10.8.3. Key Activities
  • 10.8.4. Market Entry Timelines
  • 10.8.5. Product Penetration Rate
  • 10.8.6. Sales Estimation and Projections
• 10.9. Beam Therapeutics.
• 10.10. Editas Medicine
• 10.11. Novo Nordisk A/S
• 10.12. Pfizer Inc.
• Similar data will be provided for each market player.

LIST NOT EXHAUSTIVE

11. Appendix

• 11.1. About Us and Services
• 11.2. Contact Us