ベータサラセミア市場：タイプ別、治療法別、流通チャネル別、地域別の機会および予測 (2017-2031年)

世界のベータサラセミア市場は、予測期間2024-2031年にCAGR 7.75%で成長し、2023年の5億300万米ドルから2031年には9億1,392万米ドルに成長すると予測されます。同市場は近年著しい成長を遂げており、今後も堅調な拡大ペースを維持すると予想されます。

ベータサラセミアは、両親から遺伝する最も一般的な常染色体劣性遺伝性疾患であり、βグロビン鎖の合成の欠損または欠如につながるHBB遺伝子の変異による機能的ヘモグロビンの産生低下を特徴とします。重症度により、この疾患はマイナー型、中間型、メジャー型のベータサラセミアに分類されます。輸血、鉄キレート療法、遺伝子治療、骨髄治療など様々な治療法がベータサラセミアの治療に用いられます。骨髄移植は、16歳未満の患者に最も多く行われています。

ベータサラセミア市場は、ベータサラセミアの有病率の増加、治療法の進歩の促進、ポイントオブケア検査の需要増加などの影響を受けています。近年のヘルスケアインフラの成長と医療費の増加は、ベータサラセミア市場を大きく支えています。さらに、血液関連疾患の負担を軽減するための政府の取り組みが、関連製品の需要にプラスの影響を与えています。

ベータサラセミアは、地中海、東南アジア、インド亜大陸、中東のような低所得地域で最も一般的です。これらの地域から米国のような高所得国への移住が有病率を大きく変化させており、例えば、報告された症例数が少ないにもかかわらず、ベータサラセミアの有病率は過去50年間で7.5％増加しています。イタリアのような国々は、ヘモグロビン症を減らすための特定の政策を実施し、これらのイニシアチブは、評価期間中に世界のベータサラセミア市場に影響を与えると予想されます。また、FDAによるベータサラセミア治療薬の承認は、主要企業間の健全な競争を発展させています。

増加するベータサラセミアの有病率

ベータサラセミアは、南アジア、中東、北アフリカ、南欧州で顕著な有病率を示しています。人口の移動が疾病の世界的分布を変化させています。シリアやイラクのような戦争の影響を受けた地域からの難民の流入は、疾病統計の乱れに寄与しているが、ベータサラセミアはまれな疾患と考えられています。ベータサラセミアの世界の有病率は、高い保因率、移民の動向、血族結婚を好む文化的嗜好により増加しています。ベータサラセミアの有病率の増加に伴い、その治療に対する需要は予測期間中に増加すると予想されます。

国際サラセミア連合が発表した「Global Thalassemia Review-2023」によると、世界人口の5～7％が体内のヘモグロビンの生産や機能に影響を与える変異遺伝子の保有者であると推定されています。それによると、年間33万人以上の罹患児が生まれ、そのうち83％が鎌状赤血球症、17％がサラセミアである（約5万6千人の乳児を数える）。

治療法の進歩

輸血や鉄キレート療法などの従来のベータサラセミア治療にはいくつかの限界があったが、最近のベータサラセミア治療の進歩により、グロビン鎖の不均衡の是正、非効率的な赤血球造血への対処、鉄過剰症の改善という3つの主要なカテゴリーに分類される治療法が改善されてきています。これらの新たな治療法には、治験中の赤血球成熟剤、遺伝子治療、体細胞核移植によって得られる胚様幹細胞などが含まれます。現在進行中の遺伝子編集や鉄代謝調節剤の臨床試験では、新たな改善が期待されています。現在、ベータサラセミア患者にはいくつかの治療選択肢があり、ベータサラセミア市場はさらに強化されています。

市場を活性化する規制当局の焦点

規制機関は、新規治療法を評価・承認することで、ベータサラセミア市場を活性化させる重要な役割を担っています。規制機関が新たな治療法に焦点を当てることで、患者にとって新たな治療法の選択肢が増えることになります。例えば、βグロビン遺伝子の機能的な人工コピーを導入するZYNTEGLOのような遺伝子治療の承認は、ベータサラセミア治療における重要なマイルストーンです。さらに、新規遺伝子治療に対する規制当局の支援や、遺伝子治療におけるCRISPR/Cas9と人工多能性幹細胞（IPSC）技術の組み合わせは、規制当局がこの分野の進歩を促進する可能性を示しています。このような革新的な治療法の開発と承認に明確な道筋を示すことで、規制機関は研究開発を改善し、最終的にベータサラセミアの治療状況を拡大することができます。

輸血はベータサラセミアに対する最も一般的な治療法です。重症のベータサラセミアでは、輸血依存性サラセミアとして知られる定期的な輸血を必要とする患者もいます。輸血は体内の赤血球数を増やし、組織への酸素供給を改善するのに役立ちます。しかし、定期的な輸血は鉄過剰症を引き起こす可能性があり、肝臓、心臓、膵臓などの重要な臓器を損傷する可能性がありますが、ベータサラセミア患者には有効であることが証明されています。そのため、過剰な鉄を体外に排出するために鉄キレート療法が必要になることがあります。効果を高めるために、輸血に加えていくつかの併用療法が行われます。例えば、2023年4月にPubMedに掲載された論文によると、輸血依存性サラセミア患者を対象に実施された研究で、ヒドロキシ尿素の併用療法は、これらの患者のヘモグロビン値を改善するだけでなく、輸血間隔を有意に延長することが示されました。

病院薬局が主要な流通チャネル

病院薬局部門は、ベータサラセミア治療の主要な流通チャネルになると予想されています。病院は、この疾患に必要な複雑な医療管理のため、ベータサラセミア治療薬の主要センターです。ベータサラセミア患者は、しばしば定期的な輸血、鉄キレート療法、および治癒の可能性のある治療を必要とするため、広範な医療管理が必要となります。さらに、病院はこれらの治療に関連する専門的なケアやモニタリングに対応できる設備が整っているため、ベータサラセミア治療を実施・管理する主要な場所となっています。

北米がベータサラセミア市場を独占

北米は、ベータサラセミア市場において最も高い市場シェアを持つ主要地域であると予測されています。北米の優位性は、同市場における主要プレイヤーの強い存在感、医療費の増加、サラセミア症例の増加、研究活動の増加、同地域の可処分所得の高さ、医療費の高さ、ヘモグロビン障害を減らすための政府の取り組みなどの要因によって支配されています。現在、最も承認されている遺伝子治療は米国とカナダで承認されており、ベータサラセミア市場の収益の主要なシェアに貢献しています。アジア太平洋および中東・アフリカは、未開発の市場における大きなビジネスチャンスとベータサラセミア有病率の上昇により、高いCAGRで成長すると予測されています。

今後の市場シナリオ（2024年～2031年）

移民動向と疾患分布パターンの変化によるベータサラセミア有病率の増加が、ベータサラセミア市場の成長を大きく牽引しています。

治験中の赤血球成熟剤、遺伝子治療、胚様幹細胞などのベータサラセミア治療の進歩は、ベータサラセミア市場に劇的に貢献しています。

ベータサラセミアに対する新規治療法の開発と承認に対する規制の重点化は、商品化された製品数の増加につながっており、将来の市場成長に寄与すると予想されます。

輸血療法は、高い有効性と費用対効果により、ベータサラセミア市場における治療法の主流になると予想されます。

当レポートでは、世界の除湿機の市場を調査し、市場の定義と概要、市場規模の推移・予測、各種区分・地域別の詳細分析、産業構造、市場成長への影響因子の分析、ケーススタディ、競合情勢、主要企業のプロファイルなどをまとめています。

目次

第1章 調査手法

第2章 プロジェクトの範囲と定義

第3章 エグゼクティブサマリー

第4章 世界のベータサラセミア市場の展望、2017～2031年まで

• 市場規模と予測
  • 値別
  • 数量別
• タイプ別
  • ベータサラセミアマイナー
  • ベータサラセミアインターメディア
  • ベータサラセミアメジャー
• セラピー別
  • 輸血
  • 鉄キレーション療法
  • 葉酸サプリメント
  • 遺伝子治療
  • 骨髄移植
• 流通チャネル別
  • 病院薬局
  • 小売薬局
  • オンライン薬局
• 地域別
  • 北米
  • 欧州
  • アジア太平洋
  • 南米
  • 中東とアフリカ
• 企業別市場シェア（%）、2023年

第5章 世界のベータサラセミア市場の見通し、地域別、2017-2031年

• 北米
  • 市場規模と予測
  • タイプ別
  • セラピー別
  • 流通チャネル別
  • 米国
  • カナダ
  • メキシコ

すべてのセグメントは、対象となるすべての地域および国に提供されます。

• 欧州
  • ドイツ
  • フランス
  • イタリア
  • 英国
  • ロシア
  • オランダ
  • スペイン
  • トルコ
  • ポーランド
• アジア太平洋
  • インド
  • 中国
  • 日本
  • オーストラリア
  • ベトナム
  • 韓国
  • インドネシア
  • フィリピン
• 南米
  • ブラジル
  • アルゼンチン
• 中東とアフリカ
  • サウジアラビア
  • アラブ首長国連邦
  • 南アフリカ

第6章 市場マッピング、2023年

• タイプ別
• セラピー別
• 流通チャネル別
• 地域別

第7章 マクロ環境と産業構造

• 需要供給分析
• 輸出入分析
• バリューチェーン分析
• PESTEL分析
  • 政治的要因
  • 経済システム
  • 社会的影響
  • 技術の進歩
  • 環境への影響
  • 法令順守と規制方針（法的機関を含む）
• ポーターのファイブフォース分析
  • サプライヤーパワー
  • バイヤーパワー
  • 代替の脅威
  • 新規参入者の脅威
  • 競合企業間の敵対関係

第8章 市場力学

• 成長の原動力
• 成長阻害剤（課題と抑制）

第9章 規制の枠組みとイノベーション

• 臨床試験
• 特許の情勢
• 規制当局の承認
• イノベーション/新興テクノロジー

第10章 主要企業の情勢

• 市場リーダー上位5社の競合マトリックス
• 市場リーダー上位5社の市場収益分析（%、2023年）
• 合併および買収/合弁事業（該当する場合）
• SWOT分析（市場参入企業5名向け）

第11章 ケーススタディ

第12章 主要企業の見通し

• Bluebird Bio, Inc.
  • 会社概要
  • 主要な管理担当者
  • 製品とサービス
  • 財務状況（報告どおり）
  • 重点市場と地理的プレゼンス
  • 最近の動向
• Bristol-Myers Squibb Co
• GlaxoSmithKline plc
• Incyte Corporation
• CRISPR Therapeutics AG
• Ionis Pharmaceuticals Inc
• Vertex Pharmaceuticals Incorporated
• Apotex Inc.
• Novartis International AG

上記の企業は市場シェアに応じた注文を保持しておらず、調査作業中に入手可能な情報に従って変更される可能性があります。

第13章 戦略的推奨事項

第14章 米国についてと免責事項

List of Tables

• Table 1. Pricing Analysis of Products from Key Players
• Table 2. Competition Matrix of Top 5 Market Leaders
• Table 3. Mergers & Acquisitions/ Joint Ventures (If Applicable)
• Table 4. About Us - Regions and Countries Where We Have Executed Client Projects

List of Figures

• Figure 1. Global Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 2. Global Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 3. Global Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 4. Global Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 5. Global Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 6. Global Beta Thalassemia Market Share (%), By Region, 2017-2031F
• Figure 7. North America Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 8. North America Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 9. North America Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 10. North America Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 11. North America Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 12. North America Beta Thalassemia Market Share (%), By Country, 2017-2031F
• Figure 13. United States Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 14. United States Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 15. United States Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 16. United States Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 17. United States Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 18. Canada Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 19. Canada Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 20. Canada Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 21. Canada Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 22. Canada Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 23. Mexico Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 24. Mexico Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 25. Mexico Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 26. Mexico Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 27. Mexico Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 28. Europe Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 29. Europe Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 30. Europe Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 31. Europe Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 32. Europe Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 33. Europe Beta Thalassemia Market Share (%), By Country, 2017-2031F
• Figure 34. Germany Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 35. Germany Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 36. Germany Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 37. Germany Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 38. Germany Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 39. France Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 40. France Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 41. France Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 42. France Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 43. France Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 44. Italy Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 45. Italy Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 46. Italy Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 47. Italy Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 48. Italy Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 49. United Kingdom Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 50. United Kingdom Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 51. United Kingdom Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 52. United Kingdom Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 53. United Kingdom Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 54. Russia Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 55. Russia Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 56. Russia Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 57. Russia Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 58. Russia Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 59. Netherlands Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 60. Netherlands Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 61. Netherlands Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 62. Netherlands Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 63. Netherlands Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 64. Spain Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 65. Spain Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 66. Spain Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 67. Spain Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 68. Spain Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 69. Turkey Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 70. Turkey Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 71. Turkey Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 72. Turkey Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 73. Turkey Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 74. Poland Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 75. Poland Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 76. Poland Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 77. Poland Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 78. Poland Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 79. South America Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 80. South America Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 81. South America Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 82. South America Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 83. South America Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 84. South America Beta Thalassemia Market Share (%), By Country, 2017-2031F
• Figure 85. Brazil Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 86. Brazil Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 87. Brazil Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 88. Brazil Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 89. Brazil Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 90. Argentina Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 91. Argentina Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 92. Argentina Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 93. Argentina Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 94. Argentina Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 95. Asia-Pacific Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 96. Asia-Pacific Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 97. Asia-Pacific Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 98. Asia-Pacific Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 99. Asia-Pacific Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 100. Asia-Pacific Beta Thalassemia Market Share (%), By Country, 2017-2031F
• Figure 101. India Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 102. India Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 103. India Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 104. India Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 105. India Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 106. China Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 107. China Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 108. China Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 109. China Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 110. China Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 111. Japan Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 112. Japan Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 113. Japan Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 114. Japan Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 115. Japan Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 116. Australia Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 117. Australia Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 118. Australia Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 119. Australia Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 120. Australia Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 121. Vietnam Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 122. Vietnam Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 123. Vietnam Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 124. Vietnam Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 125. Vietnam Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 126. South Korea Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 127. South Korea Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 128. South Korea Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 129. South Korea Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 130. South Korea Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 131. Indonesia Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 132. Indonesia Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 133. Indonesia Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 134. Indonesia Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 135. Indonesia Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 136. Philippines Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 137. Philippines Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 138. Philippines Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 139. Philippines Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 140. Philippines Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 141. Middle East & Africa Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 142. Middle East & Africa Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 143. Middle East & Africa Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 144. Middle East & Africa Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 145. Middle East & Africa Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 146. Middle East & Africa Beta Thalassemia Market Share (%), By Country, 2017-2031F
• Figure 147. Saudi Arabia Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 148. Saudi Arabia Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 149. Saudi Arabia Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 150. Saudi Arabia Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 151. Saudi Arabia Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 152. UAE Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 153. UAE Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 154. UAE Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 155. UAE Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 156. UAE Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 157. South Africa Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
• Figure 158. South Africa Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
• Figure 159. South Africa Beta Thalassemia Market Share (%), By Type, 2017-2031F
• Figure 160. South Africa Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
• Figure 161. South Africa Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
• Figure 162. By Type Map-Market Size (USD Million) & Growth Rate (%), 2023
• Figure 163. By Therapy Map-Market Size (USD Million) & Growth Rate (%), 2023
• Figure 164. By Distribution Channel Map-Market Size (USD Million) & Growth Rate (%), 2023
• Figure 165. By Region Map-Market Size (USD Million) & Growth Rate (%), 2023

Global beta thalassemia market is projected to witness a CAGR of 7.75% during the forecast period 2024-2031F, growing from USD 503 million in 2023, to USD 913.92 million in 2031. The market has experienced significant growth in recent years and is expected to maintain a strong pace of expansion in the coming years.

Beta thalassemia is the most common autosomal recessive disorder inherited from parents and characterized by a reduced production of functional hemoglobin due to a mutation in the HBB gene that leads to the deficient or absent synthesis of beta-globin chains. Based on the severity, the disorder is categorized into minor, intermediate, and major types of beta thalassemia. A variety of therapies like blood transfusion, iron chelation therapy, gene therapy, and bone marrow therapy treat the beta thalassemia condition. Bone marrow transplants are most common in patients under the age of 16 years.

The beta thalassemia marktherapies andly influenced by the growing prevalence of beta thalassemia, escalated advancement in therapies, and increasing demand for point-of-care testing. Growing healthcare infrastructure and increased healthcare spending in recent years have significantly supported the beta thalassemia market. Additionally, government initiatives to reduce the blood-related disease burden have positively impacted the demand for relevant products.

Beta thalassemia is most common in low-income regions like Mediterranean, Southeast Asia, the Indian subcontinent, and the Middle East. The migration of people from these regions to high-income countries like the United States has significantly changed the prevalence, for example, the prevalence of beta thalassemia has increased by 7.5% in the last 50 years despite the low number of reported cases. Countries like Italy implemented specific policies for reducing hemoglobinopathies, these initiatives are anticipated to impact the global beta thalassemia market during the assessment period. Alongside, FDA approval of beta thalassemia therapies in recent times has developed healthy competition among the key players.

In January 2024, Vertex Pharmaceuticals Incorporated announced that U.S. Food and Drug Administration (FDA) approved its CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy. The therapy is meant for treating transfusion-dependent beta thalassemia (TDT) in patients above the age of 12 years. The therapy is available in the United States for now in hospitals with expertise in stem cell transplantation. CASGEVY has shown a 91.4% success rate in patients with TDT.

Growing Prevalence of Beta Thalassemia

Beta thalassemia has a significant prevalence in South Asia, Middle East, North Africa, and Southern Europe. Population migration is changing the global distribution of disease. The flow of refugee populations from war-affected zones, like Syria and Iraq, is contributing to the disturbance of disease statistics, but beta thalassemia is considered a rare disease. The global prevalence of beta thalassemia is increasing due to a high carrier rate, migration trends, and cultural preference for consanguineous marriages. With the increasing prevalence of beta thalassemia, the demand for its treatment is anticipated to rise during the forecast period.

As per 'Global Thalassemia Review - 2023' published by Thalassemia International Federation estimates, 5-7% of the global population is the carrier of mutated genes affecting the production or function of the hemoglobin in the body. It suggests that over 330,000 affected infants are born annually out of which 83% have sickle cell disorders and 17% are thalassemia (counting around 56,000 infants).

Escalated Advancement in Therapies

Conventional therapies for beta thalassemia, such as transfusion and iron chelation, have several limitations, however, recent advancements in beta thalassemia therapies have led to improved therapies that can be categorized into three major categories, correction of the globin chain imbalance, addressing ineffective erythropoiesis, and improving iron overload. These emerging therapies include investigational erythroid maturation agents, gene therapy, and embryonic-like stem cells obtained by somatic cell nuclear transfer. Ongoing clinical trials of gene editing and agents modulating iron metabolism promise new improvements. Patients with beta thalassemia have several therapeutic options nowadays, further strengthening the beta thalassemia market.

For example, in August 2023, the U.S. FDA approved Bristol Myers Squibb's Reblozyl (luspatercept-aamt) for providing first-line treatment of anemia in adults with lower-risk myelodysplastic syndromes requiring transfusions. Reblozyl is commercialized through global collaboration between Bristol Mayer Squibb and Merck since November 2021. With this approval, Reblozyl is now approved in the United States for patients of anemia, beta thalassemia, and myelodysplastic syndromes.

Focus of Regulatory Bodies to Catalyze the Market

Regulatory bodies play a crucial role in catalyzing the beta thalassemia market by evaluating and approving novel therapies. The focus of regulatory bodies on emerging treatments can improve the availability of novel treatment options for patients. For instance, the approval of gene therapies such as ZYNTEGLO, which introduces functional, engineered copies of beta-globin genes, represents a significant milestone in the treatment of beta thalassemia. Additionally, the regulatory support for novel genetic therapies and the combination of CRISPR/Cas9 and induced pluripotent stem cell (IPSC) technologies in gene therapy demonstrate the potential for regulatory bodies to foster advancements in the field. By providing a clear pathway for developing and approving these innovative therapies, regulatory bodies can improvise the research and development, ultimately expanding the treatment landscape for beta thalassemia.

For example, in March 2023, Bristol Myers Squibb received European Commission approval for Reblozyl (luspatercept) for anemia in adult patients with non-transfusion-dependent beta thalassemia. After the approval, Reblozyl is approved and available in the European Union (EU), the United States, and Canada for treating beta thalassemia patients. The European Commission approved it based on the phase 2 BEYOND study, which proved the efficacy and safety of Reblozyl versus placebo in 145 patients.

Blood Transfusion Segment Dominate the Market

Blood transfusion is the most common type of therapy for beta thalassemia. In more severe cases, such as beta thalassemia major, some patients may require periodic blood transfusions known as transfusion-dependent thalassemia. Blood transfusions help to increase the number of red blood cells in the body and improve oxygen delivery to the tissues. However, regular blood transfusions can lead to iron overload, which can damage vital organs like the liver, heart, and pancreas, but is proven effective in beta thalassemia patients. Therefore, iron chelation therapy may be necessary to remove excess iron from the body. Several conjunctive therapies are provided in addition to blood transfusion to increase efficacy. For instance, as per an article published in PubMed in April 2023, the study conducted among transfusion-dependent thalassemia patients showed that conjunctive therapy of hydroxyurea significantly increases the time interval between transfusions as well as improves the hemoglobin level in these patients.

Hospital Pharmacies are the Leading Distribution Channel

The hospital pharmacies segment is anticipated to be the leading distribution channel for beta thalassemia treatments. Hospitals are major centers for beta thalassemia drugs due to the complex medical management required for this condition. Patients with beta thalassemia often need routine blood transfusions, iron chelation therapy, and potentially curative treatments, which demand extensive medical supervision. Additionally, hospitals are equipped to handle the specialized care and monitoring associated with these treatments, making them primary location for administering and managing beta thalassemia therapies.

North America Dominates Beta Thalassemia Market

North America is anticipated to be the leading region in the beta thalassemia market with the highest market share. The dominance of North America is governed by factors like the strong presence of major players in the market and is characterized by increasing healthcare expenditure, rising cases of thalassemia, a growing number of research activities, high disposable income of the region, high healthcare expenditure, and government initiatives to reduce hemoglobinopathies. Presently, the most approved gene therapies are approved in the United States and Canada, contributing the major share of revenue to the beta thalassemia market. Asia-Pacific and Middle East and Africa are anticipated to grow with high CAGR due to large opportunities in untapped markets and the rising prevalence of beta thalassemia.

Future Market Scenario (2024 - 2031F)

Growing prevalence of beta thalassemia due to migration trends and changes in disease distribution patterns are majorly driving the growth in the beta thalassemia market.

Advancements in beta thalassemia therapies such as investigational erythroid maturation agents, gene therapy, and embryonic-like stem cells drastically contribute to the beta thalassemia market.

Regulatory emphasis on the development and approval of novel therapies for beta thalassemia is leading to an increase in the number of commercialized products which is anticipated to contribute towards the future growth of the market.

Blood transfusion segment is anticipated to dominate therapy type in the beta thalassemia market due to high efficacy and cost-effectiveness.

Key Players Landscape and Outlook

Key participants in the beta thalassemia market include Bluebird Bio, Inc., Bristol-Myers Squibb Co., and Vertex Pharmaceuticals Incorporated. The market caters to several pharmaceutical and biotechnology companies and startups. These companies are involved in the development and commercialization of various treatments and therapies for beta thalassemia. The players in this market are actively involved in product development and approvals, collaboration, and other market tactics. Several players have promising product pipelines and therapies under different phases of clinical trials.

For instance, Shanghai BDgene Co., Ltd., Shenzhen Hemogen, Editas Medicine, Inc., Vertex Pharmaceuticals Incorporated, Bristol-Myers Squibb, Editas Medicine, Inc., CorrectSequence Therapeutics Co., Ltd, are some market players that have genetic and biological products under clinical investigation, presently.

In November 2023, MHRA authorized the world's first gene therapy (Casgevy) to cure sickle-cell anemia and transfusion-dependent B-thalassemia. The therapy was developed by CRISPR therapeutics. Casgevy is the first approved medicine that utilizes the innovative gene-editing technique CRISPR, for which its inventors were awarded the Nobel Prize in 2020.

Table of Contents

1. Research Methodology

2. Project Scope & Definitions

3. Executive Summary

4. Global Beta Thalassemia Market Outlook, 2017-2031F

• 4.1. Market Size & Forecast
  • 4.1.1. By Value
  • 4.1.2. By Volume
• 4.2. By Type
  • 4.2.1. Beta Thalassemia Minor
  • 4.2.2. Beta Thalassemia Intermedia
  • 4.2.3. Beta Thalassemia Major
• 4.3. By Therapy
  • 4.3.1. Blood Transfusions
  • 4.3.2. Iron Chelation Therapy
  • 4.3.3. Folic Acid Supplements
  • 4.3.4. Gene Therapy
  • 4.3.5. Bone Marrow Transplants
• 4.4. By Distribution Channel
  • 4.4.1. Hospital Pharmacies
  • 4.4.2. Retail Pharmacies
  • 4.4.3. Online Pharmacies
• 4.5. By Region
  • 4.5.1. North America
  • 4.5.2. Europe
  • 4.5.3. Asia-Pacific
  • 4.5.4. South America
  • 4.5.5. Middle East and Africa
• 4.6. By Company Market Share (%), 2023

5. Global Beta Thalassemia Market Outlook, By Region, 2017-2031F

• 5.1. North America*
  • 5.1.1. Market Size & Forecast
    • 5.1.1.1. By Value
    • 5.1.1.2. By Volume
  • 5.1.2. By Type
    • 5.1.2.1. Beta Thalassemia Minor
    • 5.1.2.2. Beta Thalassemia Intermedia
    • 5.1.2.3. Beta Thalassemia Major
  • 5.1.3. By Therapy
    • 5.1.3.1. Blood Transfusions
    • 5.1.3.2. Iron Chelation Therapy
    • 5.1.3.3. Folic Acid Supplements
    • 5.1.3.4. Gene Therapy
    • 5.1.3.5. Bone Marrow Transplants
  • 5.1.4. By Distribution Channel
    • 5.1.4.1. Hospital Pharmacies
    • 5.1.4.2. Retail Pharmacies
    • 5.1.4.3. Online Pharmacies
  • 5.1.5. United States*
    • 5.1.5.1. Market Size & Forecast
    • 5.1.5.1.1. By Value
    • 5.1.5.1.2. By Volume
    • 5.1.5.2. By Type
    • 5.1.5.2.1. Beta Thalassemia Minor
    • 5.1.5.2.2. Beta Thalassemia Intermedia
    • 5.1.5.2.3. Beta Thalassemia Major
    • 5.1.5.3. By Therapy
    • 5.1.5.3.1. Blood Transfusions
    • 5.1.5.3.2. Iron Chelation Therapy
    • 5.1.5.3.3. Folic Acid Supplements
    • 5.1.5.3.4. Gene Therapy
    • 5.1.5.3.5. Bone Marrow Transplants
    • 5.1.5.4. By Distribution Channel
    • 5.1.5.4.1. Hospital Pharmacies
    • 5.1.5.4.2. Retail Pharmacies
    • 5.1.5.4.3. Online Pharmacies
  • 5.1.6. Canada
  • 5.1.7. Mexico

All segments will be provided for all regions and countries covered

• 5.1. Europe
  • 5.1.6. Germany
  • 5.1.7. France
  • 5.1.8. Italy
  • 5.1.9. United Kingdom
  • 5.1.10. Russia
  • 5.1.11. Netherlands
  • 5.1.12. Spain
  • 5.1.13. Turkey
  • 5.1.14. Poland
• 5.2. Asia-Pacific
  • 5.2.6. India
  • 5.2.7. China
  • 5.2.8. Japan
  • 5.2.9. Australia
  • 5.2.10. Vietnam
  • 5.2.11. South Korea
  • 5.2.12. Indonesia
  • 5.2.13. Philippines
• 5.3. South America
  • 5.3.6. Brazil
  • 5.3.7. Argentina
• 5.4. Middle East & Africa
  • 5.4.6. Saudi Arabia
  • 5.4.7. UAE
  • 5.4.8. South Africa

6. Market Mapping, 2023

• 6.1. By Type
• 6.2. By Therapy
• 6.3. By Distribution Channel
• 6.4. By Region

7. Macro Environment and Industry Structure

• 7.1. Demand Supply Analysis
• 7.2. Import Export Analysis
• 7.3. Value Chain Analysis
• 7.4. PESTEL Analysis
  • 7.4.6. Political Factors
  • 7.4.7. Economic System
  • 7.4.8. Social Implications
  • 7.4.9. Technological Advancements
  • 7.4.10. Environmental Impacts
  • 7.4.11. Legal Compliances and Regulatory Policies (Statutory Bodies Included)
• 7.5. Porter's Five Forces Analysis
  • 7.5.6. Supplier Power
  • 7.5.7. Buyer Power
  • 7.5.8. Substitution Threat
  • 7.5.9. Threat from New Entrant
  • 7.5.10. Competitive Rivalry

8. Market Dynamics

• 8.1. Growth Drivers
• 8.2. Growth Inhibitors (Challenges and Restraints)

9. Regulatory Framework and Innovation

• 9.1. Clinical Trials
• 9.2. Patent Landscape
• 9.3. Regulatory Approvals
• 9.4. Innovations/Emerging Technologies

10. Key Players Landscape

• 10.1. Competition Matrix of Top Five Market Leaders
• 10.2. Market Revenue Analysis of Top Five Market Leaders (in %, 2023)
• 10.3. Mergers and Acquisitions/Joint Ventures (If Applicable)
• 10.4. SWOT Analysis (For Five Market Players)

11. Case Studies

12. Key Players Outlook

• 12.1. Bluebird Bio, Inc.
  • 12.1.1. Company Details
  • 12.1.2. Key Management Personnel
  • 12.1.3. Products & Services
  • 12.1.4. Financials (As reported)
  • 12.1.5. Key Market Focus & Geographical Presence
  • 12.1.6. Recent Developments
• 12.2. Bristol-Myers Squibb Co
• 12.3. GlaxoSmithKline plc
• 12.4. Incyte Corporation
• 12.5. CRISPR Therapeutics AG
• 12.6. Ionis Pharmaceuticals Inc
• 12.7. Vertex Pharmaceuticals Incorporated
• 12.8. Apotex Inc.
• 12.9. Novartis International AG

Companies mentioned above DO NOT hold any order as per market share and can be changed as per information available during research work.

13. Strategic Recommendations

14. About Us & Disclaimer