ポンペ病市場：治療タイプ、分子タイプ、剤形、投与経路別-2025～2030年の世界予測

ポンペ病市場は、2023年に15億米ドルと評価され、2024年には15億9,000万米ドルに達すると予測され、CAGR 5.61％で成長し、2030年には22億1,000万米ドルに達すると予測されています。

ポンペ病は、体内の細胞内にグリコーゲンが蓄積することで生じるまれな遺伝性疾患であり、特殊な治療ニーズと課題によって特徴づけられるユニークな市場環境を提示しています。市場の中心は酵素補充療法と潜在的な遺伝子治療で、乳児期から晩期発症まで様々な病型に対応しています。治療の必要性は、心筋や骨格筋に影響を及ぼすこの病気の衰弱性によって強調されており、継続的な医療介入の重要な役割が強調されています。主要用途セグメントは、遺伝子研究、酵素開発、患者管理システムなどです。最終用途は主に、これらの治療を実施し、患者のケアを管理するための医療施設、研究機関、専門クリニックです。

市場の成長は、バイオテクノロジーの進歩や希少遺伝性疾患に対する認識の高まりに大きく影響され、より正確な診断や効果的な治療に対する需要の高まりにつながっています。さらに、希少疾病用医薬品の指定や助成金による政府支援は、研究努力や医薬品開発を後押しし、成長の道を開きます。個別化医療の拡大やCRISPRのような新しい遺伝子編集技術の統合は、画期的な治療法として期待されています。しかし、高額な治療費、限られた患者数、厳格な規制プロセスといった課題が大きな制約となり、市場拡大に影響を与えています。また、安全で効果的な治療法を開発することの複雑さも、新興企業の市場参入を妨げています。

このセグメントでの技術革新は、酵素補充療法の送達システムの強化、遺伝子治療技術の進歩、早期かつ正確な発見のための診断ツールの改良に焦点を当てるべきです。共同研究イニシアチブを拡大し、バイオテクノロジー企業との戦略的パートナーシップを模索することは、貴重な洞察とリソースを提供し、市場の成長を促進することができます。ポンペ病市場の性質は比較的ニッチであるが、技術的能力の進化に伴い大幅な進歩が見込まれるため、コストと規制という高い障壁を乗り越えながら最先端の研究を活用して治療イノベーションを実現できる企業には大きな可能性があります。

市場力学：急速に進化するポンペ病市場の主要市場洞察を公開

ポンペ病市場は、需要と供給のダイナミックな相互作用によって変貌を遂げています。このような市場力学の進化を理解することで、企業は十分な情報に基づいた投資決定、戦略的決定の精緻化、そして新たなビジネス機会の獲得に備えることができます。こうした動向を包括的に把握することで、企業は政治的、地理的、技術的、社会的、経済的な領域にわたる様々なリスクを軽減することができるとともに、消費者行動とそれが製造コストや購買動向に与える影響についてより明確に理解することができます。

• 市場促進要因
  • ポンペ病の普及拡大
  • 長期治療の普及と診断・治療の進歩
  • 遺伝子治療と酵素補充療法の開発の増加
• 市場抑制要因
  • ポンペ病の高い治療費
• 市場機会
  • 研究開発への投資の増加
  • 革新的な製品の開発・製造に対する政府の取り組み
• 市場課題
  • 治療に対する認識不足

ポーターのファイブフォース：ポンペ病市場をナビゲートする戦略ツール

ポーターのファイブフォースフレームワークは、市場情勢の競合情勢を理解するための重要なツールです。ポーターのファイブフォースフレームワークは、企業の競合を評価し、戦略的機会を探るための明確な手法を記載しています。このフレームワークは、企業が市場内の勢力図を評価し、新規事業の収益性を判断するのに役立ちます。これら洞察により、企業は自社の強みを活かし、弱みに対処し、潜在的な課題を回避することができ、より強靭な市場でのポジショニングを確保することができます。

PESTLE分析：ポンペ病市場における外部からの影響の把握

外部マクロ環境要因は、ポンペ病市場の業績力学を形成する上で極めて重要な役割を果たします。政治的、経済的、社会的、技術的、法的、環境的要因の分析は、これらの影響をナビゲートするために必要な情報を記載しています。PESTLE要因を調査することで、企業は潜在的なリスクと機会をよりよく理解することができます。この分析により、企業は規制、消費者の嗜好、経済動向の変化を予測し、先を見越した積極的な意思決定を行う準備ができます。

市場シェア分析：ポンペ病市場における競合情勢の把握

ポンペ病市場の詳細な市場シェア分析により、ベンダーの業績を包括的に評価することができます。企業は、収益、顧客ベース、成長率などの主要指標を比較することで、競争上のポジショニングを明らかにすることができます。この分析により、市場の集中、セグメント化、統合の動向が明らかになり、ベンダーは競争が激化する中で自社の地位を高める戦略的意思決定を行うために必要な知見を得ることができます。

FPNVポジショニングマトリックス：ポンペ病市場におけるベンダーのパフォーマンス評価

FPNVポジショニングマトリックスは、ポンペ病市場においてベンダーを評価するための重要なツールです。このマトリックスにより、ビジネス組織はベンダーのビジネス戦略と製品満足度に基づき評価することで、目標に沿った十分な情報に基づいた意思決定を行うことができます。4つの象限によってベンダーを明確かつ正確にセグメント化し、戦略目標に最適なパートナーやソリューションを特定することができます。

戦略分析と推奨：ポンペ病市場における成功への道筋を描く

ポンペ病市場の戦略分析は、世界市場でのプレゼンス強化を目指す企業にとって不可欠です。主要なリソース、能力、業績指標を検討することで、企業は成長機会を特定し、改善に取り組むことができます。このアプローチにより、競合情勢における課題を克服し、新たなビジネス機会を活かして長期的な成功を収めるための体制を整えることができます。

本レポートでは、主要な注目セグメントを網羅した市場の包括的な分析を提供しています。

1.市場の浸透度：現在の市場環境の詳細なレビュー、主要企業による広範なデータ、市場でのリーチと全体的な影響力を評価します。

2.市場の開拓度：新興市場における成長機会を特定し、既存セグメントにおける拡大可能性を評価し、将来の成長に向けた戦略的ロードマップを記載しています。

3.市場の多様化：最近の製品発売、未開拓の地域、産業の主要な進歩、市場を形成する戦略的投資を分析します。

4.競合の評価と情報：競合情勢を徹底的に分析し、市場シェア、事業戦略、製品ポートフォリオ、認証、規制当局の承認、特許動向、主要企業の技術進歩などを検証します。

5.製品開発とイノベーション：将来の市場成長を促進すると期待される最先端技術、研究開発活動、製品イノベーションをハイライトしています。

また、利害関係者が十分な情報を得た上で意思決定できるよう、重要な質問にも答えています。

1.現在の市場規模と今後の成長予測は？

2.最高の投資機会を提供する製品、地域はどこか？

3.市場を形成する主要技術動向と規制の影響とは？

4.主要ベンダーの市場シェアと競合ポジションは？

5.ベンダーの市場参入・撤退戦略の原動力となる収益源と戦略的機会は何か？

目次

第1章 序文

第2章 調査手法

第3章 エグゼクティブサマリー

第4章 市場概要

第5章 市場洞察

• 市場力学
  • 促進要因
    • ポンペ病の普及率の上昇
    • 長期治療の普及と診断・治療の進歩
    • 遺伝子治療と酵素補充療法の進歩
  • 抑制要因
    • ポンペ病の高額な治療費
  • 機会
    • 研究開発への投資の増加
    • 革新的な製品の開発と製造に向けた政府の取り組み
  • 課題
    • 治療に関する認識の欠如
• 市場セグメンテーション分析
• ポーターのファイブフォース分析
• PESTEL分析
  • 政治
  • 経済
  • 社会
  • 技術
  • 法律
  • 環境

第6章 ポンペ病市場：治療タイプ別

• イントロダクション
• 酵素
• 基板

第7章 ポンペ病市場：分子タイプ別

• イントロダクション
• 生物製剤
• 小分子

第8章 ポンペ病市場：剤形別

• イントロダクション
• 液体
• 固体

第9章 ポンペ病市場：投与経路別

• イントロダクション
• 経口
• 保護者による

第10章 南北アメリカのポンペ病市場

• イントロダクション
• アルゼンチン
• ブラジル
• カナダ
• メキシコ
• 米国

第11章 アジア太平洋のポンペ病市場

• イントロダクション
• オーストラリア
• 中国
• インド
• インドネシア
• 日本
• マレーシア
• フィリピン
• シンガポール
• 韓国
• 台湾
• タイ
• ベトナム

第12章 欧州・中東・アフリカのポンペ病市場

• イントロダクション
• デンマーク
• エジプト
• フィンランド
• フランス
• ドイツ
• イスラエル
• イタリア
• オランダ
• ナイジェリア
• ノルウェー
• ポーランド
• カタール
• ロシア
• サウジアラビア
• 南アフリカ
• スペイン
• スウェーデン
• スイス
• トルコ
• アラブ首長国連邦
• 英国

第13章 競合情勢

• 市場シェア分析、2023年
• FPNVポジショニングマトリックス、2023年
• 競合シナリオ分析
• 戦略分析と提言

企業一覧

• Astellas Pharma Europe Ltd
• B Braun Melsungen AG
• Becton, Dickinson & Company
• CENTOGENE N.V.
• Flowserve Corporation
• Genethon
• GlaxoSmithKline PLC
• Koninklijke Philips NV
• Merck KGaA
• Novartis AG
• Oxyrane UK Ltd.
• Pfizer Inc.
• Pharming Group NV
• Sanofi S.A.
• Takeda Pharmaceutical Company Limited

LIST OF FIGURES

• FIGURE 1. POMPE DISEASE MARKET RESEARCH PROCESS
• FIGURE 2. POMPE DISEASE MARKET SIZE, 2023 VS 2030
• FIGURE 3. GLOBAL POMPE DISEASE MARKET SIZE, 2018-2030 (USD MILLION)
• FIGURE 4. GLOBAL POMPE DISEASE MARKET SIZE, BY REGION, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 5. GLOBAL POMPE DISEASE MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 6. GLOBAL POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2023 VS 2030 (%)
• FIGURE 7. GLOBAL POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 8. GLOBAL POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2023 VS 2030 (%)
• FIGURE 9. GLOBAL POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 10. GLOBAL POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2023 VS 2030 (%)
• FIGURE 11. GLOBAL POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 12. GLOBAL POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2023 VS 2030 (%)
• FIGURE 13. GLOBAL POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 14. AMERICAS POMPE DISEASE MARKET SIZE, BY COUNTRY, 2023 VS 2030 (%)
• FIGURE 15. AMERICAS POMPE DISEASE MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 16. UNITED STATES POMPE DISEASE MARKET SIZE, BY STATE, 2023 VS 2030 (%)
• FIGURE 17. UNITED STATES POMPE DISEASE MARKET SIZE, BY STATE, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 18. ASIA-PACIFIC POMPE DISEASE MARKET SIZE, BY COUNTRY, 2023 VS 2030 (%)
• FIGURE 19. ASIA-PACIFIC POMPE DISEASE MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 20. EUROPE, MIDDLE EAST & AFRICA POMPE DISEASE MARKET SIZE, BY COUNTRY, 2023 VS 2030 (%)
• FIGURE 21. EUROPE, MIDDLE EAST & AFRICA POMPE DISEASE MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 22. POMPE DISEASE MARKET SHARE, BY KEY PLAYER, 2023
• FIGURE 23. POMPE DISEASE MARKET, FPNV POSITIONING MATRIX, 2023

LIST OF TABLES

• TABLE 1. POMPE DISEASE MARKET SEGMENTATION & COVERAGE
• TABLE 2. UNITED STATES DOLLAR EXCHANGE RATE, 2018-2023
• TABLE 3. GLOBAL POMPE DISEASE MARKET SIZE, 2018-2030 (USD MILLION)
• TABLE 4. GLOBAL POMPE DISEASE MARKET SIZE, BY REGION, 2018-2030 (USD MILLION)
• TABLE 5. GLOBAL POMPE DISEASE MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 6. POMPE DISEASE MARKET DYNAMICS
• TABLE 7. GLOBAL POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 8. GLOBAL POMPE DISEASE MARKET SIZE, BY ENZYME, BY REGION, 2018-2030 (USD MILLION)
• TABLE 9. GLOBAL POMPE DISEASE MARKET SIZE, BY SUBSTRATE, BY REGION, 2018-2030 (USD MILLION)
• TABLE 10. GLOBAL POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 11. GLOBAL POMPE DISEASE MARKET SIZE, BY BIOLOGICS, BY REGION, 2018-2030 (USD MILLION)
• TABLE 12. GLOBAL POMPE DISEASE MARKET SIZE, BY SMALL MOLECULES, BY REGION, 2018-2030 (USD MILLION)
• TABLE 13. GLOBAL POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 14. GLOBAL POMPE DISEASE MARKET SIZE, BY LIQUID, BY REGION, 2018-2030 (USD MILLION)
• TABLE 15. GLOBAL POMPE DISEASE MARKET SIZE, BY SOLID, BY REGION, 2018-2030 (USD MILLION)
• TABLE 16. GLOBAL POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 17. GLOBAL POMPE DISEASE MARKET SIZE, BY ORAL, BY REGION, 2018-2030 (USD MILLION)
• TABLE 18. GLOBAL POMPE DISEASE MARKET SIZE, BY PARENTAL, BY REGION, 2018-2030 (USD MILLION)
• TABLE 19. AMERICAS POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 20. AMERICAS POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 21. AMERICAS POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 22. AMERICAS POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 23. AMERICAS POMPE DISEASE MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 24. ARGENTINA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 25. ARGENTINA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 26. ARGENTINA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 27. ARGENTINA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 28. BRAZIL POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 29. BRAZIL POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 30. BRAZIL POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 31. BRAZIL POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 32. CANADA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 33. CANADA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 34. CANADA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 35. CANADA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 36. MEXICO POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 37. MEXICO POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 38. MEXICO POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 39. MEXICO POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 40. UNITED STATES POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 41. UNITED STATES POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 42. UNITED STATES POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 43. UNITED STATES POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 44. UNITED STATES POMPE DISEASE MARKET SIZE, BY STATE, 2018-2030 (USD MILLION)
• TABLE 45. ASIA-PACIFIC POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 46. ASIA-PACIFIC POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 47. ASIA-PACIFIC POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 48. ASIA-PACIFIC POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 49. ASIA-PACIFIC POMPE DISEASE MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 50. AUSTRALIA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 51. AUSTRALIA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 52. AUSTRALIA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 53. AUSTRALIA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 54. CHINA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 55. CHINA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 56. CHINA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 57. CHINA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 58. INDIA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 59. INDIA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 60. INDIA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 61. INDIA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 62. INDONESIA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 63. INDONESIA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 64. INDONESIA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 65. INDONESIA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 66. JAPAN POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 67. JAPAN POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 68. JAPAN POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 69. JAPAN POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 70. MALAYSIA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 71. MALAYSIA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 72. MALAYSIA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 73. MALAYSIA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 74. PHILIPPINES POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 75. PHILIPPINES POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 76. PHILIPPINES POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 77. PHILIPPINES POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 78. SINGAPORE POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 79. SINGAPORE POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 80. SINGAPORE POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 81. SINGAPORE POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 82. SOUTH KOREA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 83. SOUTH KOREA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 84. SOUTH KOREA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 85. SOUTH KOREA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 86. TAIWAN POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 87. TAIWAN POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 88. TAIWAN POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 89. TAIWAN POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 90. THAILAND POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 91. THAILAND POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 92. THAILAND POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 93. THAILAND POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 94. VIETNAM POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 95. VIETNAM POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 96. VIETNAM POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 97. VIETNAM POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 98. EUROPE, MIDDLE EAST & AFRICA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 99. EUROPE, MIDDLE EAST & AFRICA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 100. EUROPE, MIDDLE EAST & AFRICA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 101. EUROPE, MIDDLE EAST & AFRICA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 102. EUROPE, MIDDLE EAST & AFRICA POMPE DISEASE MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 103. DENMARK POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 104. DENMARK POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 105. DENMARK POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 106. DENMARK POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 107. EGYPT POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 108. EGYPT POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 109. EGYPT POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 110. EGYPT POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 111. FINLAND POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 112. FINLAND POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 113. FINLAND POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 114. FINLAND POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 115. FRANCE POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 116. FRANCE POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 117. FRANCE POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 118. FRANCE POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 119. GERMANY POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 120. GERMANY POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 121. GERMANY POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 122. GERMANY POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 123. ISRAEL POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 124. ISRAEL POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 125. ISRAEL POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 126. ISRAEL POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 127. ITALY POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 128. ITALY POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 129. ITALY POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 130. ITALY POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 131. NETHERLANDS POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 132. NETHERLANDS POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 133. NETHERLANDS POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 134. NETHERLANDS POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 135. NIGERIA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 136. NIGERIA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 137. NIGERIA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 138. NIGERIA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 139. NORWAY POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 140. NORWAY POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 141. NORWAY POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 142. NORWAY POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 143. POLAND POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 144. POLAND POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 145. POLAND POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 146. POLAND POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 147. QATAR POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 148. QATAR POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 149. QATAR POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 150. QATAR POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 151. RUSSIA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 152. RUSSIA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 153. RUSSIA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 154. RUSSIA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 155. SAUDI ARABIA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 156. SAUDI ARABIA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 157. SAUDI ARABIA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 158. SAUDI ARABIA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 159. SOUTH AFRICA POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 160. SOUTH AFRICA POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 161. SOUTH AFRICA POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 162. SOUTH AFRICA POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 163. SPAIN POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 164. SPAIN POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 165. SPAIN POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 166. SPAIN POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 167. SWEDEN POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 168. SWEDEN POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 169. SWEDEN POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 170. SWEDEN POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 171. SWITZERLAND POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 172. SWITZERLAND POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 173. SWITZERLAND POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 174. SWITZERLAND POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 175. TURKEY POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 176. TURKEY POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 177. TURKEY POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 178. TURKEY POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 179. UNITED ARAB EMIRATES POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 180. UNITED ARAB EMIRATES POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 181. UNITED ARAB EMIRATES POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 182. UNITED ARAB EMIRATES POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 183. UNITED KINGDOM POMPE DISEASE MARKET SIZE, BY THERAPY TYPE, 2018-2030 (USD MILLION)
• TABLE 184. UNITED KINGDOM POMPE DISEASE MARKET SIZE, BY MOLECULE TYPE, 2018-2030 (USD MILLION)
• TABLE 185. UNITED KINGDOM POMPE DISEASE MARKET SIZE, BY DOSAGE FORM, 2018-2030 (USD MILLION)
• TABLE 186. UNITED KINGDOM POMPE DISEASE MARKET SIZE, BY ROUTE OF ADMINISTRATION, 2018-2030 (USD MILLION)
• TABLE 187. POMPE DISEASE MARKET SHARE, BY KEY PLAYER, 2023
• TABLE 188. POMPE DISEASE MARKET, FPNV POSITIONING MATRIX, 2023

The Pompe Disease Market was valued at USD 1.50 billion in 2023, expected to reach USD 1.59 billion in 2024, and is projected to grow at a CAGR of 5.61%, to USD 2.21 billion by 2030.

Pompe Disease, a rare inherited disorder resulting from the buildup of glycogen in the body's cells, presents a unique market landscape characterized by specialized therapeutic needs and challenges. The scope of the market centers on enzyme replacement therapies and potential gene therapies to address varying forms of the disease, ranging from infantile-onset to late-onset Pompe Disease. The necessity for treatment is underscored by the disease's debilitating nature, which impacts cardiac and skeletal muscles, emphasizing the vital role of ongoing medical intervention. Key application areas include genetic research, enzyme development, and patient management systems. End-use primarily involves healthcare facilities, research institutes, and specialty clinics equipped to administer these treatments and manage patient care.

Market growth is significantly influenced by advances in biotechnology and increasing awareness of rare genetic disorders, leading to more accurate diagnoses and increased demand for effective treatments. Furthermore, governmental support through orphan drug designations and grants propels research endeavors and drug development, creating avenues for growth. Potential opportunities abound in the expansion of personalized medicine and the integration of novel gene-editing technologies like CRISPR, which hold promise for groundbreaking interventions. However, challenges such as high treatment costs, limited patient population, and stringent regulatory processes pose considerable limitations and impact market expansion. The complexity of developing safe and effective therapies also deters market entry for new firms.

Innovation in this field should focus on enhancing delivery systems for enzyme replacement therapies, advancing genetic therapy techniques, and improving diagnostic tools for early and accurate detection. Expanding collaborative research initiatives and exploring strategic partnerships with biotech firms can provide valuable insights and resources, fostering market growth. The nature of the Pompe Disease market is relatively niche yet poised for substantial advancement as technological capabilities evolve, presenting significant potential for firms that can navigate the high barriers of cost and regulation while leveraging cutting-edge research for therapeutic innovation.

Market Dynamics: Unveiling Key Market Insights in the Rapidly Evolving Pompe Disease Market

The Pompe Disease Market is undergoing transformative changes driven by a dynamic interplay of supply and demand factors. Understanding these evolving market dynamics prepares business organizations to make informed investment decisions, refine strategic decisions, and seize new opportunities. By gaining a comprehensive view of these trends, business organizations can mitigate various risks across political, geographic, technical, social, and economic domains while also gaining a clearer understanding of consumer behavior and its impact on manufacturing costs and purchasing trends.

• Market Drivers
  • Growing prevalence of pompe disease
  • Uptake of long term therapies and advancement in the diagnosis and treatment
  • Increasing developments in the gene therapy and enzyme replacement therapies
• Market Restraints
  • High treatment costs of pompe diseases
• Market Opportunities
  • Increasing investment in the research and development
  • Government initiatives for the development and manufacture of innovative products
• Market Challenges
  • Lack of awareness about the treatment

Porter's Five Forces: A Strategic Tool for Navigating the Pompe Disease Market

Porter's five forces framework is a critical tool for understanding the competitive landscape of the Pompe Disease Market. It offers business organizations with a clear methodology for evaluating their competitive positioning and exploring strategic opportunities. This framework helps businesses assess the power dynamics within the market and determine the profitability of new ventures. With these insights, business organizations can leverage their strengths, address weaknesses, and avoid potential challenges, ensuring a more resilient market positioning.

PESTLE Analysis: Navigating External Influences in the Pompe Disease Market

External macro-environmental factors play a pivotal role in shaping the performance dynamics of the Pompe Disease Market. Political, Economic, Social, Technological, Legal, and Environmental factors analysis provides the necessary information to navigate these influences. By examining PESTLE factors, businesses can better understand potential risks and opportunities. This analysis enables business organizations to anticipate changes in regulations, consumer preferences, and economic trends, ensuring they are prepared to make proactive, forward-thinking decisions.

Market Share Analysis: Understanding the Competitive Landscape in the Pompe Disease Market

A detailed market share analysis in the Pompe Disease Market provides a comprehensive assessment of vendors' performance. Companies can identify their competitive positioning by comparing key metrics, including revenue, customer base, and growth rates. This analysis highlights market concentration, fragmentation, and trends in consolidation, offering vendors the insights required to make strategic decisions that enhance their position in an increasingly competitive landscape.

FPNV Positioning Matrix: Evaluating Vendors' Performance in the Pompe Disease Market

The Forefront, Pathfinder, Niche, Vital (FPNV) Positioning Matrix is a critical tool for evaluating vendors within the Pompe Disease Market. This matrix enables business organizations to make well-informed decisions that align with their goals by assessing vendors based on their business strategy and product satisfaction. The four quadrants provide a clear and precise segmentation of vendors, helping users identify the right partners and solutions that best fit their strategic objectives.

Strategy Analysis & Recommendation: Charting a Path to Success in the Pompe Disease Market

A strategic analysis of the Pompe Disease Market is essential for businesses looking to strengthen their global market presence. By reviewing key resources, capabilities, and performance indicators, business organizations can identify growth opportunities and work toward improvement. This approach helps businesses navigate challenges in the competitive landscape and ensures they are well-positioned to capitalize on newer opportunities and drive long-term success.

Key Company Profiles

The report delves into recent significant developments in the Pompe Disease Market, highlighting leading vendors and their innovative profiles. These include Astellas Pharma Europe Ltd, B Braun Melsungen AG, Becton, Dickinson & Company, CENTOGENE N.V., Flowserve Corporation, Genethon, GlaxoSmithKline PLC, Koninklijke Philips NV, Merck KGaA, Novartis AG, Oxyrane UK Ltd., Pfizer Inc., Pharming Group NV, Sanofi S.A., and Takeda Pharmaceutical Company Limited.

Market Segmentation & Coverage

This research report categorizes the Pompe Disease Market to forecast the revenues and analyze trends in each of the following sub-markets:

• Based on Therapy Type, market is studied across Enzyme and Substrate.
• Based on Molecule Type, market is studied across Biologics and Small Molecules.
• Based on Dosage Form, market is studied across Liquid and Solid.
• Based on Route of Administration, market is studied across Oral and Parental.
• Based on Region, market is studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, Thailand, and Vietnam. The Europe, Middle East & Africa is further studied across Denmark, Egypt, Finland, France, Germany, Israel, Italy, Netherlands, Nigeria, Norway, Poland, Qatar, Russia, Saudi Arabia, South Africa, Spain, Sweden, Switzerland, Turkey, United Arab Emirates, and United Kingdom.

The report offers a comprehensive analysis of the market, covering key focus areas:

1. Market Penetration: A detailed review of the current market environment, including extensive data from top industry players, evaluating their market reach and overall influence.

2. Market Development: Identifies growth opportunities in emerging markets and assesses expansion potential in established sectors, providing a strategic roadmap for future growth.

3. Market Diversification: Analyzes recent product launches, untapped geographic regions, major industry advancements, and strategic investments reshaping the market.

4. Competitive Assessment & Intelligence: Provides a thorough analysis of the competitive landscape, examining market share, business strategies, product portfolios, certifications, regulatory approvals, patent trends, and technological advancements of key players.

5. Product Development & Innovation: Highlights cutting-edge technologies, R&D activities, and product innovations expected to drive future market growth.

The report also answers critical questions to aid stakeholders in making informed decisions:

1. What is the current market size, and what is the forecasted growth?

2. Which products, segments, and regions offer the best investment opportunities?

3. What are the key technology trends and regulatory influences shaping the market?

4. How do leading vendors rank in terms of market share and competitive positioning?

5. What revenue sources and strategic opportunities drive vendors' market entry or exit strategies?

Table of Contents

1. Preface

• 1.1. Objectives of the Study
• 1.2. Market Segmentation & Coverage
• 1.3. Years Considered for the Study
• 1.4. Currency & Pricing
• 1.5. Language
• 1.6. Stakeholders

2. Research Methodology

• 2.1. Define: Research Objective
• 2.2. Determine: Research Design
• 2.3. Prepare: Research Instrument
• 2.4. Collect: Data Source
• 2.5. Analyze: Data Interpretation
• 2.6. Formulate: Data Verification
• 2.7. Publish: Research Report
• 2.8. Repeat: Report Update

3. Executive Summary

4. Market Overview

5. Market Insights

• 5.1. Market Dynamics
  • 5.1.1. Drivers
    • 5.1.1.1. Growing prevalence of pompe disease
    • 5.1.1.2. Uptake of long term therapies and advancement in the diagnosis and treatment
    • 5.1.1.3. Increasing developments in the gene therapy and enzyme replacement therapies
  • 5.1.2. Restraints
    • 5.1.2.1. High treatment costs of pompe diseases
  • 5.1.3. Opportunities
    • 5.1.3.1. Increasing investment in the research and development
    • 5.1.3.2. Government initiatives for the development and manufacture of innovative products
  • 5.1.4. Challenges
    • 5.1.4.1. Lack of awareness about the treatment
• 5.2. Market Segmentation Analysis
• 5.3. Porter's Five Forces Analysis
  • 5.3.1. Threat of New Entrants
  • 5.3.2. Threat of Substitutes
  • 5.3.3. Bargaining Power of Customers
  • 5.3.4. Bargaining Power of Suppliers
  • 5.3.5. Industry Rivalry
• 5.4. PESTLE Analysis
  • 5.4.1. Political
  • 5.4.2. Economic
  • 5.4.3. Social
  • 5.4.4. Technological
  • 5.4.5. Legal
  • 5.4.6. Environmental

6. Pompe Disease Market, by Therapy Type

• 6.1. Introduction
• 6.2. Enzyme
• 6.3. Substrate

7. Pompe Disease Market, by Molecule Type

• 7.1. Introduction
• 7.2. Biologics
• 7.3. Small Molecules

8. Pompe Disease Market, by Dosage Form

• 8.1. Introduction
• 8.2. Liquid
• 8.3. Solid

9. Pompe Disease Market, by Route of Administration

• 9.1. Introduction
• 9.2. Oral
• 9.3. Parental

10. Americas Pompe Disease Market

• 10.1. Introduction
• 10.2. Argentina
• 10.3. Brazil
• 10.4. Canada
• 10.5. Mexico
• 10.6. United States

11. Asia-Pacific Pompe Disease Market

• 11.1. Introduction
• 11.2. Australia
• 11.3. China
• 11.4. India
• 11.5. Indonesia
• 11.6. Japan
• 11.7. Malaysia
• 11.8. Philippines
• 11.9. Singapore
• 11.10. South Korea
• 11.11. Taiwan
• 11.12. Thailand
• 11.13. Vietnam

12. Europe, Middle East & Africa Pompe Disease Market

• 12.1. Introduction
• 12.2. Denmark
• 12.3. Egypt
• 12.4. Finland
• 12.5. France
• 12.6. Germany
• 12.7. Israel
• 12.8. Italy
• 12.9. Netherlands
• 12.10. Nigeria
• 12.11. Norway
• 12.12. Poland
• 12.13. Qatar
• 12.14. Russia
• 12.15. Saudi Arabia
• 12.16. South Africa
• 12.17. Spain
• 12.18. Sweden
• 12.19. Switzerland
• 12.20. Turkey
• 12.21. United Arab Emirates
• 12.22. United Kingdom

13. Competitive Landscape

• 13.1. Market Share Analysis, 2023
• 13.2. FPNV Positioning Matrix, 2023
• 13.3. Competitive Scenario Analysis
• 13.4. Strategy Analysis & Recommendation

Companies Mentioned

• 1. Astellas Pharma Europe Ltd
• 2. B Braun Melsungen AG
• 3. Becton, Dickinson & Company
• 4. CENTOGENE N.V.
• 5. Flowserve Corporation
• 6. Genethon
• 7. GlaxoSmithKline PLC
• 8. Koninklijke Philips NV
• 9. Merck KGaA
• 10. Novartis AG
• 11. Oxyrane UK Ltd.
• 12. Pfizer Inc.
• 13. Pharming Group NV
• 14. Sanofi S.A.
• 15. Takeda Pharmaceutical Company Limited