脊髓性肌肉萎縮症治療市場、機會、成長動力、產業趨勢分析與預測，2024-2032

全球脊髓性肌肉萎縮症 (SMA) 治療市場價值為 73 億美元，預計 2024 年至 2032 年複合年成長率為 19.1%。主要促進因素包括擴大新生兒篩檢計劃，從而實現早期診斷並提高治療資格。早期診斷可以改善患者的治療效果並增加對神經系統藥物的需求。 Health Union, LLC 報告稱，全球每 6,000 至 10,000 名新生兒中就有 1 人受 SMA 影響，其中 1 型 SMA 最為嚴重，佔所有病例的一半以上。目前大約有 25,000 名美國人患有 SMA。診斷技術的進步以及醫療保健提供者和患者意識的提高預計將推動市場成長。

整個脊髓性肌肉萎縮症治療產業根據類型、治療類型、給藥途徑和地區進行分類。

全球 SMA 治療市場按類型分為嬰兒 SMA、Werdnig-Hoffmann 病、成人 SMA 和 Kugelberg-Welander 病。韋德尼格-霍夫曼病細分市場到2023 年以29 億美元的銷售額引領市場。及時治療。這一趨勢將推動市場成長。研究和開發以及降低治療成本的製藥合作擴大了患者的取得範圍，進一步推動了市場成長。 SMA 1 型治療的早期使用或同情使用計劃也增強了市場動力。

按治療類型分類，市場包括藥物、支持性護理和脊椎手術。包括反義寡核苷酸、基因治療和其他藥物在內的藥物細分市場，到2023年將佔64.2%的佔有率。 SMN蛋白，改變了SMA治療方法產量，拉動市場成長。注射劑和口服製劑的出現增加了治療的可及性，滿足了不同患者的需求和偏好，推動了市場的成長。

北美，尤其是美國，到 2023 年將佔據全球 SMA 治療市場 45.6% 的佔有率，預計將保持其主導地位。對罕見疾病的支持政策、先進的醫療設施和正在進行的臨床試驗是推動該地區市場成長的關鍵因素。此外，領先製藥公司的存在以及研發資金的增加也進一步提振了市場。對脊髓性肌肉萎縮症認知的提高和早期診斷也有助於擴大治療範圍。

目錄

第 1 章：方法與範圍

第 2 章：執行摘要

第 3 章：產業洞察

• 產業生態系統分析
• 產業影響力
  • 成長動力
    • 提高嬰兒和兒童脊髓性肌肉萎縮症的診斷率
    • 基因治療的進展
    • 醫療保健提供者提高意識和篩檢計劃
  • 產業陷阱與挑戰
    • 新療法獲得嚴格的監管批准
    • 治療費用高
• 成長潛力分析
• 監管環境
• 未來市場趨勢
• 管道分析
• 波特的分析
• PESTEL分析

第 4 章：競爭格局

• 介紹
• 公司市佔率分析
• 公司矩陣分析
• 主要市場參與者的競爭分析
• 競爭定位矩陣
• 戰略儀表板

第 5 章：市場估計與預測：按類型，2021 - 2032

• 主要趨勢
• 韋德尼格-霍夫曼病
• 嬰兒SMA
• 庫格伯格-韋蘭德病
• 成人SMA

第 6 章：市場估計與預測：按治療類型，2021 - 2032 年

• 主要趨勢
• 藥物
  • 基因治療
  • 反義寡核苷酸
  • 其他藥物
• 支持性護理
  • 物理治療
  • 呼吸支持
  • 營養支持
• 脊椎手術

第 7 章：市場估計與預測：按管理途徑，2021 - 2032 年

• 主要趨勢
• 口服
• 注射用

第 8 章：市場估計與預測：按最終用途，2021 - 2032 年

• 主要趨勢
• 醫院
• 專科診所
• 家庭護理設置
• 其他最終用戶

第 9 章：市場估計與預測：按地區，2021 - 2032

• 主要趨勢
• 北美洲
  • 美國
  • 加拿大
• 歐洲
  • 德國
  • 英國
  • 法國
  • 西班牙
  • 義大利
  • 荷蘭
  • 歐洲其他地區
• 亞太地區
  • 日本
  • 中國
  • 印度
  • 澳洲
  • 韓國
  • 亞太地區其他地區
• 拉丁美洲
  • 巴西
  • 墨西哥
  • 拉丁美洲其他地區
• 中東和非洲
  • 南非
  • 沙烏地阿拉伯
  • 阿拉伯聯合大公國
  • 中東和非洲其他地區

第 10 章：公司簡介

• American Physical Therapy Association
• Astellas Pharma
• Beijing Jinlan Gene Technology Co., Ltd.
• Biogen Inc.
• Boston's Children Hospital
• Children's Hospital of The King's Daughters
• F. Hoffmann-La Roche Ltd.
• Hanugen Therapeutics
• Nationwide Children's Hospital
• NMD Pharma A/S
• Novartis AG
• Pfizer, Inc.
• Sanofi (Genzyme Corporation)
• Scholar Rock, Inc

The Global Spinal Muscular Atrophy (SMA) Treatments Market was valued at USD 7.3 billion and is projected to grow at a CAGR of 19.1% from 2024-2032. Key drivers include the expansion of newborn screening programs, leading to earlier diagnoses and increased treatment eligibility. Early diagnosis enhances patient outcomes and boosts demand for neurological medications. Health Union, LLC reports that SMA affects 1 in every 6,000 to 10,000 newborns globally, with Type 1 SMA being the most severe and accounting for over half of all cases. Approximately 25,000 Americans currently live with SMA. Advancements in diagnostic technologies and increased awareness among healthcare providers and patients are anticipated to drive market growth.

The overall spinal muscular atrophy treatment industry is classified based on type, treatment type, route of administration, and region.

The global SMA treatment market is segmented by type into infant SMA, Werdnig-Hoffmann disease, adult SMA, and Kugelberg-Welander disease. The Werdnig-Hoffmann disease segment led the market with USD 2.9 billion in 2023. Mandatory newborn screenings have facilitated earlier detections, enabling prompt treatments that are most effective before significant motor neuron loss. This trend is set to drive market growth. Research and development, along with pharmaceutical collaborations to reduce treatment costs, have broadened patient access, further energizing market growth. Early access or compassionate use programs for SMA Type 1 treatment also bolster market momentum.

By treatment type, the market includes medication, supportive care, and spinal surgery. The medication segment, which includes antisense oligonucleotides, gene therapy, and other drugs, dominated with a 64.2% share in 2023. Innovative drugs like Spinraza (Nusinersen) and Evrysdi (Risdiplam) have transformed SMA treatment by targeting the genetic root and enhancing SMN protein production, driving market growth. The availability of both injectable and oral formulations has increased treatment accessibility, catering to diverse patient needs and preferences, thus fueling market growth.

North America, particularly the U.S., captured a significant 45.6% share of the global SMA treatment market in 2023 and is expected to maintain its dominance. Supportive policies for rare diseases, advanced healthcare facilities, and ongoing clinical trials are key factors driving market growth in the region. Additionally, the presence of leading pharmaceutical companies and increased funding for research and development are further bolstering the market. Rising awareness and early diagnosis of spinal muscular atrophy are also contributing to the expanding treatment landscape.

Table of Contents

Chapter 1 Methodology and Scope

• 1.1 Market scope and definitions
• 1.2 Research design
  • 1.2.1 Research approach
  • 1.2.2 Data collection methods
• 1.3 Base estimates and calculations
  • 1.3.1 Base year calculation
  • 1.3.2 Key trends for market estimation
• 1.4 Forecast model
• 1.5 Primary research and validation
  • 1.5.1 Primary sources
  • 1.5.2 Data mining sources

Chapter 2 Executive Summary

• 2.1 Industry 360°synopsis

Chapter 3 Industry Insights

• 3.1 Industry ecosystem analysis
• 3.2 Industry impact forces
  • 3.2.1 Growth drivers
    • 3.2.1.1 Increase in diagnosis rate of spinal muscular atrophy in infants and children
    • 3.2.1.2 Advancement in gene therapy
    • 3.2.1.3 Growing awareness and screening programs by healthcare providers
  • 3.2.2 Industry pitfalls and challenges
    • 3.2.2.1 Stringent regulatory approval for new treatments
    • 3.2.2.2 High cost of treatment
• 3.3 Growth potential analysis
• 3.4 Regulatory landscape
• 3.5 Future market trends
• 3.6 Pipeline analysis
• 3.7 Porter's analysis
• 3.8 PESTEL analysis

Chapter 4 Competitive Landscape, 2023

• 4.1 Introduction
• 4.2 Company market share analysis
• 4.3 Company matrix analysis
• 4.4 Competitive analysis of major market players
• 4.5 Competitive positioning matrix
• 4.6 Strategic dashboard

Chapter 5 Market Estimates and Forecast, By Type, 2021 - 2032 ($ Mn)

• 5.1 Key trends
• 5.2 Werdnig-Hoffmann disease
• 5.3 Infant SMA
• 5.4 Kugelberg-Welander disease
• 5.5 Adult SMA

Chapter 6 Market Estimates and Forecast, By Treatment Type, 2021 - 2032 ($ Mn)

• 6.1 Key trends
• 6.2 Medication
  • 6.2.1 Gene therapy
  • 6.2.2 Antisense oligonucleotide
  • 6.2.3 Other medications
• 6.3 Supportive care
  • 6.3.1 Physical therapy
  • 6.3.2 Respiratory support
  • 6.3.3 Nutritional support
• 6.4 Spinal surgery

Chapter 7 Market Estimates and Forecast, By Route of Administration, 2021 - 2032 ($ Mn)

• 7.1 Key trends
• 7.2 Oral
• 7.3 Parenteral

Chapter 8 Market Estimates and Forecast, By End-Use, 2021 - 2032 ($ Mn)

• 8.1 Key trends
• 8.2 Hospitals
• 8.3 Specialty clinics
• 8.4 Homecare settings
• 8.5 Other end-users

Chapter 9 Market Estimates and Forecast, By Region, 2021 - 2032 ($ Mn)

• 9.1 Key trends
• 9.2 North America
  • 9.2.1 U.S.
  • 9.2.2 Canada
• 9.3 Europe
  • 9.3.1 Germany
  • 9.3.2 UK
  • 9.3.3 France
  • 9.3.4 Spain
  • 9.3.5 Italy
  • 9.3.6 Netherlands
  • 9.3.7 Rest of Europe
• 9.4 Asia Pacific
  • 9.4.1 Japan
  • 9.4.2 China
  • 9.4.3 India
  • 9.4.4 Australia
  • 9.4.5 South Korea
  • 9.4.6 Rest of Asia Pacific
• 9.5 Latin America
  • 9.5.1 Brazil
  • 9.5.2 Mexico
  • 9.5.3 Rest of Latin America
• 9.6 Middle East and Africa
  • 9.6.1 South Africa
  • 9.6.2 Saudi Arabia
  • 9.6.3 UAE
  • 9.6.4 Rest of Middle East and Africa

Chapter 10 Company Profiles

• 10.1 American Physical Therapy Association
• 10.2 Astellas Pharma
• 10.3 Beijing Jinlan Gene Technology Co., Ltd.
• 10.4 Biogen Inc.
• 10.5 Boston's Children Hospital
• 10.6 Children's Hospital of The King's Daughters
• 10.7 F. Hoffmann-La Roche Ltd.
• 10.8 Hanugen Therapeutics
• 10.9 Nationwide Children's Hospital
• 10.10 NMD Pharma A/S
• 10.11 Novartis AG
• 10.12 Pfizer, Inc.
• 10.13 Sanofi (Genzyme Corporation)
• 10.14 Scholar Rock, Inc