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運動神經元疾病治療的全球市場:洞察，競爭情形，市場預測:2032年

Motor Neuron Disease Treatment - Market Insights, Competitive Landscape, and Market Forecast - 2032
出版日期: | 出版商: DelveInsight | 英文 150 Pages | 商品交期: 2-10個工作天內

價格

預計在2025年至2032年的預測期內，運動神經元疾病 (MND) 治療市場將以5.87%的複合年增長率成長。由於肌萎縮側索硬化症 (ALS) 和脊髓性肌肉萎縮症 (SMA) 等運動神經元疾病的盛行率不斷上升，對運動神經元疾病 (MND) 治療的需求正在顯著增長。人們越來越意識到早期和準確診斷的重要性，這進一步推動了市場發展勢頭。此外，生物療法的顯著轉變，提供了更有針對性和更有效的治療選擇，正在改變治療格局。再加上研發投入的不斷增加以及 MND 治療領域的持續技術創新，為市場擴張創造了極為有利的環境。綜合來看，這些因素將使運動神經元疾病 (MND) 治療市場在 2025 年至 2032 年的預測期內保持強勁持續的成長。

運動神經元疾病治療市場動態：

根據 Taylor & Francis Online 發布的最新數據（2025 年），全球肌萎縮側索硬化症 (ALS) 的負擔預計將穩定增加。預計2022年報告的ALS病例約為32,893例，到2030年將成長10%以上，達到36,308例。這一上升趨勢不僅反映了盛行率的上升，也凸顯了診斷能力的提高和疾病意識的增強。

DelveInsight的2023年市場分析顯示，在美國、英國、德國、法國、義大利、西班牙和日本等七個主要市場，ALS的盛行率約為67,000例，這佐證了這一點。這表明，在現有的醫療保健體系中，對有效的治療幹預措施的需求巨大。根據澳洲運動神經元疾病協會 (MND Australia) 2024 年的數據，每天至少有兩人被診斷出患有運動神經元疾病 (MND)，目前約有 2,688 人患有該疾病，這凸顯了對可及治療策略的迫切需求。

就脊髓性肌肉萎縮症 (SMA) 而言，這是一種罕見但嚴重的遺傳性神經肌肉疾病。脊髓性肌肉萎縮症基金會 (SMA) 2023 年估計，目前美國有 10,000 至 25,000 人受其影響。此病的出生盛行率為 1/6,000 至 1/10,000，凸顯了早期篩檢和介入的重要性。基因療法，特別是使用腺相關病毒載體進行的SMN1基因替換，已成為突破性的解決方案，顯著改善了患者的長期療效，並在某些情況下恢復了運動功能。

總而言之，運動神經元疾病（尤其是ALS和SMA）的盛行率上升，是全球運動神經元疾病（MND）治療市場快速擴張的主要驅動力。這些以肌肉無力、呼吸功能障礙以及最終的運動技能喪失為特徵的進行性神經系統疾病，日益被視為嚴重的公共衛生問題。因此，對創新疾病改良療法的需求日益增長。

在治療方面，MND治療的典範轉移正在進行中。對於ALS，治療重點已從緩解症狀擴展到開發旨在減緩神經元退化進展的神經保護劑。對於SMA，基因療法正在重新定義治療標準，為治癒疾病提供了可能性，並改變了許多患者的疾病軌跡。

同時，監管環境對技術創新的支持力道也不斷增加。美國食品藥物管理局 (FDA) 和歐洲藥品管理局 (EMA) 近期的批准增強了市場信心。尤其是 Zolgensma® (onasemnogene abeparvovec) 的獲批，驗證了基因療法的有效性，並加速了針對運動神經元保存和肌肉增強的下一代療法的投資。

市場開發公司也在加強研發力度，以將先進的標靶療法商業化。例如，2023 年 4 月，Biogen Inc. 獲得 FDA 加速批准，其鞘內注射 QALSODY™（通用名：tofersen）用於治療已確診超氧化物歧化酶 1 (SOD1) 基因突變的成人肌萎縮側索硬化症 (ALS)。這項決定是基於血漿神經絲輕鏈 (NfL) 水平的降低，神經絲輕鏈 (NfL) 是一種與神經元損傷相關的生物標誌物，這標誌著生物標記驅動的治療發展邁出了重要一步。

總而言之，ALS 和 SMA 的盛行率和認知度不斷提高，加上治療創新的進步和有利的監管環境，正在創造一個強勁而充滿活力的運動神經元疾病治療市場。這些發展為該領域在 2025 年至 2032 年的預測期內實現持續成長和持續的科學突破奠定了基礎。

然而，高昂的治療費用以及監管機構和政府的嚴格指導方針是限制運動神經元疾病治療市場成長的主要限制因素。

運動神經元疾病治療市場區隔分析：

按藥物類別劃分的運動神經元疾病治療市場中，預計存活運動神經元 (SMN) 激動劑類別將在 2024 年佔據最大市場佔有率。此類別的成長主要得益於脊髓性肌肉萎縮症 (SMA) 盛行率的上升以及 SMN1 標靶療法帶來的誘人療效。 SMN 蛋白對於運動神經元的存活和功能至關重要，而運動神經元對於隨意肌肉運動至關重要。 SMN 激動劑透過增強 SMN 蛋白的生成發揮作用，幫助維持運動功能、減緩病情進展並改善 SMA 患者的整體生活品質。

例如，根據英國國家醫療服務體系 (NHS England) (2023) 的數據，英國每年約有 70 名 SMA 兒童出生。同樣，澳洲人類遺傳學會 (2024) 報告稱，每 10,000 人中就有 1 人出生時患有 SMA，每 40 人中就有 1 人是相關基因突變的健康攜帶者，這凸顯了 SMA 的高風險人群以及對有效幹預措施的迫切需求。

SMN 標靶療法的一個顯著優勢是其能夠提供跨類型療效，從而為不同 SMA 亞型（從嚴重的 1 型到較輕的 3 型）帶來臨床益處。例如，被診斷為最嚴重 SMA（1 型）的嬰兒在運動功能方面表現出顯著改善，達到了無需輔助即可坐起等發育里程碑，這在未經治療的病例中很少見。

除了改善運動功能外，口服 SMN 激動劑療法還具有改善可及性和依從性的優勢，尤其是在兒科患者中。這使得長期治療管理更加可行且對患者更友好，有助於提高依從性並改善療效。透過提高SMN蛋白水平，SMN激動劑有助於減緩SMA的進展，緩解相關的神經肌肉症狀，並延長生存期，尤其是在早發重症病例中。

不斷增加的研發投入和監管發展進一步推動了這一類別的成長。主要的行業參與者正在積極開發下一代SMN標靶療法，以提高療效和便利性。一個顯著的例子是羅氏公司於2025年2月宣布，美國食品藥物管理局(FDA)已批准Evrysdi® (risdiplam)片劑的新藥申請(NDA)。 Evrysdi是一種SMN2前mRNA剪接修飾劑，旨在治療SMA，該疾病是由5q染色體基因突變導致SMN蛋白缺失引起的。該片劑是一種口服全身性治療藥物，可在家服用，減少了住院輸液的需求，使患者和家屬更容易獲得治療。

這些因素預計將推動細分市場的發展，並最終推動全球運動神經元疾病治療市場的發展。

預計到2024年，北美將佔據全球運動神經元疾病治療市場的最大佔有率，這得益於其關鍵的結構性和戰略優勢。該地區的主導地位主要源於脊髓性肌肉萎縮症 (SMA) 和肌萎縮側索硬化症 (ALS) 等運動神經元疾病的盛行率不斷上升，這持續增加了醫療保健系統對有效治療方案的需求。此外，該地區擁有強勁的製藥環境，大型生物技術和製藥公司積極開發、臨床試驗和商業化創新療法。新產品審批速度維持較高水平，並得益於良好的監管架構。特別是，美國食品藥物管理局 (FDA) 等機構已推出罕見疾病藥物加速審批流程，支持快速提供尖端的MND治療方案。加重的疾病負擔、強大的研發活動以及積極的監管支持等因素共同鞏固了北美在運動神經元疾病治療市場的全球領先地位。

根據美國疾病管制與預防中心 (CDC, 2025) 的最新數據，2022 年美國將記錄約 33,000 例肌萎縮側索硬化症 (ALS) 病例，預計到 2030 年這一數字將增加 10% 以上，超過 36,000 例。同樣，美國醫學會 (2024) 報告稱，2024 年美國每 14,694 名新生兒中就有 1 人將被診斷患有脊髓性肌肉萎縮症 (SMA)，這凸顯了北美運動神經元疾病 (MND) 負擔的日益加重。

針對運動神經元的療法越來越多地被用來對抗這些漸進性且往往致命的疾病。具體而言，治療著重於替換缺失或缺陷的SMN1基因，提供功能性拷貝，以維護運動神經元健康並減緩肌肉退化。美國國立衛生研究院（2022年）指出，據估計，加拿大每年每10,000名新生兒中約有6,000至1人患有SMA，這支持了這一趨勢，凸顯了該地區對及時診斷和高級幹預的需求。

SMA盛行率的上升是北美運動神經元疾病治療市場成長的主要驅動力，從而導致對疾病修飾療法的需求增加。由於SMA通常發生在嬰兒期和幼兒期，因此早期基因篩檢和創新療法（例如針對SMN的基因療法）的需求日益迫切，從而刺激了研發投資和治療方法的開發。

推動該地區市場擴張的一個主要因素是嚴格的監管環境。美國食品藥物管理局 (FDA) 正在透過為有前景的藥物提供快速通道和突破性療法認定，加速運動神經元疾病 (MND) 治療的進展。值得注意的是，用於脊髓性肌肉萎縮症 (SMA) 的基因療法 Zolgensma® 獲得了加速審批，這標誌著疾病修飾治療取得了突破，並進一步驗證了基於基因的治療方法。

除了監管支援外，美國主要製藥公司的持續創新也增強了市場發展動能。例如，2023 年 4 月，百健公司 (Biogen Inc.) 宣布其鞘內注射 QALSODY™（通用名：tofersen）已獲得 FDA 加速審批，用於治療已確診 SOD1 基因突變的肌萎縮側索硬化症 (ALS) 患者。此審批是基於血漿中 IL-16（一種與神經元損傷相關的生物標記）的檢測結果。QALSODY 作為一種新型標靶療法在美國市場上銷售，該藥物能夠降低運動神經元疾病患者的神經絲輕鏈 (NfL) 水平。

不斷上升的疾病盛行率、持續的產品創新、監管支持以及行業領導者的策略性舉措，都是推動北美運動神經元疾病治療市場在 2025 年至 2032 年預測期內強勁增長的因素。

運動神經元疾病治療市場的主要參與者：

運動神經元疾病治療市場的主要參與者包括 Biogen、三菱化學集團株式會社、羅氏製藥有限公司、大塚控股株式會社、賽諾菲、Ionis Pharmaceuticals, Inc.、諾華公司、EDW Pharmace Inc.、PTC Therapeutics, Inc.、中外株式會社和 AMYLYXceticals。

運動神經元疾病治療市場的最新進展：

  • 2025年6月，羅氏公司宣布，歐盟委員會 (EC) 批准延長 Evrysdi® (risdiplam) 的說明書，為脊髓性肌肉萎縮症 (SMA) 患者添加一種新的常溫片劑型。這項進展提高了治療的便利性和可近性，尤其對於需要靈活居家給藥的患者而言。
  • 2025年1月，百健公司 (Biogen Inc.) 宣布，美國食品藥物管理局 (FDA) 已受理 Nusinersen 高劑量製劑的監管申請，歐洲藥品管理局 (EMA) 也已批准該申請。這項進展標誌著透過優化劑量以改善脊髓性肌肉萎縮症 (SMA) 患者的臨床療效，朝著潛在增強 Nusinersen 治療效果邁出了重要一步。
  • 2022年9月，Amylyx Pharmaceuticals, Inc. 宣布，美國食品藥物管理局 (FDA) 批准 RELYVRIO™（苯丁酸鈉和牛磺酸二醇）用於治療肌萎縮側索硬化症 (ALS) 成年患者。此批准標誌著一個重要的里程碑，為患者提供了一種旨在減緩病情進展和保留神經功能的全新治療選擇。

運動神經元疾病治療市場研究報告的主要發現

  • 目前運動神經元疾病治療市場規模分析（2024 年）及八年市場預測（2025-2032 年）
  • 過去三年主要產品/技術發展、合併、收購、合作及合資企業
  • 主導運動神經元疾病治療市場的主要公司
  • 運動神經元疾病治療市場中競爭對手面臨的各種機會
  • 2024 年和 2025 年的頂級細分市場以及這些細分市場在 2032 年的表現
  • 目前運動神經元疾病治療市場中表現最佳的地區和國家
  • 未來公司應關注運動神經元疾病治療市場成長機會的地區和國家

關於運動神經元疾病治療的常見問題市場

1. 運動神經元疾病治療

  • 運動神經元疾病 (MND) 治療是指使用藥物介入措施來減緩疾病進展和控制症狀。核心療法旨在延長患者存活期並緩解功能衰退。此外，支持治療針對肌肉僵硬、痙攣和疼痛等繼發症狀，提高患者舒適度和整體生活質量，從而改善護理效果並提升醫療價值。

    2. 運動神經元疾病治療市場

    • 預計在 2025 年至 2032 年的預測期內，運動神經元疾病治療市場將以 5.87% 的複合年增長率成長。

    3.運動神經元疾病治療市場的驅動因素

    • 運動神經元疾病治療的需求受到多種關鍵因素的驅動，包括早產數量增加及其相關併發症，例如低體溫、低血糖、呼吸窘迫和代謝性酸中毒，以及感染和其他新生兒疾病風險的增加。此外，先天性畸形盛行率的上升，以及新興市場主要公司持續的產品開發和上市活動，也進一步推動了市場的成長。這些因素共同為持續的市場擴張創造了有利環境，預計運動神經元疾病治療市場將在2025年至2032年期間實現強勁穩定的成長。

    4.運動神經元疾病治療市場的主要公司

    • 運動神經元疾病治療市場的主要參與者包括百健（Biogen）、三菱化學集團株式會社、羅氏製藥有限公司、大塚控股株式會社、賽諾菲、Ionis Pharmaceuticals, Inc.、諾華公司、EDW Pharma, Inc.、PTC Therapeutics, Inc.、中外製藥株式會合AMYs。

    5. 運動神經元疾病治療市場最大佔有率的地區

    • 得益於關鍵的結構性和策略優勢，預計到2024年，北美將佔據全球運動神經元疾病治療市場的最大佔有率。該地區的主導地位主要源於脊髓性肌肉萎縮症 (SMA) 和肌萎縮側索硬化症 (ALS) 等運動神經元疾病的盛行率不斷上升，這持續增加了醫療保健系統對有效治療方案的需求。此外，該地區還受益於強勁的醫藥市場格局，大型生物技術和製藥公司正在積極開發、臨床測試和商業化創新療法。新產品審批速度保持較高水平，並得到有利的監管框架的進一步支持。尤其是美國食品藥物管理局 (FDA) 等機構已推出罕見疾病藥物加速審批途徑，支持快速提供尖端的 MND 治療方案。加重的疾病負擔、強勁的研發活動以及積極的監管支持等因素共同鞏固了北美作為運動神經元疾病治療市場全球領導者的地位。

    目錄

    第1章 運動神經元疾病治療市場報告的簡介

    第2章 運動神經元疾病治療市場摘要整理

    第3章 競爭情形

    第4章 法規分析

    • 美國
    • 歐洲
    • 日本
    • 中國

    第5章 運動神經元疾病治療市場主要原因分析

    • 運動神經元疾病治療市場的驅動因素
      • 運動神經元疾病盛行率不斷上升
      • 對早期準確診斷的需求和認知不斷提高
      • 加大運動神經元疾病的投資與創新運動神經元疾病藥物研發
    • 運動神經元疾病治療市場的限制與課題
      • 高昂的治療費用
      • 監管機構和政府機構的嚴格指導方針
    • 運動神經元疾病治療市場的機遇
      • 日益轉向個人化與標靶治療
      • 運動神經元疾病適應症的藥物組合不斷擴展

    第6章 運動神經元疾病治療市場上波特的五力分析

    第7章 運動神經元疾病治療市場評估

    • 各類藥物
      • NMDA（N-甲基-D-天門冬胺酸）受體拮抗劑
      • SOD1 突變激動劑
      • α-2 腎上腺素激動劑
      • 存活運動神經元 (SMN) 激動劑
      • 其他
    • 各給藥途徑
      • 口服
      • 非口服
    • 各適應症
      • 肌肉萎縮性脊髓側索硬化症(ALS)
      • 脊髓性肌萎縮症(SMA)
    • 各流通管道
      • 醫院及藥局
      • 線上藥局
    • 地區
      • 北美
      • 歐洲
      • 亞太地區
      • 其他地區

    第8章 運動神經元疾病治療市場企業與產品簡介

    • Biogen
    • Mitsubishi Chemical Group Corporation
    • F. Hoffmann-La Roche Ltd.
    • Otsuka Holdings Co., Ltd.
    • Sanofi
    • Ionis Pharmaceuticals, Inc.
    • Novartis AG
    • EW Pharma, Inc.
    • PTC Therapeutics, Inc.
    • Chugai Pharmaceutical Co., Ltd.
    • AMYLYX Pharmaceuticals

    第9章 KOL的見解

    第10章 計劃方法

    第11章 關於DelveInsight

    第12章 免責聲明和諮詢方式

Product Code: DIMDCL0873

Motor Neuron Disease Treatment Market by Drug Class (NMDA (N-methyl-D-aspartate) Receptor Antagonist, SOD1 Gene Mutation Agonist, Alpha-2 Adrenergic Agonist, Survival Motor Neuron (SMN) Agonist, and Others), Route of Administration (Oral and Parenteral), Indication (Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA), and Others), Distribution Channel (Hospitals and Retail Pharmacies and Online Pharmacies), and Geography (North America, Europe, Asia-Pacific, and Rest of the World) is expected to grow at a steady CAGR forecast till 2032 owing to the growing prevalence of motor neuron diseases and increasing investments and innovations in the research and development of MND drugs.

The motor neuron disease treatment market is estimated to grow at a CAGR of 5.87% during the forecast period from 2025 to 2032. The demand for motor neuron disease (MND) treatment is experiencing significant growth, driven by the rising prevalence of motor neuron disorders such as Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA), and others. Increasing awareness about the importance of early and accurate diagnosis is further accelerating market momentum. Moreover, a notable shift toward biological therapies which offer targeted and more effective treatment options is transforming the therapeutic landscape. This, combined with increasing investments and continuous innovation in the research and development of MND therapies, is fostering a highly favorable environment for market expansion. These factors collectively position the motor neuron disease treatment market for robust and sustained growth throughout the forecast period from 2025 to 2032.

Motor Neuron Disease Treatment Market Dynamics:

According to recent data published by Taylor & Francis Online (2025), the global burden of Amyotrophic Lateral Sclerosis (ALS) is projected to grow steadily. From approximately 32,893 reported cases in 2022, the number was expected to increase by over 10%, reaching an estimated 36,308 cases by 2030. This upward trend not only reflected a rise in incidence rates but also highlighted improvements in diagnostic capabilities and heightened disease awareness.

Supporting this, a 2023 market analysis by DelveInsight revealed that across the seven major markets including the United States, United Kingdom, Germany, France, Italy, Spain, and Japan, the prevalence of ALS stood at around 67,000 cases. This indicated a substantial demand for effective therapeutic interventions within well-established healthcare systems. Regionally, Australia mirrors this growing burden, data from MND Australia (2024), showed that at least two individuals were diagnosed with motor neuron disease (MND) each day, with approximately 2,688 people who were currently living with that condition, underscoring the pressing need for accessible treatment strategies.

In the case of Spinal Muscular Atrophy (SMA), a rare but severe genetic neuromuscular disorder, the Spinal Muscular Atrophy Foundation (2023) estimated that between 10,000 and 25,000 individuals were currently affected in the United States. The condition had a birth prevalence of approximately 1 in every 6,000 to 10,000 live births, emphasizing the critical importance of early screening and intervention. Gene therapy, particularly SMN1 gene replacement using adeno-associated viral vectors, has emerged as a transformative solution, significantly improving long-term outcomes and, in some instances, restoring motor function.

Collectively, the rising prevalence of motor neuron diseases particularly ALS and SMA is a key driver behind the rapid expansion of the global MND treatment market. These progressive neurological disorders, characterized by muscle weakness, respiratory compromise, and eventual loss of mobility, are increasingly recognized as serious public health concerns. As a result, there is mounting demand for innovative, disease-modifying therapies.

Therapeutically, the MND treatment landscape is undergoing a paradigm shift. In ALS, the focus is expanding beyond symptomatic relief to the development of neuroprotective agents designed to slow the progression of neuronal degeneration. In SMA, gene therapy has redefined the standard of care, offering potentially curative benefits and altering the disease trajectory for many patients.

At the same time, the regulatory environment is becoming more supportive of innovation. Recent approvals by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have bolstered confidence in the market. Notably, the approval of Zolgensma(R) (onasemnogene abeparvovec) has validated gene therapy approaches and accelerated investment in next-generation treatments that target motor neuron preservation and muscle function enhancement.

Market players are also intensifying their research and development efforts to commercialize advanced targeted therapies. For example, in April 2023, Biogen Inc. received FDA accelerated approval for QALSODY(TM) (tofersen), an intrathecal injection indicated for the treatment of ALS in adults with a confirmed mutation in the superoxide dismutase 1 (SOD1) gene. The decision was based on reductions in plasma neurofilament light chain (NfL) levels, a biomarker linked to neuronal damage, marking a significant step forward in biomarker-driven therapeutic development.

In conclusion, the growing incidence and improved recognition of ALS and SMA, coupled with advancements in therapeutic innovation and a favorable regulatory backdrop, are collectively shaping a robust and dynamic motor neuron disease treatment market. These developments position the sector for sustained growth and ongoing scientific breakthroughs throughout the forecast period from 2025 to 2032.

However, the high cost of the treatment and stringent guidelines by regulatory bodies and governments, among others are some of the key constraints that may limit the growth of the motor neuron disease treatment market.

Motor Neuron Disease Treatment Market Segment Analysis:

Motor Neuron Disease Treatment Market by Drug Class (NMDA (N-methyl-D-aspartate) Receptor Antagonist, SOD1 Gene Mutation Agonist, Alpha-2 Adrenergic Agonist, Survival Motor Neuron (SMN) Agonist, and Others), Route of Administration (Oral and Parenteral), Indication (Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA), and Others), Distribution Channel (Hospital and Retail Pharmacies and Online Pharmacies), and Geography (North America, Europe, Asia-Pacific, and Rest of the World)

In the drug class segment of the motor neuron disease treatment market, the survival motor neuron (SMN) agonist category is estimated to account for the largest market share in 2024. The growth of this category is primarily driven by the increasing prevalence of Spinal Muscular Atrophy (SMA) and the compelling therapeutic benefits associated with SMN1-targeted treatments. The SMN protein is critical for the survival and function of motor neurons, which are essential for voluntary muscle movement. SMN agonists work by enhancing the production of SMN protein, helping to preserve motor function, delay disease progression, and improve the overall quality of life for patients living with SMA.

For instance, according to NHS England (2023), approximately 70 children were born with SMA each year in the United Kingdom. Similarly, the Human Genetics Society of Australia (2024) reported that 1 in 10,000 individuals were born with SMA, while 1 in 40 people were healthy carriers of the associated genetic mutation highlighting the significant at-risk population and the urgent need for effective interventions.

One notable advantage of SMN-targeted therapies is their ability to provide cross-type efficacy, offering clinical benefits across different forms of SMA, from severe Type 1 to milder Type 3. For example, infants diagnosed with Type 1 SMA typically the most severe form have demonstrated remarkable improvements in motor function, including reaching developmental milestones such as sitting unassisted, an outcome that is rare in untreated cases.

In addition to motor function improvements, SMN agonist therapies offer the benefit of oral formulations, enhancing treatment accessibility and compliance, especially among pediatric patients. This makes long-term treatment management more feasible and patient-friendly, contributing to better adherence and improved outcomes. By boosting SMN protein levels, SMN agonists help slow the progression of SMA, mitigate its associated neuromuscular symptoms, and prolong survival, particularly in early-onset and severe cases.

Further fueling this category's growth is the increase in R&D investments and regulatory advancements. Key industry players are actively developing next-generation SMN-targeted treatments to improve efficacy and convenience. A notable example is from February 2025, when Roche announced that the U.S. Food and Drug Administration (FDA) approved a New Drug Application (NDA) for a tablet formulation of Evrysdi(R) (risdiplam). Evrysdi is an SMN2 pre-mRNA splicing modifier, designed to treat SMA caused by mutations in the chromosome 5q gene, which result in a deficiency of the SMN protein. This tablet formulation offers an oral, systemic treatment alternative that can be taken at home, reducing the need for hospital-based infusions and making care more accessible for patients and families.

Thus, the factors mentioned above are likely to boost the market segment and thereby increase the overall market of motor neuron disease treatment across the globe.

North America is expected to dominate the overall motor neuron disease treatment market:

North America is expected to hold the largest share of the global motor neuron disease treatment market in 2024, driven by a combination of key structural and strategic advantages. This regional dominance is primarily attributed to the rising prevalence of motor neuron disorders such as Spinal Muscular Atrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS), which continue to place increasing demand on healthcare systems for effective treatment solutions. Additionally, the region benefits from a robust pharmaceutical landscape, with leading biotech and pharmaceutical companies actively engaged in the development, clinical trials, and commercialization of innovative therapies. The pace of new product approvals remains high, further supported by favorable regulatory frameworks. Notably, agencies such as the U.S. Food and Drug Administration (FDA) have implemented accelerated approval pathways for rare disease drugs, helping to fast-track the availability of cutting-edge treatments for MNDs. Collectively, these factors such as the rising disease burden, strong R&D activity, and proactive regulatory support are solidifying North America's position as the global leader in the motor neuron disease treatment market.

According to recent data from the Centers for Disease Control and Prevention (CDC, 2025), approximately 33,000 cases of Amyotrophic Lateral Sclerosis (ALS) were recorded in the U.S. in 2022, with the number projected to rise by over 10% to exceed 36,000 cases by 2030. Similarly, the American Medical Association (2024) reported that 1 in every 14,694 newborns in the U.S. was diagnosed with Spinal Muscular Atrophy (SMA) in 2024, underscoring the growing burden of motor neuron diseases (MNDs) in North America.

To combat these progressive and often fatal disorders, motor neuron-targeted therapies are increasingly being adopted, particularly those focused on replacing defective or missing SMN1 genes, thereby providing functional copies to preserve motor neuron health and slow muscular degeneration. Supporting this trend, the National Institutes of Health (2022), noted that in Canada, between 1 in 6,000 to 10,000 babies were born with SMA annually, highlighting a regional need for timely diagnosis and advanced interventions.

The rising incidence of SMA is a key driver of growth in the North American motor neuron disease treatment market, as it has led to increased demand for disease-modifying therapies. Given that SMA often manifests in infancy or early childhood, there is a heightened urgency for early genetic screening and innovative treatments such as SMN-targeted gene therapy, which has spurred substantial R&D investment and therapeutic development.

A significant enabler of market expansion in the region is the proactive regulatory environment. The U.S. Food and Drug Administration (FDA) has accelerated progress in the MND treatment landscape by offering fast-track and breakthrough therapy designations for promising drugs. Notably, Zolgensma(R), a gene therapy for SMA, received accelerated approval, marking a breakthrough in disease-modifying treatment and further validating gene-based approaches.

In addition to regulatory support, ongoing innovation by leading U.S.-based pharmaceutical companies is reinforcing market momentum. For example, in April 2023, Biogen Inc. announced that the FDA had granted accelerated approval for QALSODY(TM) (tofersen), an intrathecal injection indicated for ALS patients with a confirmed SOD1 gene mutation. The approval was based on the drug's ability to reduce plasma neurofilament light chain (NfL) levels, a biomarker linked to neuronal damage, making QALSODY available in the U.S. market as a new targeted treatment option.

Collectively, the combination of increasing disease prevalence, continuous product innovation, regulatory support, and strategic initiatives by major industry players is expected to drive robust growth in the North American motor neuron disease treatment market throughout the forecast period from 2025 to 2032.

Motor Neuron Disease Treatment Market Key Players:

Some of the key market players operating in the motor neuron disease treatment market include Biogen, Mitsubishi Chemical Group Corporation, F. Hoffmann-La Roche Ltd., Otsuka Holdings Co., Ltd., Sanofi, Ionis Pharmaceuticals, Inc., Novartis AG, EDW Pharma, Inc., PTC Therapeutics, Inc., Chugai Pharmaceutical Co., Ltd., AMYLYX Pharmaceuticals, and others.

Recent Developmental Activities in the Motor Neuron Disease Treatment Market:

  • In June 2025, Roche announced that the European Commission (EC) had approved a label extension for Evrysdi(R) (risdiplam), allowing the inclusion of a new room-temperature stable tablet formulation for individuals living with Spinal Muscular Atrophy (SMA). This advancement enhances treatment convenience and accessibility, particularly for patients who require flexible, at-home administration.
  • In January 2025, Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) had accepted the company's supplemental New Drug Application (sNDA), and the European Medicines Agency (EMA) had validated its application for a higher dose regimen of nusinersen for the treatment of spinal muscular atrophy (SMA). This development marks an important step toward potentially enhancing the therapeutic benefits of nusinersen by optimizing dosing for improved clinical outcomes in SMA patient.
  • In September 2022, Amylyx Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) had approved RELYVRIO(TM) (sodium phenylbutyrate and taurursodiol) for the treatment of adults with Amyotrophic Lateral Sclerosis (ALS). This approval marked a significant milestone, offering patients a new therapeutic option aimed at slowing disease progression and preserving neurological function.

Key takeaways from the motor neuron disease treatment market report study

  • Market size analysis for current motor neuron disease treatment market size (2024), and market forecast for 8 years (2025 to 2032)
  • Top key product/technology developments, mergers, acquisitions, partnerships, and joint ventures happened over the last 3 years.
  • Key companies dominating the motor neuron disease treatment market.
  • Various opportunities available for the other competitors in the motor neuron disease treatment market space.
  • What are the top-performing segments in 2024? How these segments will perform in 2032?
  • Which are the top-performing regions and countries in the current motor neuron disease treatment market scenario?
  • Which are the regions and countries where companies should have concentrated on opportunities for motor neuron disease treatment market growth in the coming future?

Target audience who can benefit from this motor neuron disease treatment market report study

  • Motor neuron disease treatment product providers
  • Research organizations and consulting companies
  • Motor neuron disease treatment-related organizations, associations, forums, and other alliances.
  • Government and corporate offices
  • Start-up companies, venture capitalists, and private equity firms
  • Distributors and traders dealing in motor neuron disease treatment
  • Various end-users who want to know more about the motor neuron disease treatment market and the latest technological developments in the motor neuron disease treatment market.

Frequently Asked Questions for the Motor Neuron Disease Treatment Market:

1. What are motor neuron disease treatments?

  • Motor Neuron Disease (MND) Treatment refers to the use of pharmaceutical interventions aimed at slowing disease progression and managing symptoms. Core treatments are designed to potentially extend patient survival and mitigate functional decline. Additionally, supportive medications target secondary symptoms, such as muscle stiffness, cramps, and pain, to enhance patient comfort and overall quality of life, contributing to improved care outcomes and healthcare value delivery.

2. What is the market for motor neuron disease treatment?

  • The motor neuron disease treatment market is estimated to grow at a CAGR of 5.87% during the forecast period from 2025 to 2032.

3. What are the drivers for the motor neuron disease treatment market?

  • The demand for motor neuron disease treatment is being driven by several critical factors, including the rising incidence of neonatal disorders such as hypothermia and related complications like hypoglycemia, respiratory distress, metabolic acidosis, and increased risk of infections, along with the growing number of preterm births. Additionally, the increasing prevalence of congenital anomalies and ongoing product development and launch activities by leading market players are further fueling growth. Collectively, these factors are creating a supportive environment for sustained market expansion, positioning the motor neuron disease treatment market for robust and steady growth from 2025 to 2032.

4. Who are the key players operating in the motor neuron disease treatment market?

  • Some of the key market players operating in the motor neuron disease treatment market include Biogen, Mitsubishi Chemical Group Corporation, F. Hoffmann-La Roche Ltd., Otsuka Holdings Co., Ltd., Sanofi, Ionis Pharmaceuticals, Inc., Novartis AG, EDW Pharma, Inc., PTC Therapeutics, Inc., Chugai Pharmaceutical Co., Ltd., AMYLYX Pharmaceuticals, and others.

5. Which region has the highest share in the motor neuron disease treatment market?

  • North America is expected to hold the largest share of the global motor neuron disease treatment market in 2024, driven by a combination of key structural and strategic advantages. This regional dominance is primarily attributed to the rising prevalence of motor neuron disorders such as Spinal Muscular Atrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS), which continue to place increasing demand on healthcare systems for effective treatment solutions. Additionally, the region benefits from a robust pharmaceutical landscape, with leading biotech and pharmaceutical companies actively engaged in the development, clinical trials, and commercialization of innovative therapies. The pace of new product approvals remains high, further supported by favorable regulatory frameworks. Notably, agencies such as the U.S. Food and Drug Administration (FDA) have implemented accelerated approval pathways for rare disease drugs, helping to fast-track the availability of cutting-edge treatments for MNDs. Collectively, these factors such as the rising disease burden, strong R&D activity, and proactive regulatory support are solidifying North America's position as the global leader in the motor neuron disease treatment market.

Table of Contents

1. Motor Neuron Disease Treatment Market Report Introduction

  • 1.1. Scope of the Study
  • 1.2. Market Segmentation
  • 1.3. Market Assumption

2. Motor Neuron Disease Treatment Market Executive Summary

  • 2.1. Market at Glance

3. Competitive Landscape

4. Regulatory Analysis

  • 4.1. The United States
  • 4.2. Europe
  • 4.3. Japan
  • 4.4. China

5. Motor Neuron Disease Treatment Market Key Factors Analysis

  • 5.1. Motor Neuron Disease Treatment Market Drivers
    • 5.1.1. The growing prevalence of motor neuron diseases
    • 5.1.2. Increasing demand and awareness about early and accurate diagnosis
    • 5.1.3. Increasing investments and innovations in the research and development of MND drugs
  • 5.2. Motor Neuron Disease Treatment Market Restraints and Challenges
    • 5.2.1. High cost of the treatment
    • 5.2.2. Stringent guidelines by regulatory bodies and government agencies
  • 5.3. Motor Neuron Disease Treatment Market Opportunity
    • 5.3.1. Growing shift towards personalized and targeted therapies
    • 5.3.2. Presence of an extensive drug portfolio in the pipeline for motor neuron indications

6. Motor Neuron Disease Treatment Market Porter's Five Forces Analysis

  • 6.1. Bargaining Power of Suppliers
  • 6.2. Bargaining Power of Consumers
  • 6.3. Threat of New Entrants
  • 6.4. Threat of Substitutes
  • 6.5. Competitive Rivalry

7. Motor Neuron Disease Treatment Market Assessment

  • 7.1. By Drug Class
    • 7.1.1. NMDA (N-methyl-D-aspartate) Receptor Antagonist
    • 7.1.2. SOD1 Gene Mutation Agonist
    • 7.1.3. Alpha-2 Adrenergic Agonist
    • 7.1.4. Survival Motor Neuron (SMN) Agonist
    • 7.1.5. Others
  • 7.2. By Route of Administration
    • 7.2.1. Oral
    • 7.2.2. Parenteral
  • 7.3. By Indication
    • 7.3.1. Amyotrophic Lateral Sclerosis (ALS)
    • 7.3.2. Spinal Muscular Atrophy (SMA)
    • 7.3.3. Others
  • 7.4. By Distribution Channel
    • 7.4.1. Hospital & Retail Pharmacies
    • 7.4.2. Online Pharmacies
  • 7.5. By Geography
    • 7.5.1. North America
      • 7.5.1.1. United States Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.1.2. Canada Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.1.3. Mexico Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
    • 7.5.2. Europe
      • 7.5.2.1. France Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.2.2. Germany Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.2.3. United Kingdom Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.2.4. Italy Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.2.5. Spain Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.2.6. Rest of Europe Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
    • 7.5.3. Asia-Pacific
      • 7.5.3.1. China Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.3.2. Japan Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.3.3. India Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.3.4. Australia Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.3.5. South Korea Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.3.6. Rest of Asia-Pacific Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
    • 7.5.4. Rest of the World (RoW)
      • 7.5.4.1. Middle East Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.4.2. Africa Motor Neuron Disease Treatment Market Size in USD million (2022-2032)
      • 7.5.4.3. South America Motor Neuron Disease Treatment Market Size In USD Million (2022-2032)

8. Motor Neuron Disease Treatment Market Company and Product Profiles

  • 8.1. Biogen
    • 8.1.1. Company Overview
    • 8.1.2. Company Snapshot
    • 8.1.3. Financial Overview
    • 8.1.4. Product Listing
    • 8.1.5. Entropy
  • 8.2. Mitsubishi Chemical Group Corporation
    • 8.2.1. Company Overview
    • 8.2.2. Company Snapshot
    • 8.2.3. Financial Overview
    • 8.2.4. Product Listing
    • 8.2.5. Entropy
  • 8.3. F. Hoffmann-La Roche Ltd.
    • 8.3.1. Company Overview
    • 8.3.2. Company Snapshot
    • 8.3.3. Financial Overview
    • 8.3.4. Product Listing
    • 8.3.5. Entropy
  • 8.4. Otsuka Holdings Co., Ltd.
    • 8.4.1. Company Overview
    • 8.4.2. Company Snapshot
    • 8.4.3. Financial Overview
    • 8.4.4. Product Listing
    • 8.4.5. Entropy
  • 8.5. Sanofi
    • 8.5.1. Company Overview
    • 8.5.2. Company Snapshot
    • 8.5.3. Financial Overview
    • 8.5.4. Product Listing
    • 8.5.5. Entropy
  • 8.6. Ionis Pharmaceuticals, Inc.
    • 8.6.1. Company Overview
    • 8.6.2. Company Snapshot
    • 8.6.3. Financial Overview
    • 8.6.4. Product Listing
    • 8.6.5. Entropy
  • 8.7. Novartis AG
    • 8.7.1. Company Overview
    • 8.7.2. Company Snapshot
    • 8.7.3. Financial Overview
    • 8.7.4. Product Listing
    • 8.7.5. Entropy
  • 8.8. EW Pharma, Inc.
    • 8.8.1. Company Overview
    • 8.8.2. Company Snapshot
    • 8.8.3. Financial Overview
    • 8.8.4. Product Listing
    • 8.8.5. Entropy
  • 8.9. PTC Therapeutics, Inc.
    • 8.9.1. Company Overview
    • 8.9.2. Company Snapshot
    • 8.9.3. Financial Overview
    • 8.9.4. Product Listing
    • 8.9.5. Entropy
  • 8.10. Chugai Pharmaceutical Co., Ltd.
    • 8.10.1. Company Overview
    • 8.10.2. Company Snapshot
    • 8.10.3. Financial Overview
    • 8.10.4. Product Listing
    • 8.10.5. Entropy
  • 8.11. AMYLYX Pharmaceuticals
    • 8.11.1. Company Overview
    • 8.11.2. Company Snapshot
    • 8.11.3. Financial Overview
    • 8.11.4. Product Listing
    • 8.11.5. Entropy

9. KOL Views

10. Project Approach

11. About DelveInsight

12. Disclaimer & Contact Us

List of Tables

  • Table 1: Competitive Analysis
  • Table 2: Motor Neuron Disease Treatment Market in Global (2022-2032)
  • Table 3: Motor Neuron Disease Treatment Market in Global by Drug Class (2022-2032)
  • Table 4: Motor Neuron Disease Treatment Market in Global by Route of Administration (2022-2032)
  • Table 5: Motor Neuron Disease Treatment Market in Global by Indication (2022-2032)
  • Table 6: Motor Neuron Disease Treatment Market in Global by Distribution Channel (2022-2032)
  • Table 7: Motor Neuron Disease Treatment Market in Global by Geography (2022-2032)
  • Table 8: Motor Neuron Disease Treatment Market in North America (2022-2032)
  • Table 9: Motor Neuron Disease Treatment Market in the United States (2022-2032)
  • Table 10: Motor Neuron Disease Treatment Market in Canada (2022-2032)
  • Table 11: Motor Neuron Disease Treatment Market in Mexico (2022-2032)
  • Table 12: Motor Neuron Disease Treatment Market in Europe (2022-2032)
  • Table 13: Motor Neuron Disease Treatment Market in France (2022-2032)
  • Table 14: Motor Neuron Disease Treatment Market in Germany (2022-2032)
  • Table 15: Motor Neuron Disease Treatment Market in United Kingdom (2022-2032)
  • Table 16: Motor Neuron Disease Treatment Market in Italy (2022-2032)
  • Table 17: Motor Neuron Disease Treatment Market in Spain (2022-2032)
  • Table 18: Motor Neuron Disease Treatment Market in the Rest of Europe (2022-2032)
  • Table 19: Motor Neuron Disease Treatment Market in Asia-Pacific (2022-2032)
  • Table 20: Motor Neuron Disease Treatment Market in China (2022-2032)
  • Table 21: Motor Neuron Disease Treatment Market in Japan (2022-2032)
  • Table 22: Motor Neuron Disease Treatment Market in India (2022-2032)
  • Table 23: Motor Neuron Disease Treatment Market in Australia (2022-2032)
  • Table 24: Motor Neuron Disease Treatment Market in South Korea (2022-2032)
  • Table 25: Motor Neuron Disease Treatment Market in Rest of Asia-Pacific (2022-2032)
  • Table 26: Motor Neuron Disease Treatment Market in the Rest of the World (2022-2032)
  • Table 27: Motor Neuron Disease Treatment Market in the Middle East (2022-2032)
  • Table 28: Motor Neuron Disease Treatment Market in Africa (2022-2032)
  • Table 29: Motor Neuron Disease Treatment Market in South America (2022-2032)

List of Figures

  • Figure 1: Competitive Analysis
  • Figure 2: Motor Neuron Disease Treatment Market in Global (2022-2032)
  • Figure 3: Motor Neuron Disease Treatment Market in Global by Drug Class (2022-2032)
  • Figure 4: Motor Neuron Disease Treatment Market in Global by Route of Administration (2022-2032)
  • Figure 5: Motor Neuron Disease Treatment Market in Global by Indication (2022-2032)
  • Figure 6: Motor Neuron Disease Treatment Market in Global by Distribution Channel (2022-2032)
  • Figure 7: Motor Neuron Disease Treatment Market in Global by Geography (2022-2032)
  • Figure 8: Motor Neuron Disease Treatment Market in North America (2022-2032)
  • Figure 9: Motor Neuron Disease Treatment Market in the United States (2022-2032)
  • Figure 10: Motor Neuron Disease Treatment Market in Canada (2022-2032)
  • Figure 11: Motor Neuron Disease Treatment Market in Mexico (2022-2032)
  • Figure 12: Motor Neuron Disease Treatment Market in Europe (2022-2032)
  • Figure 13: Motor Neuron Disease Treatment Market in France (2022-2032)
  • Figure 14: Motor Neuron Disease Treatment Market in Germany (2022-2032)
  • Figure 15: Motor Neuron Disease Treatment Market in United Kingdom (2022-2032)
  • Figure 16: Motor Neuron Disease Treatment Market in Italy (2022-2032)
  • Figure 17: Motor Neuron Disease Treatment Market in Spain (2022-2032)
  • Figure 18: Motor Neuron Disease Treatment Market in the Rest of Europe (2022-2032)
  • Figure 19: Motor Neuron Disease Treatment Market in Asia-Pacific (2022-2032)
  • Figure 20: Motor Neuron Disease Treatment Market in China (2022-2032)
  • Figure 21: Motor Neuron Disease Treatment Market in Japan (2022-2032)
  • Figure 22: Motor Neuron Disease Treatment Market in India (2022-2032)
  • Figure 23: Motor Neuron Disease Treatment Market in Australia (2022-2032)
  • Figure 24: Motor Neuron Disease Treatment Market in South Korea (2022-2032)
  • Figure 25: Motor Neuron Disease Treatment Market in Rest of Asia-Pacific (2022-2032)
  • Figure 26: Motor Neuron Disease Treatment Market in the Rest of the World (2022-2032)
  • Figure 27: Motor Neuron Disease Treatment Market in the Middle East (2022-2032)
  • Figure 28: Motor Neuron Disease Treatment Market in Africa (2022-2032)
  • Figure 29: Motor Neuron Disease Treatment Market in South America (2022-2032)
  • Figure 30: Market Drivers
  • Figure 31: Market Barriers
  • Figure 32: Marker Opportunities
  • Figure 33: PORTER'S Five Force Analysis