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市场调查报告书
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1776219
运动神经元疾病治疗的全球市场:洞察,竞争情形,市场预测:2032年Motor Neuron Disease Treatment - Market Insights, Competitive Landscape, and Market Forecast - 2032 |
预计在2025年至2032年的预测期内,运动神经元疾病 (MND) 治疗市场将以5.87%的复合年增长率成长。由于肌萎缩侧索硬化症 (ALS) 和脊髓性肌肉萎缩症 (SMA) 等运动神经元疾病的盛行率不断上升,对运动神经元疾病 (MND) 治疗的需求正在显着增长。人们越来越意识到早期和准确诊断的重要性,这进一步推动了市场发展势头。此外,生物疗法的显着转变,提供了更有针对性和更有效的治疗选择,正在改变治疗格局。再加上研发投入的不断增加以及 MND 治疗领域的持续技术创新,为市场扩张创造了极为有利的环境。综合来看,这些因素将使运动神经元疾病 (MND) 治疗市场在 2025 年至 2032 年的预测期内保持强劲持续的成长。
运动神经元疾病治疗市场动态:
根据 Taylor & Francis Online 发布的最新数据(2025 年),全球肌萎缩侧索硬化症 (ALS) 的负担预计将稳定增加。预计2022年报告的ALS病例约为32,893例,到2030年将成长10%以上,达到36,308例。这一上升趋势不仅反映了盛行率的上升,也凸显了诊断能力的提高和疾病意识的增强。
DelveInsight的2023年市场分析显示,在美国、英国、德国、法国、义大利、西班牙和日本等七个主要市场,ALS的盛行率约为67,000例,这佐证了这一点。这表明,在现有的医疗保健体系中,对有效的治疗干预措施的需求巨大。根据澳洲运动神经元疾病协会 (MND Australia) 2024 年的数据,每天至少有两人被诊断出患有运动神经元疾病 (MND),目前约有 2,688 人患有该疾病,这凸显了对可及治疗策略的迫切需求。
就脊髓性肌肉萎缩症 (SMA) 而言,这是一种罕见但严重的遗传性神经肌肉疾病。脊髓性肌肉萎缩症基金会 (SMA) 2023 年估计,目前美国有 10,000 至 25,000 人受其影响。此病的出生盛行率为 1/6,000 至 1/10,000,凸显了早期筛检和介入的重要性。基因疗法,特别是使用腺相关病毒载体进行的SMN1基因替换,已成为突破性的解决方案,显着改善了患者的长期疗效,并在某些情况下恢復了运动功能。
总而言之,运动神经元疾病(尤其是ALS和SMA)的盛行率上升,是全球运动神经元疾病(MND)治疗市场快速扩张的主要驱动力。这些以肌肉无力、呼吸功能障碍以及最终的运动技能丧失为特征的进行性神经系统疾病,日益被视为严重的公共卫生问题。因此,对创新疾病改良疗法的需求日益增长。
在治疗方面,MND治疗的典范转移正在进行中。对于ALS,治疗重点已从缓解症状扩展到开发旨在减缓神经元退化进展的神经保护剂。对于SMA,基因疗法正在重新定义治疗标准,为治癒疾病提供了可能性,并改变了许多患者的疾病轨迹。
同时,监管环境对技术创新的支持力道也不断增加。美国食品药物管理局 (FDA) 和欧洲药品管理局 (EMA) 近期的批准增强了市场信心。尤其是 Zolgensma® (onasemnogene abeparvovec) 的获批,验证了基因疗法的有效性,并加速了针对运动神经元保存和肌肉增强的下一代疗法的投资。
市场开发公司也在加强研发力度,以将先进的标靶疗法商业化。例如,2023 年 4 月,Biogen Inc. 获得 FDA 加速批准,其鞘内注射 QALSODY™(通用名:tofersen)用于治疗已确诊超氧化物歧化酶 1 (SOD1) 基因突变的成人肌萎缩侧索硬化症 (ALS)。这项决定是基于血浆神经丝轻链 (NfL) 水平的降低,神经丝轻链 (NfL) 是一种与神经元损伤相关的生物标誌物,这标誌着生物标记驱动的治疗发展迈出了重要一步。
总而言之,ALS 和 SMA 的盛行率和认知度不断提高,加上治疗创新的进步和有利的监管环境,正在创造一个强劲而充满活力的运动神经元疾病治疗市场。这些发展为该领域在 2025 年至 2032 年的预测期内实现持续成长和持续的科学突破奠定了基础。
然而,高昂的治疗费用以及监管机构和政府的严格指导方针是限制运动神经元疾病治疗市场成长的主要限制因素。
运动神经元疾病治疗市场区隔分析:
按药物类别划分的运动神经元疾病治疗市场中,预计存活运动神经元 (SMN) 激动剂类别将在 2024 年占据最大市场占有率。此类别的成长主要得益于脊髓性肌肉萎缩症 (SMA) 盛行率的上升以及 SMN1 标靶疗法带来的诱人疗效。 SMN 蛋白对于运动神经元的存活和功能至关重要,而运动神经元对于随意肌肉运动至关重要。 SMN 激动剂透过增强 SMN 蛋白的生成发挥作用,帮助维持运动功能、减缓病情进展并改善 SMA 患者的整体生活品质。
例如,根据英国国家医疗服务体系 (NHS England) (2023) 的数据,英国每年约有 70 名 SMA 儿童出生。同样,澳洲人类遗传学会 (2024) 报告称,每 10,000 人中就有 1 人出生时患有 SMA,每 40 人中就有 1 人是相关基因突变的健康携带者,这凸显了 SMA 的高风险人群以及对有效干预措施的迫切需求。
SMN 标靶疗法的一个显着优势是其能够提供跨类型疗效,从而为不同 SMA 亚型(从严重的 1 型到较轻的 3 型)带来临床益处。例如,被诊断为最严重 SMA(1 型)的婴儿在运动功能方面表现出显着改善,达到了无需辅助即可坐起等发育里程碑,这在未经治疗的病例中很少见。
除了改善运动功能外,口服 SMN 激动剂疗法还具有改善可及性和依从性的优势,尤其是在儿科患者中。这使得长期治疗管理更加可行且对患者更友好,有助于提高依从性并改善疗效。透过提高SMN蛋白水平,SMN激动剂有助于减缓SMA的进展,缓解相关的神经肌肉症状,并延长生存期,尤其是在早发重症病例中。
不断增加的研发投入和监管发展进一步推动了这一类别的成长。主要的行业参与者正在积极开发下一代SMN标靶疗法,以提高疗效和便利性。一个显着的例子是罗氏公司于2025年2月宣布,美国食品药物管理局(FDA)已批准Evrysdi® (risdiplam)片剂的新药申请(NDA)。 Evrysdi是一种SMN2前mRNA剪接修饰剂,旨在治疗SMA,该疾病是由5q染色体基因突变导致SMN蛋白缺失引起的。该片剂是一种口服全身性治疗药物,可在家服用,减少了住院输液的需求,使患者和家属更容易获得治疗。
这些因素预计将推动细分市场的发展,并最终推动全球运动神经元疾病治疗市场的发展。
预计到2024年,北美将占据全球运动神经元疾病治疗市场的最大占有率,这得益于其关键的结构性和战略优势。该地区的主导地位主要源于脊髓性肌肉萎缩症 (SMA) 和肌萎缩侧索硬化症 (ALS) 等运动神经元疾病的盛行率不断上升,这持续增加了医疗保健系统对有效治疗方案的需求。此外,该地区拥有强劲的製药环境,大型生物技术和製药公司积极开发、临床试验和商业化创新疗法。新产品审批速度维持较高水平,并得益于良好的监管架构。特别是,美国食品药物管理局 (FDA) 等机构已推出罕见疾病药物加速审批流程,支持快速提供尖端的MND治疗方案。加重的疾病负担、强大的研发活动以及积极的监管支持等因素共同巩固了北美在运动神经元疾病治疗市场的全球领先地位。
根据美国疾病管制与预防中心 (CDC, 2025) 的最新数据,2022 年美国将记录约 33,000 例肌萎缩侧索硬化症 (ALS) 病例,预计到 2030 年这一数字将增加 10% 以上,超过 36,000 例。同样,美国医学会 (2024) 报告称,2024 年美国每 14,694 名新生儿中就有 1 人将被诊断患有脊髓性肌肉萎缩症 (SMA),这凸显了北美运动神经元疾病 (MND) 负担的日益加重。
针对运动神经元的疗法越来越多地被用来对抗这些渐进性且往往致命的疾病。具体而言,治疗着重于替换缺失或缺陷的SMN1基因,提供功能性拷贝,以维护运动神经元健康并减缓肌肉退化。美国国立卫生研究院(2022年)指出,据估计,加拿大每年每10,000名新生儿中约有6,000至1人患有SMA,这支持了这一趋势,凸显了该地区对及时诊断和高级干预的需求。
SMA盛行率的上升是北美运动神经元疾病治疗市场成长的主要驱动力,从而导致对疾病修饰疗法的需求增加。由于SMA通常发生在婴儿期和幼儿期,因此早期基因筛检和创新疗法(例如针对SMN的基因疗法)的需求日益迫切,从而刺激了研发投资和治疗方法的开发。
推动该地区市场扩张的一个主要因素是严格的监管环境。美国食品药物管理局 (FDA) 正在透过为有前景的药物提供快速通道和突破性疗法认定,加速运动神经元疾病 (MND) 治疗的进展。值得注意的是,用于脊髓性肌肉萎缩症 (SMA) 的基因疗法 Zolgensma® 获得了加速审批,这标誌着疾病修饰治疗取得了突破,并进一步验证了基于基因的治疗方法。
除了监管支援外,美国主要製药公司的持续创新也增强了市场发展动能。例如,2023 年 4 月,百健公司 (Biogen Inc.) 宣布其鞘内注射 QALSODY™(通用名:tofersen)已获得 FDA 加速审批,用于治疗已确诊 SOD1 基因突变的肌萎缩侧索硬化症 (ALS) 患者。此审批是基于血浆中 IL-16(一种与神经元损伤相关的生物标记)的检测结果。QALSODY 作为一种新型标靶疗法在美国市场上销售,该药物能够降低运动神经元疾病患者的神经丝轻链 (NfL) 水平。
不断上升的疾病盛行率、持续的产品创新、监管支持以及行业领导者的策略性举措,都是推动北美运动神经元疾病治疗市场在 2025 年至 2032 年预测期内强劲增长的因素。
运动神经元疾病治疗市场的主要参与者:
运动神经元疾病治疗市场的主要参与者包括 Biogen、三菱化学集团株式会社、罗氏製药有限公司、大冢控股株式会社、赛诺菲、Ionis Pharmaceuticals, Inc.、诺华公司、EDW Pharmace Inc.、PTC Therapeutics, Inc.、中外株式会社和 AMYLYXceticals。
运动神经元疾病治疗市场的最新进展:
运动神经元疾病治疗市场研究报告的主要发现
关于运动神经元疾病治疗的常见问题市场
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:
Key takeaways from the motor neuron disease treatment market report study
Target audience who can benefit from this motor neuron disease treatment market report study
Frequently Asked Questions for the Motor Neuron Disease Treatment Market: