全球溶酶体贮积症药物市场 – 行业趋势和 2030 年预测

溶酶体储存障碍药物市场分析和规模

溶酶体贮积症是一种遗传性代谢疾病，其特征是由于酶缺乏导致体内细胞中大量有害化学物质异常积累。此类疾病大约有 50 种，会影响骨骼、大脑、皮肤、心脏和中枢神经系统等身体部位。还有更多的溶酶体贮积症正在被发现。虽然一些此类疾病的潜在治疗方法的临床试验正在进行中，但许多溶酶体贮积症尚无获批的药物。                           

Data Bridge Market Research 分析认为，溶酶体贮积症药物的市场规模从 2022 年的 97.3863 亿美元飙升至 2030 年的 216.4692 亿美元，预计预测期内的复合年增长率为 10.50%。由于溶酶体疾病的患病率不断上升，“酶替代疗法”在溶酶体贮积症药物的治疗领域占据主导地位，除了对市场价值、增长率、细分、地理覆盖范围和主要参与者等市场情景的见解外，Data Bridge Market Research 整理的市场报告还包括深度专家分析、患者流行病学、管道分析、定价分析和监管框架。

溶酶体储存症药物市场范围和细分

Market Definition

Lysosomal or lysosomal storage disease is a rare hereditary metabolic disease. It is caused by faulty or mutant metabolism, which results in the lack or deficiency of enzymes, resulting in an abnormal material build-up in various body cells. If done before age two, bone marrow transplantation (BMT) successfully prevents progressive mental retardation in children with MPS IH (Hurler disease). Enzyme Replacement Therapy (ERT) is successful for those with Gaucher disease type I. Anemia and low platelet counts have improved, liver and spleen enlargement has decreased significantly, and skeletal abnormalities have improved. Individuals with Gaucher disease types II and III who get ERT also see improvements in these systemic signs.

Lysosomal Storage Disorder Drugs Market Dynamics

Drivers

• Prevalence and Awareness:

The increasing recognition and diagnosis of lysosomal storage disorders worldwide have driven the demand for effective treatment options. As awareness about these rare genetic disorders grows, more patients are being identified, leading to a greater need for drug development.

• Unmet Medical Need

Lysosomal storage disorders are a group of rare genetic diseases characterized by the deficiency of specific enzymes within lysosomes, resulting in the accumulation of undigested materials in cells. These disorders often lead to severe, progressive symptoms and can be life-threatening. Due to the limited treatment options available, there is a significant unmet medical need for effective therapies, driving the development of drugs in this field.

• Orphan Drug Incentives

Governments and regulatory agencies worldwide have implemented various incentives to promote the development of drugs for rare diseases, including lysosomal storage disorders. These incentives may include extended market exclusivity, tax credits, research grants, and accelerated regulatory pathways. Such incentives encourage pharmaceutical companies to invest in research and development efforts for these rare conditions.

• Advances in Biotechnology

The advancements in biotechnology and drug discovery techniques have significantly contributed to developing novel therapies for lysosomal storage disorders. Innovative approaches such as enzyme replacement therapies, substrate reduction therapies, gene therapies, and small molecule inhibitors have shown promising results in preclinical and clinical studies, fueling further investment in this field.

Opportunities

• Large Addressable Market

While individual LSDs are rare, there are over 50 known lysosomal storage disorders, each with unique genetic defects and clinical manifestations. Collectively, these disorders affect a substantial number of patients worldwide. The combined prevalence of LSDs makes for a sizable addressable market, presenting an opportunity for drug developers and manufacturers.

• Limited Treatment Options

Historically, treatment options for LSDs were limited, primarily focusing on symptomatic management. However, recent advancements in understanding the underlying biology of these disorders have led to the development of innovative therapies. The availability of disease-modifying treatments, such as enzyme replacement therapies, substrate reduction therapies, gene therapies, and small molecule inhibitors, has expanded the treatment landscape, providing better options for patients and driving market growth.

Restraints/Challenges

• Limited Patient Population

Lysosomal storage disorders are rare genetic diseases, and each specific disorder affects a relatively small number of patients. The limited patient population poses challenges for drug developers and manufacturers in achieving economies of scale and generating substantial revenue. The small market size may also deter some companies from investing in the research and development of LSD drugs.

• High Development Cost

Developing drugs for rare diseases, including LSDs, can be expensive and time-consuming. The cost of conducting clinical trials, obtaining regulatory approvals, and ensuring safety and efficacy can be substantial. The relatively small patient population and the complex nature of LSDs may result in higher per-patient costs, making the development of LSD drugs financially challenging for some companies.

This   Hospice  Market report provides details of new recent developments, trade regulations, import-export analysis, production analysis, value chain optimization, market share, the impact of domestic and localized market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographic expansions, technological innovations in the market. To gain more info on the Hospice  Market Contact Data Bridge Market Research for an Analyst Brief, our team will help you take an informed market decision to achieve market growth.

Recent Development

• 2021 年 1 月，开发用于治疗溶酶体贮积症 (LSD) 的下一代重组酶和基因疗法的私营生命科学公司 M6P Therapeutics 宣布，美国食品药品监督管理局 (FDA) 已授予其 LSD 开发管线内各种项目六项儿科罕见病认定 (RPDD)，其中包括四种重组酶和两种基因疗法项目
• 2019 年 7 月，武田制药公司在印度推出了针对溶酶体贮积症 (LSD) 的酶替代疗法产品组合，扩大了其在全国范围内的罕见病治疗范围

全球溶酶体贮积症药物范围

溶酶体贮积症药物根据治疗、适应症和最终用户进行细分。这些细分市场之间的增长将帮助您分析行业中增长缓慢的细分市场，并为用户提供有价值的市场概览和市场洞察，帮助他们做出战略决策，确定核心市场应用。

治疗

• 酶替代疗法
• 干细胞治疗
•  底物还原疗法
•  其他的

指征  

• 戈谢氏病
• 法布里病
•  庞贝氏症候群
•  粘多糖贮积症，
• 其他的

最终用户

• 医院，
• 诊所
• 其他的

溶酶体贮积症药物区域分析/见解

对溶酶体贮积症药物进行了分析，并按国家、治疗、适应症和最终用户提供了市场规模见解和趋势。

溶酶体贮积症药物报告涵盖的国家包括美国、加拿大和墨西哥、德国、法国、英国、荷兰、瑞士、比利时、俄罗斯、意大利、西班牙、土耳其、欧洲其他地区、中国、日本、印度、韩国、新加坡、马来西亚、澳大利亚、泰国、印度尼西亚、菲律宾、亚太其他地区、沙特阿拉伯、阿联酋、南非、埃及、以色列、中东和非洲其他地区、巴西、阿根廷和南美洲其他地区

北美在溶酶体贮积症药物领域占据主导地位，因为该地区拥有强大的医疗设施基础、主要市场参与者的强大影响力、溶酶体疾病的患病率不断上升以及研究活动数量不断增加。   

预计亚太地区将在 2023 年至 2030 年的预测期内出现显著增长，这得益于政府加强宣传力度、医疗旅游业兴起、该地区研究活动增多、有大量未开发市场、人口众多以及对优质医疗服务的需求不断增长。

报告的国家部分还提供了影响市场当前和未来趋势的各个市场影响因素和国内市场监管变化。下游和上游价值链分析、技术趋势和波特五力分析、案例研究等数据点是用于预测各个国家市场情景的一些指标。此外，在提供国家数据的预测分析时，还考虑了全球品牌的存在和可用性以及它们因来自本地和国内品牌的大量或稀缺竞争而面临的挑战、国内关税和贸易路线的影响。   

医疗保健基础设施增长安装基础和新技术渗透

溶酶体贮积症药物还为您提供每个国家/地区医疗保健资本设备支出增长、溶酶体贮积症药物不同类型产品的安装基数、使用生命线曲线的技术影响以及医疗保健监管情景的变化及其对溶酶体贮积症药物的影响的详细市场分析。数据适用于 2010-2020 年的历史时期。

竞争格局和溶酶体贮积症药物份额分析

溶酶体贮积症药物竞争格局提供了按竞争对手划分的详细信息。详细信息包括公司概况、公司财务状况、收入、市场潜力、研发投资、新市场计划、全球影响力、生产基地和设施、生产能力、公司优势和劣势、产品发布、产品宽度和广度、应用主导地位。以上提供的数据点仅与公司对溶酶体贮积症药物的关注有关。

从事溶酶体贮积症药物研发的一些主要公司包括：

• 诺华公司（瑞士）
• 强生私人有限公司（美国）
• Teva Pharmaceutical Industries Ltd.（爱尔兰）
• 默克公司（美国）
• 艾尔建（爱尔兰）
• 辉瑞公司（美国）
• 葛兰素史克公司 (英国)
• 赛诺菲（法国）
• 默克集团（德国）
• 雅培（美国）
• 勃林格殷格翰国际有限公司（德国）
• 武田药品工业株式会社 (日本)
• Amicus Therapeutics, Inc.（美国）
• Moderna, Inc.（美国）
• Greenovation Biotech GmbH（德国）
• Biomarin（美国）
• JCR制药有限公司（日本）
• ISU ABXIS（韩国）
• Idorsia Pharmaceuticals Ltd（瑞士）
• AVROBIO, Inc.（美国）
• Resverlogix Corp.（加拿大）
• Enzyvant（美国）
• CHIESI Farmaceutici SpA（意大利） 

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