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市场调查报告书
商品编码
1449597

遗传性乳清酸尿症的全球市场：按药物类型、适应症、按分销管道、按地区

Global Hereditary Orotic Aciduria Market, By Drug Type, By Indication, By Distribution Channel, By Geography
出版日期: | 出版商: Coherent Market Insights | 英文 160 Pages | 商品交期: 2-3个工作天内

价格
简介目录

2023年，全球遗传性乳清酸尿症市值为3.579亿美元，预计2031年将达到5.098亿美元，2024年至2031年的复合年增长率为4.5%。

报告范围 报告详情
基准年 2023年 2023/2024年市场规模 3.579 亿美元
实际资料 2019-2023 预测期 2024-2031
预测期2023/2024~2030/2031年复合成长率： 4.50% 2030/2031 年预测值 5.098 亿美元
图 1. 2024 年遗传性乳清酸尿症的全球市场占有率（%），依地区划分
全球遗传性乳清酸尿症市场-IMG1

遗传性乳清酸尿症（HOA）是一种极为罕见的嘧啶代谢先天性疾病。它是由 5-尿苷单磷酸合成酶 (UMPS) 基因缺陷引起的。遗传性乳清酸尿症是一种极为罕见的遗传疾病。如果不治疗，受影响的婴儿会出现一种称为巨幼红血球贫血的血液（血液）疾病，以及发育不良、对感染疾病的易感性增加，并且乳清酸从尿液中排出，乳清酸结晶（结晶）可能会出现在尿液中。遗传性乳清酸尿症并不为人所知，因为很少人患有这种疾病。遗传性乳清酸尿症是嘧啶生物合成途径中唯一已知的酵素缺乏症，由双功能酶 5-单磷酸尿苷合成酶 (UMPS) 的一种或两种活性缺陷引起。据推测，HOA 的原因是由于两个错义突变导致酶水平降低和基材结合受损。 UMPS 缺乏会导致乳清酸 (OA) 和乳清苷单磷酸 (OMP) 积聚，最终透过尿液排放。此疾病的特征是巨幼细胞骨髓对凝血治疗具有抵抗力，而尿乳清酸排泄量显着增加。

市场动态：

政府对罕见疾病采取的倡议的增加预计将在预测期内推动市场成长。例如，2021年3月，印度政府推出了国家罕见疾病政策（NPRD），用于治疗罕见疾病患者。 2021年NPRD的主要特征如下： 罕见疾病分为三组：第1组、第2组和第3组。

  • 第一组：可以治疗一次的疾病。
  • 第 2 组：需要长期、终身治疗、相对便宜、文献已证明效用、需要每年或更频繁监测的疾病。
  • 第 3 组：可以根治性治疗的疾病，但面临的挑战包括最佳患者选择以获得益处、高昂的成本和终身治疗。

研究的主要特点

  • 本报告对全球遗传性乳清酸尿症市场进行了详细分析，并列出了以2023年为基准年的预测期（2024-2031）的市场规模和復合年增长率。
  • 它还说明了各个细分市场的潜在收益成长机会，并为该市场提供了一系列有吸引力的投资提案。
  • 它还提供了有关市场驱动因素、限制因素、机会、新产品发布和核准、市场趋势、区域前景、主要企业采取的竞争策略等的主要考察。
  • 它根据公司亮点、产品系列、主要亮点、绩效和策略等参数，介绍了全球遗传性乳清酸尿症市场的主要企业。
  • 该报告的见解使负责人和公司经营团队能够就未来的产品发布、升级、市场扩张和行销策略做出明智的决策。
  • 全球遗传性乳清酸尿症市场报告迎合了该行业的各种相关人员，如投资者、供应商、产品製造商、分销商、新进业者和财务分析师。
  • 透过用于分析全球遗传性乳清酸尿市场的各种策略矩阵，将促进相关人员的决策。

目录

第一章 研究目的与前提

  • 研究目标
  • 先决条件
  • 简称

第二章 市场展望

  • 报告说明
    • 市场定义和范围
  • 执行摘要
  • Coherent Opportunity Map（COM）

第三章 市场动态、法规与趋势分析

  • 市场动态
  • 主要亮点
  • 监管场景
  • 最近的趋势
  • PEST分析
  • 波特的分析
  • 合併、收购和合作

第四章全球遗传性乳清酸尿症市场－冠状病毒（COVID-19）大流行的影响

  • 新型冠状病毒感染疾病（COVID-19）的流行病学
  • 供需面分析
  • 经济影响

第五章全球遗传性乳清酸尿症市场，依药物类型，2019-2031

  • 单磷酸胞苷
  • 尿苷一磷酸

第六章 全球遗传性乳清酸尿症市场，依适应症分类，2019-2031

  • 类型1
  • 2型

第7章全球遗传性乳清酸尿症市场，依通路，2019-2031

  • 医院药房
  • 零售药房
  • 网路药房

第八章全球遗传性乳清酸尿症市场，依地区划分，2019-2031

  • 北美洲
  • 美国
  • 加拿大
  • 欧洲
  • 德国
  • 英国
  • 西班牙
  • 法国
  • 义大利
  • 俄罗斯
  • 其他欧洲国家
  • 亚太地区
  • 中国
  • 印度
  • 日本
  • 澳洲
  • 韩国
  • ASEAN
  • 其他亚太地区
  • 拉丁美洲
  • 巴西
  • 阿根廷
  • 墨西哥
  • 其他拉丁美洲
  • 中东
  • 海湾合作委员会国家
  • 以色列
  • 其他中东地区
  • 非洲
  • 北非
  • 中部非洲
  • 南非

第9章 竞争格局

  • 公司简介
    • Merck &Co. Inc
    • SERB Pharmaceuticals

第10章

  • 参考
  • 调查方法
  • 关于出版商
简介目录
Product Code: CMI6626

Global hereditary orotic aciduria market was valued at US$ 357.9 Mn in 2023 and is expected to reach US$ 509.8 Mn by 2031, exhibiting at a compound annual growth rate (CAGR) of 4.5% from 2024 to 2031.

Report Coverage Report Details
Base Year: 2023 Market Size in 2023/2024: US$ 357.9 Mn
Historical Data for: 2019 to 2023 Forecast Period: 2024 - 2031
Forecast Period 2023/2024 to 2030/2031 CAGR: 4.50% 2030/2031 Value Projection: US$ 509.8 Mn
Figure 1. Global Hereditary Orotic Aciduria Market Share (%), By Region, 2024
Global Hereditary Orotic Aciduria Market - IMG1

Hereditary orotic aciduria (HOA) is a very rare inborn error of pyrimidine metabolism. It results from a defect of the uridine-5-monophosphate synthase (UMPS) gene. Hereditary orotic aciduria is an extremely rare genetic disorder. When untreated, affected infants can develop a blood (hematologic) disorder called megaloblastic anemia as well as failure to thrive, susceptibility to infection, and orotic acid crystals in the urine (crystalluria), resulting from excretion of orotic acid in the urine. Since, very few individuals have been identified with this disorder, not much is known of hereditary orotic aciduria. It is the only known as the enzyme deficiency of the pyrimidine biosynthetic pathway, resulting from a deficiency in one or both of the activities of the bifunctional enzyme uridine-5-monophosphate synthase (UMPS). The presumed cause of HOA is biallelic missense mutations resulting in decreased levels of the enzyme and impaired substrate binding. UMPS defects lead to the accumulation of orotate (OA) and/or of orotidine monophosphate (OMP), which eventually getsexcreted in the urine. The hallmarks of the disease are a megaloblastic bone marrow that is refractory to hematinic therapy, accompanied by a markedly increased excretion of orotic acid in the urine.

Market Dynamics:

Increasing government initiatives for rare diseases is expected to drive the growth of the market over the forecast period. For instance, in March 2021, the government of India has launched National Policy for Rare Diseases (NPRD) for the treatment of rare disease patients. The salient features of NPRD, 2021 are as under: The rare diseases have been identified and categorized into 3 groups namely Group 1, Group 2 and Group 3.

  • Group 1: Disorders amenable to one-time curative treatment.
  • Group-2: Diseases requiring long term/lifelong treatment having relatively lower cost of treatment and benefit has been documented in literature and annual or more frequent surveillance is required.
  • Group 3: Diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost, and lifelong therapy.

Key features of the study:

  • This report provides an in-depth analysis of the global hereditary orotic aciduria market, and provides market size (US$ Mn) and compound annual growth rate (CAGR %) for the forecast period (2024-2031), considering 2023 as the base year.
  • It elucidates potential revenue growth opportunities across different segments and explains attractive investment proposition matrices for this market.
  • This study also provides key insights about market drivers, restraints, opportunities, new product launches or approvals, market trends, regional outlook, and competitive strategies adopted by key players.
  • It profiles key players in the global hereditary orotic aciduria market based on the following parameters - company highlights, products portfolio, key highlights, financial performance, and strategies.
  • Key companies covered as a part of this study include Merck & Co. Inc and SERB Pharmaceuticals.
  • Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics.
  • Global hereditary orotic aciduria market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts.
  • Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global hereditary orotic aciduria market.

Detailed Segmentation:

  • Global Hereditary Orotic Aciduria Market, By Drug Type
    • Cytidine monophosphate
    • Uridine monophosphate
  • Global Hereditary Orotic Aciduria Market, By Indication
    • Type 1
    • Type 2
  • Global Hereditary Orotic Aciduria Market, By Distribution Channel
    • Hospital Pharmacies
    • Retail Pharmacies
    • Online Pharmacies
  • Global Hereditary Orotic Aciduria Market, By Region
    • North America
    • Europe
    • Asia Pacific
    • Latin America
    • Middle East
    • Africa
  • Top Companies in the Global Hereditary Orotic Aciduria Market:
    • Merck & Co. Inc
    • SERB Pharmaceuticals

Table of Contents

1. Research Objectives and Assumptions

  • Research Objectives
  • Assumptions
  • Abbreviations

2. Market Purview

  • Report Description
    • Market Definition and Scope
  • Executive Summary
    • Global Hereditary Orotic Aciduria Market, By Drug Type
    • Global Hereditary Orotic Aciduria Market, By Indication
    • Global Hereditary Orotic Aciduria Market, By Distribution Channel
    • Global Hereditary Orotic Aciduria Market, By Region
  • Coherent Opportunity Map (COM)

3. Market Dynamics, Regulations, and Trends Analysis

  • Market Dynamics
    • Government support in the form of funding for rare disease research
    • High cost of treatment
    • Growth opportunities in developing countries
  • Key Highlights
  • Regulatory Scenario
  • Recent Trends
  • PEST Analysis
  • PORTER's Analysis
  • Mergers, Acquisitions, and Collaborations

4. Global Hereditary Orotic Aciduria Market - Impact of Coronavirus (COVID-19) Pandemic

  • COVID-19 Epidemiology
  • Supply Side and Demand Side Analysis
  • Economic Impact

5. Global Hereditary Orotic Aciduria Market, By Drug Type, 2019-2031, (US$ Mn)

  • Introduction
    • Market Share Analysis, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, 2020 - 2031
    • Segment Trends
  • Cytidine monophosphate
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
  • Uridine monophosphate
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)

6. Global Hereditary Orotic Aciduria Market, By Indication, 2019-2031, (US$ Mn)

  • Introduction
    • Market Share Analysis, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, 2020 - 2031
    • Segment Trends
  • Type 1
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
  • Type 2
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)

7. Global Hereditary Orotic Aciduria Market, By Distribution Channel, 2019-2031, (US$ Mn)

  • Introduction
    • Market Share Analysis, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, 2020 - 2031
    • Segment Trends
  • Hospital Pharmacies
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
  • Retail Pharmacies
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
  • Online Pharmacies
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)

8. Global Hereditary Orotic Aciduria Market, By Region, 2019-2031, (US$ Mn)

  • Introduction
    • Market Share Analysis, By Region, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, For Region, 2020 - 2031
    • Country Trends
  • North America
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
  • U.S.
  • Canada
  • Europe
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
  • Germany
  • U.K.
  • Spain
  • France
  • Italy
  • Russia
  • Rest of Europe
  • Asia Pacific
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
  • China
  • India
  • Japan
  • Australia
  • South Korea
  • ASEAN
  • Rest of Asia Pacific
  • Latin America
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
  • Brazil
  • Argentina
  • Mexico
  • Rest of Latin America
  • Middle East
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
  • GCC Countries
  • Israel
  • Rest of Middle East
  • Africa
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
    • Market Size and Forecast, and Y-o-Y Growth, By Country/Region, 2019-2031,(US$ Mn)
  • North Africa
  • Central Africa
  • South Africa

9. Competitive Landscape

  • Company Profile
    • Merck & Co. Inc
  • Company Highlights
  • Drug Type Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
    • SERB Pharmaceuticals
  • Company Highlights
  • Drug Type Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
  • Analyst Views

10. Section

  • References
  • Research Methodology
  • About us