遺傳性乳清酸尿症的全球市場：按藥物類型、適應症、按分銷管道、按地區

2023年，全球遺傳性乳清酸尿症市值為3.579億美元，預計2031年將達到5.098億美元，2024年至2031年的年複合成長率為4.5%。

圖 1. 2024 年遺傳性乳清酸尿症的全球市場佔有率（%），依地區分類

遺傳性乳清酸尿症（HOA）是一種極為罕見的嘧啶代謝先天性疾病。它是由 5-尿苷單磷酸合成酶 (UMPS) 基因缺陷引起的。遺傳性乳清酸尿症是一種極為罕見的遺傳疾病。如果不治療，受影響的嬰兒會出現一種稱為巨幼紅血球貧血的血液（血液）疾病，以及發育不良、對感染疾病的易感性增加，並且乳清酸從尿液中排出，乳清酸結晶（結晶）可能會出現在尿液中。遺傳性乳清酸尿症並不為人所知，因為很少人患有這種疾病。遺傳性乳清酸尿症是嘧啶生物合成途徑中唯一已知的酵素缺乏症，由雙功能酶 5-單磷酸尿苷合成酶 (UMPS) 的一種或兩種活性缺陷引起。據推測，HOA 的原因是由於兩個錯義突變導致酶水平降低和基材結合受損。 UMPS 缺乏會導致乳清酸 (OA) 和乳清苷單磷酸 (OMP) 積聚，最終透過尿液排放。此疾病的特徵是巨幼細胞骨髓對凝血治療具有抵抗力，而尿乳清酸排泄量顯著增加。

市場動態：

政府對罕見疾病採取的舉措的增加預計將在預測期內推動市場成長。例如，2021年3月，印度政府推出了國家罕見疾病政策（NPRD），用於治療罕見疾病患者。 2021年NPRD的主要特徵如下： 罕見疾病分為三組：第1組、第2組和第3組。

• 第一組：可以治療一次的疾病。
• 第 2 組：需要長期、終身治療、相對便宜、文獻已證明效用、需要每年或更頻繁監測的疾病。
• 第 3 組：可以根治性治療的疾病，但面臨的挑戰包括最佳患者選擇以獲得益處、高昂的成本和終身治療。

研究的主要特點

• 本報告對全球遺傳性乳清酸尿症市場進行了詳細分析，並列出了以2023年為基準年的預測期（2024-2031）的市場規模和年複合成長率。
• 它還說明了各個細分市場的潛在收益成長機會，並為該市場提供了一系列有吸引力的投資提案。
• 它還提供了有關市場促進因素、限制因素、機會、新產品發布和核准、市場趨勢、區域前景、主要企業採取的競爭策略等的主要考察。
• 它根據公司亮點、產品系列、主要亮點、績效和策略等參數，介紹了全球遺傳性乳清酸尿症市場的主要企業。
• 該報告的見解使負責人和公司經營團隊能夠就未來的產品發布、升級、市場擴張和行銷策略做出明智的決策。
• 全球遺傳性乳清酸尿症市場報告迎合了該行業的各種相關人員，如投資者、供應商、產品製造商、分銷商、新進業者和財務分析師。
• 透過用於分析全球遺傳性乳清酸尿市場的各種策略矩陣，將促進相關人員的決策。

目錄

第1章 研究目的與前提

• 研究目標
• 先決條件
• 簡稱

第2章 市場展望

• 報告說明
  • 市場定義和範圍
• 執行摘要
• Coherent Opportunity Map（COM）

第3章 市場動態、法規與趨勢分析

• 市場動態
• 主要亮點
• 監管場景
• 最近的趨勢
• PEST分析
• 波特的分析
• 合併、收購和合作

第4章全球遺傳性乳清酸尿症市場－冠狀病毒（COVID-19）大流行的影響

• 新型冠狀病毒感染疾病（COVID-19）的流行病學
• 供需面分析
• 經濟影響

第5章全球遺傳性乳清酸尿症市場，依藥物類型，2019-2031

• 單磷酸胞苷
• 尿苷一磷酸

第6章 全球遺傳性乳清酸尿症市場，依適應症分類，2019-2031

• 類型1
• 2型

第7章全球遺傳性乳清酸尿症市場，依通路，2019-2031

• 醫院藥房
• 零售藥房
• 網路藥房

第8章全球遺傳性乳清酸尿症市場，依地區分類，2019-2031

• 北美洲
• 美國
• 加拿大
• 歐洲
• 德國
• 英國
• 西班牙
• 法國
• 義大利
• 俄羅斯
• 其他歐洲國家
• 亞太地區
• 中國
• 印度
• 日本
• 澳洲
• 韓國
• ASEAN
• 其他亞太地區
• 拉丁美洲
• 巴西
• 阿根廷
• 墨西哥
• 其他拉丁美洲
• 中東
• 海灣合作理事會國家
• 以色列
• 其他中東地區
• 非洲
• 北非
• 中部非洲
• 南非

第9章 競爭格局

• 公司簡介
  • Merck &Co. Inc
  • SERB Pharmaceuticals

第10章

• 參考
• 調查方法
• 關於出版商

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.

Figure 1. Global Hereditary Orotic Aciduria Market Share (%), By Region, 2024

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