全球鸟胺酸氨甲酰基转移酶缺乏症市场规模：依产品类型、最终用户、区域范围和预测

鸟胺酸氨甲酰基转移酶缺乏症的市场规模及预测

2024年鸟胺酸氨甲酰基转移酶缺乏症市场规模价值 6.8874亿美元，预计到2031年将达到 9.3033亿美元，2024年至2031年的年复合成长率为 3.83%。

鸟胺酸氨甲酰基转移酶缺乏症市场的主要参与者针对家庭和医疗保健专业人士开展的越来越多的宣传活动推动鸟胺酸氨甲酰基转移酶缺乏症治疗市场的发展。全球鸟胺酸氨甲酰基转移酶缺乏症市场报告对市场进行了全面的评估。它对关键细分市场、趋势、市场驱动因素、限制因素、竞争格局以及在市场中发挥关键作用的因素进行了全面的分析。

全球鸟胺酸氨甲酰基转移酶缺乏症市场的定义

遗传性疾病鸟胺酸氨甲酰基转移酶缺乏症会导致氨在血液中积聚。当人体分解蛋白质时，会产生氨，过量的氨可能有害。过量的氨对神经系统的损害尤其大。鸟胺酸氨甲酰基转移酶缺乏症可发生在任何年龄。生命最初几天是最严重的。这种新生儿发病形式在男性中比在女性中更常见。患有新生儿鸟胺酸氨甲酰基转移酶缺乏症的新生儿可能会表现得昏睡不醒，不愿进食，呼吸频率和体温无法控制地升高。氨对神经系统特别有毒性，因此鸟氨酸转氨甲酰酶缺乏会导致神经系统问题以及肝臟疾病。

鸟胺酸氨甲酰基转移酶缺乏症的发生率估计为 1/14,000 至 1/77,000。患有新生儿发病疾病的患者更有可能被纳入这些估计值，因为患有晚期疾病的患者不太可能寻求医疗协助。 OTC 缺乏症是一种透过 X 染色体遗传的遗传性疾病。男性比女性更容易罹患 OTC 缺乏症，但只有男性才会出现全部症状。男性的症状通常出现在婴儿期的最初几天，但晚髮型 OTC 缺乏症也可能出现在以后的生活中。

约20%的女性带因者会出现轻微的疾病症状，只有一小部分人在儿童时期会出现严重的症状。多学科团队包括儿科医生、遗传学家、营养学家、神经科医生和内科医生。 OTC缺乏症的治疗目标是防止过量氨的产生或去除过量的氨。 OTC 缺乏症患者的饮食限制目的是尽量减少蛋白质的摄入，以避免氨的蓄积。患有 OTC 缺乏症的婴儿需要接受高热量、低蛋白质且含有必需胺基酸的饮食。帮助消除体内氮的药物也用于治疗非处方药缺乏症患者。

全球鸟胺酸氨甲酰基转移酶缺乏症市场概况

患有鸟胺酸氨甲酰基转移酶缺乏症的COVID-19 患者风险较高。此类患者因该疾病而患有先前存在的合併症。患有鸟胺酸氨甲酰基转移酶缺乏症的患者中 COVID-19 病例的惊人激增推动该行业的发展。患有糖尿病、高血压和肾病变等其他合併症的患者特别容易感染 COVID-19。有证据表明，对非处方药敏感的患者更容易受到伤害。这就是为什么医护人员不断努力增加基本药物供应的原因。

鸟胺酸氨甲酰基转移酶缺乏症与许多婴儿猝死症候群（SIDS）有关。 COVID-19 疫苗为鸟胺酸氨甲酰基转移酶缺乏症市场中的公司创造收入前景。 COVID-19 疫苗为鸟胺酸氨甲酰基转移酶缺乏症市场中的公司提供了收入前景，并有望在疫情期间维持需求、最大化公司的利润率并维持稳定的现金流。鸟胺酸氨甲酰基转移酶缺乏症市场正受益于这些原因。此外，鸟胺酸氨甲酰基转移酶缺乏症市场的主要公司针对家庭和医疗保健专业人员开展的提高认识的计划支持鸟胺酸氨甲酰基转移酶缺乏症治疗市场的成长。

预计全球鸟胺酸氨甲酰基转移酶缺乏症市场将受到患有鸟氨酸转氨甲酰酶（OTC）缺乏症的患者数量的增加的推动。推动全球鸟胺酸氨甲酰基转移酶缺乏症市场的关键因素是引进针对鸟氨酸转氨甲酰酶（OTC）缺乏症治疗的新疗法、针对 OTC 缺乏症治疗的优惠偿付政策以及多个国际和地区组织提供的各种患者支持计划。阻碍市场成长的因素之一是缺乏非处方药缺陷治疗的预后和诊断的资料。由于新冠肺炎疫情的影响，许多国家治疗非处方药缺陷所需原料的供应链已经中断。

另外，受疫情影响，药厂停产，导致医药业成长下滑。预计这将对鸟胺酸氨甲酰基转移酶缺乏症治疗市场产生重大影响。全球鸟胺酸氨甲酰基转移酶缺乏症市场的关键参与者收购新兴公司以扩大其产品范围。公司喜欢购买新创企业的研发部门并共同开发突破性技术。这项策略为企业提供了利用製造业和商业化其产品的巨大机会。

市场吸引力

市场吸引力有助于获取有关领先全球鸟胺酸氨甲酰基转移酶缺乏症市场的地区的资讯。涵盖了推动特定区域产业成长的关键影响因素。

波特五力模型

波特五力模型是用来理解产业内竞争对手行为和策略定位的架构。波特五力模型可用于评估全球鸟胺酸氨甲酰基转移酶缺乏症市场的竞争格局，衡量特定产业的吸引力，并评估投资潜力。

目录

第1章 全球鸟胺酸氨甲酰基转移酶缺乏症市场简介

• 市场概况
• 研究范围
• 先决条件

第2章 执行摘要

第3章 VERIFIED MARKET RESEARCH的研究方法

• 资料探勘
• 验证
• 主要来源
• 资料来源列表

第4章 全球鸟胺酸氨甲酰基转移酶缺乏症市场展望

• 概述
• 市场动态
  • 促进因素
  • 抑制因素
  • 机会
• 波特五力模型
• 价值链分析

第5章 全球鸟胺酸氨甲酰基转移酶缺乏症市场（依产品类型）

• 概述
• DTX-301
• SEL-313
• SHP-641
• PRX-OTC
• 其他

第6章 全球鸟胺酸氨甲酰基转移酶缺乏症市场（依最终用户）

• 概述
• 医院药房
• 零售药局
• 网路药局

第7章 全球鸟胺酸氨甲酰基转移酶缺乏症市场（依地区）

• 概述
• 北美洲
  • 美国
  • 加拿大
  • 墨西哥
• 欧洲
  • 德国
  • 英国
  • 法国
  • 其他欧洲国家
• 亚太地区
  • 中国
  • 日本
  • 印度
  • 其他亚太地区
• 世界其他地区
  • 拉丁美洲
  • 中东和非洲

第8章 全球鸟胺酸氨甲酰基转移酶缺乏症市场的竞争格局

• 概述
• 各公司的市场排名
• 主要发展策略

第9章 公司简介

• Abbott
• Nutricia
• Ultragenyx Pharmaceuticals
• Acer Therapeutics
• Bausch Health Companies Inc
• Mead Johnson
• Arcturus Therapeutics Inc
• Horizon Therapeutics
• Nestle
• OrphanPacific Inc

第10章 重大进展

• 产品发布/开发
• 合併和收购
• 业务扩展
• 伙伴关係和合作关係

第11章 附录

• 相关研究

Ornithine-Transcarbamylase Deficiency Market Size And Forecast

Ornithine-Transcarbamylase Deficiency Market size was valued at USD 688.74 Million in 2024 and is projected to reach USD 930.33 Million by 2031, growing at a CAGR of 3.83% from 2024 to 2031.

An increase in the number of awareness campaigns run by key players of the Ornithine-Transcarbamylase Deficiency Market for families and healthcare experts has boosted the market development for ornithine transcarbamylase insufficiency treatment. The Global Ornithine-Transcarbamylase Deficiency Market report provides a holistic evaluation of the market. The report offers a comprehensive analysis of key segments, trends, drivers, restraints, competitive landscape, and factors that are playing a substantial role in the market.

Global Ornithine-Transcarbamylase Deficiency Market Definition

Ammonia builds up in the bloodstream due to Ornithine Transcarbamylase deficiency, a hereditary condition. When proteins are broken down inside the body, ammonia is produced, which can be harmful if quantities rise too high. Excess ammonia has a particularly negative impact on the nervous system. Deficiency in ornithine transcarbamylase can occur at any age. In the first few days of life, the most severe form occurs. Males are more likely than females to develop this neonatal-onset variant of the illness. A newborn with neonatal-onset ornithine transcarbamylase deficiency may be getting lethargic or hesitant to eat, and his or her respiration rate and body temperature may be uncontrollably high. Because ammonia is particularly harmful to the nervous system, ornithine transcarbamylase deficiency results in neurological issues as well as liver disease.

The incidence of ornithine transcarbamylase deficiency has been estimated to be between 1 in 14,000 and 1 in 77,000 people. Because patients with the late-onset form of the illness are less likely to seek medical help, those with the neonatal-onset variant are more likely to be included in these estimations. OTC deficiency is a hereditary disease that is passed down through the X-chromosome. Males are more likely than females to be affected by OTC deficiency, which is only completely manifested in males. Symptoms in males usually appear in the first few days of infancy, however, late-onset OTC deficiency might appear later in life.

Approximately 20% of carrier females experience modest symptoms of the illness, and only a small percentage of them are severely affected as children. Pediatricians, geneticists, dieticians, neurologists, neurologists, and physicians make up a multidisciplinary team. The goal of treatment for OTC deficiency is to prevent excessive ammonia formation or to remove excessive ammonia. Dietary limitations in people with OTC deficiency are designed to keep protein consumption to a minimum to avoid ammonia buildup. Infants with OTC deficiency are put on a high-calorie, low-protein diet that includes necessary amino acids. Medications that promote the elimination of nitrogen from the body are also used to treat people with OTC deficiency.

Global Ornithine-Transcarbamylase Deficiency Market Overview

COVID-19 patients with ornithine transcarbamylase deficiency are at a higher risk. In such patients, this illness has resulted in previous comorbidities. The industry is being driven by an alarming surge in COVID-19 instances among patients who also have ornithine transcarbamylase deficiency. Patients with other co-morbidities, such as diabetes, hypertension, or kidney disease, are especially vulnerable to COVID-19. Evidence has shown that patients who are weak in OTCs are more vulnerable. As a result, healthcare providers are constantly seeking to boost the availability of essential pharmaceuticals.

Ornithine transcarbamylase deficiency has been linked to a large number of cases of sudden infant death syndrome (SIDS). COVID-19 vaccines are providing revenue prospects for companies in the Ornithine-Transcarbamylase Deficiency Market. This is expected to keep demand afloat for the duration of the epidemic, maximizing company margins and maintaining consistent cash flow. The Ornithine-Transcarbamylase Deficiency Market is benefiting from this reason. Furthermore, an increase in the number of awareness programs for families and healthcare professionals performed by key companies in the Ornithine-Transcarbamylase Deficiency Market has supported the market growth for ornithine transcarbamylase deficiency treatment.

The Global Ornithine-Transcarbamylase Deficiency Market is expected to be fueled by an increase in the number of patients with ornithine transcarbamylase (OTC) deficiency. Major factors that are driving the global Ornithine-Transcarbamylase Deficiency Market include the implementation of novel therapies for ornithine transcarbamylase (OTC) deficiency treatment, favorable reimbursement policies for OTC deficiency treatment, and various patient assistance programs provided by several international and regional organizations. One of the reasons that could slow the market's growth is a lack of data on the prognosis & diagnosis of the OTC deficiency treatment. The supply chain for the raw material needed to make medications for OTC deficiency has been interrupted in numerous countries as a result of COVID-19.

Furthermore, due to the pandemic, medicine makers have halted their manufacturing, resulting in a decline in pharmaceutical industry growth. This is predicted to have a big influence on the market for ornithine transcarbamylase deficient treatments. To extend their product range, key companies in the global Ornithine-Transcarbamylase Deficiency Market are acquiring emerging players. Companies prefer to buy rising players' R&D units to collaborate on revolutionary technology. The strategy provides businesses with an enormous opportunity to leverage manufacturing and commercialize products.

Market Attractiveness

The image of market attractiveness provided would further help to get information about the region that is majorly leading in the Global Ornithine-Transcarbamylase Deficiency Market. We cover the major impacting factors that are responsible for driving the industry growth in the given region.

Porter's Five Forces

The image provided would further help to get information about Porter's five forces framework providing a blueprint for understanding the behavior of competitors and a player's strategic positioning in the respective industry. Porter's five forces model can be used to assess the competitive landscape in the Global Ornithine-Transcarbamylase Deficiency Market, gauge the attractiveness of a certain sector, and assess investment possibilities.

Global Ornithine-Transcarbamylase Deficiency Market Segmentation Analysis

The Global Ornithine-Transcarbamylase Deficiency Market is Segmented on the basis of Product Type, End-User, And Geography.

Ornithine-Transcarbamylase Deficiency Market, By Product Type

• DTX-301
• SEL-313
• SHP-641
• PRX-OTC
• Others

Based on Product Type, the market is bifurcated into DTX-301, SEL-313, SHP-641, PRX-OTC, and Others. The DTX 301 segment holds the largest market share during the forecast period. DTX 301 is an investigational AAV gene therapy being developed for the treatment of individuals with ornithine transcarbamylase (OTC) deficiency.

Ornithine-Transcarbamylase Deficiency Market, By End-User

• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies

Based on End-User, the market is bifurcated into Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies. Because of the larger number of prescriptions filled at these hospital pharmacies, strong supply chain management, favorable reimbursement scenarios, and various patient assistance programs run by pharmaceutical companies, the hospital pharmacies segment accounted for a significant portion of the market.

Ornithine-Transcarbamylase Deficiency Market, By Geography

• North America
• Europe
• Asia Pacific
• Latin America
• Middle East and Africa
• On the basis of Regional Analysis, the Global Ornithine-Transcarbamylase Deficiency Market is classified into North America, Europe, Asia Pacific, Latin America, and Middle East and Africa. North America holds the largest market share. The availability of well-defined reimbursement policies from public and private health insurance firms, which help provide patients easy access to diagnosis, and ongoing projects will boost the market in the North American region.

Key Players

The "Global Ornithine-Transcarbamylase Deficiency Market" study report will provide valuable insight with an emphasis on the global market including some of the major players such as Abbott, Nutricia, Ultragenyx Pharmaceuticals, Acer Therapeutics, Bausch Health Companies Inc, Mead Johnson, Arcturus Therapeutics Inc, Horizon Therapeutics, Nestle, OrphanPacific Inc.

Our market analysis also entails a section solely dedicated to such major players wherein our analysts provide an insight into the financial statements of all the major players, along with product benchmarking and SWOT analysis. The competitive landscape section also includes key development strategies, market share, and market ranking analysis of the above-mentioned players globally.

Key Developments

• Feb 8, 2023 - Abbott and Cardiovascular Systems, Inc. (CSI) announced a definitive agreement for Abbott to acquire CSI, a medical device company with an innovative atherectomy system used in treating peripheral and coronary artery disease. Under terms of the agreement, CSI stockholders will receive $20 per common share at a total expected equity value of approximately $890 million.
• Dec 19, 2022 - Abbot announced the U.S. Food and Drug Administration (FDA) approval of the company's Eterna(TM) spinal cord stimulation (SCS) system - the smallest implantable, rechargeable spinal cord stimulator currently available on the market for the treatment of chronic pain.*4 Eterna SCS utilizes Abbott's proprietary low-dose BurstDR(TM) stimulation, the only SCS waveform technology with the highest level of clinical evidence (1A evidence), proven to reduce pain 23% more than traditional waveform technology approaches.
• DEC 15, 2022 - Nestle invested over CHF 100 million by 2030 in low-carbon logistics for its three international water brands - S. Pellegrino, Acqua Panna and Perrier. This investment will focus on optimizing logistics routes and accelerating train transport, increasing the use of alternative fuels and transportation options and testing innovative alternative transportation options. By eliminating unnecessary journeys and shifting towards alternative modes of transport, Nestle's international water brands will be able to reduce their total greenhouse gas emissions and contribute to Nestle's 2050 net zero commitment.
• Ace Matrix Analysis
• The Ace Matrix provided in the report would help to understand how the major key players involved in this industry are performing as we provide a ranking for these companies based on various factors such as service features & innovations, scalability, innovation of services, industry coverage, industry reach, and growth roadmap. Based on these factors, we rank the companies into four categories as Active, Cutting Edge, Emerging, and Innovators.

TABLE OF CONTENTS

1 INTRODUCTION OF GLOBAL ORNITHINE-TRANSCARBAMYLASE DEFICIENCY MARKET

• 1.1 Overview of the Market
• 1.2 Scope of Report
• 1.3 Assumptions

2 EXECUTIVE SUMMARY

3 RESEARCH METHODOLOGY OF VERIFIED MARKET RESEARCH

• 3.1 Data Mining
• 3.2 Validation
• 3.3 Primary Interviews
• 3.4 List of Data Sources

4 GLOBAL ORNITHINE-TRANSCARBAMYLASE DEFICIENCY MARKET OUTLOOK

• 4.1 Overview
• 4.2 Market Dynamics
  • 4.2.1 Drivers
  • 4.2.2 Restraints
  • 4.2.3 Opportunities
• 4.3 Porters Five Force Model
• 4.4 Value Chain Analysis

5 GLOBAL ORNITHINE-TRANSCARBAMYLASE DEFICIENCY MARKET, BY PRODUCT TYPE

• 5.1 Overview
• 5.2 DTX-301
• 5.3 SEL-313
• 5.4 SHP-641
• 5.5 PRX-OTC
• 5.6 Others

6 GLOBAL ORNITHINE-TRANSCARBAMYLASE DEFICIENCY MARKET, BY END-USER

• 6.1 Overview
• 6.2 Hospital Pharmacies
• 6.3 Retail Pharmacies
• 6.4 Online Pharmacies

7 GLOBAL ORNITHINE-TRANSCARBAMYLASE DEFICIENCY MARKET, BY GEOGRAPHY

• 7.1 Overview
• 7.2 North America
  • 7.2.1 U.S.
  • 7.2.2 Canada
  • 7.2.3 Mexico
• 7.3 Europe
  • 7.3.1 Germany
  • 7.3.2 U.K.
  • 7.3.3 France
  • 7.3.4 Rest of Europe
• 7.4 Asia Pacific
  • 7.4.1 China
  • 7.4.2 Japan
  • 7.4.3 India
  • 7.4.4 Rest of Asia Pacific
• 7.5 Rest of the World
  • 7.5.1 Latin America
  • 7.5.2 Middle East and Africa

8 GLOBAL ORNITHINE-TRANSCARBAMYLASE DEFICIENCY MARKET COMPETITIVE LANDSCAPE

• 8.1 Overview
• 8.2 Company Market Ranking
• 8.3 Key Development Strategies

9 COMPANY PROFILES

• 9.1 Abbott
  • 9.1.1 Overview
  • 9.1.2 Financial Performance
  • 9.1.3 Product Outlook
  • 9.1.4 Key Developments
• 9.2 Nutricia
  • 9.2.1 Overview
  • 9.2.2 Financial Performance
  • 9.2.3 Product Outlook
  • 9.2.4 Key Developments
• 9.3 Ultragenyx Pharmaceuticals
  • 9.3.1 Overview
  • 9.3.2 Financial Performance
  • 9.3.3 Product Outlook
  • 9.3.4 Key Developments
• 9.4 Acer Therapeutics
  • 9.4.1 Overview
  • 9.4.2 Financial Performance
  • 9.4.3 Product Outlook
  • 9.4.4 Key Developments
• 9.5 Bausch Health Companies Inc
  • 9.5.1 Overview
  • 9.5.2 Financial Performance
  • 9.5.3 Product Outlook
  • 9.5.4 Key Developments
• 9.6 Mead Johnson
  • 9.6.1 Overview
  • 9.6.2 Financial Performance
  • 9.6.3 Product Outlook
  • 9.6.4 Key Developments
• 9.7 Arcturus Therapeutics Inc
  • 9.7.1 Overview
  • 9.7.2 Financial Performance
  • 9.7.3 Product Outlook
  • 9.7.4 Key Developments
• 9.8 Horizon Therapeutics
  • 9.8.1 Overview
  • 9.8.2 Financial Performance
  • 9.8.3 Product Outlook
  • 9.8.4 Key Developments
• 9.9 Nestle
  • 9.9.1 Overview
  • 9.9.2 Financial Performance
  • 9.9.3 Product Outlook
  • 9.9.4 Key Developments
• 9.10 OrphanPacific Inc
  • 9.10.1 Overview
  • 9.10.2 Financial Performance
  • 9.10.3 Product Outlook
  • 9.10.4 Key Developments

10 KEY DEVELOPMENTS

• 10.1 Product Launches/Developments
• 10.2 Mergers and Acquisitions
• 10.3 Business Expansions
• 10.4 Partnerships and Collaborations

11 Appendix

• 11.1 Related Research