全球鳥胺酸氨甲醯基轉移酶缺乏症市場規模：依產品類型、最終用戶、區域範圍和預測

鳥胺酸氨甲醯基轉移酶缺乏症的市場規模及預測

2024年鳥胺酸氨甲醯基轉移酶缺乏症市場規模價值 6.8874億美元，預計到2031年將達到 9.3033億美元，2024年至2031年的年複合成長率為 3.83%。

鳥胺酸氨甲醯基轉移酶缺乏症市場的主要參與者針對家庭和醫療保健專業人士開展的越來越多的宣傳活動推動鳥胺酸氨甲醯基轉移酶缺乏症治療市場的發展。全球鳥胺酸氨甲醯基轉移酶缺乏症市場報告對市場進行了全面的評估。它對關鍵細分市場、趨勢、市場驅動因素、限制因素、競爭格局以及在市場中發揮關鍵作用的因素進行了全面的分析。

全球鳥胺酸氨甲醯基轉移酶缺乏症市場的定義

遺傳性疾病鳥胺酸氨甲醯基轉移酶缺乏症會導致氨在血液中積聚。當人體分解蛋白質時，會產生氨，過量的氨可能有害。過量的氨對神經系統的損害尤其大。鳥胺酸氨甲醯基轉移酶缺乏症可發生在任何年齡。生命最初幾天是最嚴重的。這種新生兒發病形式在男性中比在女性中更常見。患有新生兒鳥胺酸氨甲醯基轉移酶缺乏症的新生兒可能會表現得昏睡不醒，不願進食，呼吸頻率和體溫無法控制地升高。氨對神經系統特別有毒性，因此鳥氨酸轉氨甲酰酶缺乏會導致神經系統問題以及肝臟疾病。

鳥胺酸氨甲醯基轉移酶缺乏症的發生率估計為 1/14,000 至 1/77,000。患有新生兒發病疾病的患者更有可能被納入這些估計值，因為患有晚期疾病的患者不太可能尋求醫療協助。 OTC 缺乏症是一種透過 X 染色體遺傳的遺傳性疾病。男性比女性更容易罹患 OTC 缺乏症，但只有男性才會出現全部症狀。男性的症狀通常出現在嬰兒期的最初幾天，但晚髮型 OTC 缺乏症也可能出現在以後的生活中。

約20%的女性帶因者會出現輕微的疾病症狀，只有一小部分人在兒童時期會出現嚴重的症狀。多學科團隊包括兒科醫生、遺傳學家、營養學家、神經科醫生和內科醫生。 OTC缺乏症的治療目標是防止過量氨的產生或去除過量的氨。 OTC 缺乏症患者的飲食限制目的是盡量減少蛋白質的攝入，以避免氨的蓄積。患有 OTC 缺乏症的嬰兒需要接受高熱量、低蛋白質且含有必需胺基酸的飲食。幫助消除體內氮的藥物也用於治療非處方藥缺乏症患者。

全球鳥胺酸氨甲醯基轉移酶缺乏症市場概況

患有鳥胺酸氨甲醯基轉移酶缺乏症的COVID-19 患者風險較高。此類患者因該疾病而患有先前存在的合併症。患有鳥胺酸氨甲醯基轉移酶缺乏症的患者中 COVID-19 病例的驚人激增推動該行業的發展。患有糖尿病、高血壓和腎病變等其他合併症的患者特別容易感染 COVID-19。有證據表明，對非處方藥敏感的患者更容易受到傷害。這就是為什麼醫護人員不斷努力增加基本藥物供應的原因。

鳥胺酸氨甲醯基轉移酶缺乏症與許多嬰兒猝死症候群（SIDS）有關。 COVID-19 疫苗為鳥胺酸氨甲醯基轉移酶缺乏症市場中的公司創造收入前景。 COVID-19 疫苗為鳥胺酸氨甲醯基轉移酶缺乏症市場中的公司提供了收入前景，並有望在疫情期間維持需求、最大化公司的利潤率並維持穩定的現金流。鳥胺酸氨甲醯基轉移酶缺乏症市場正受益於這些原因。此外，鳥胺酸氨甲醯基轉移酶缺乏症市場的主要公司針對家庭和醫療保健專業人員開展的提高認識的計劃支持鳥胺酸氨甲醯基轉移酶缺乏症治療市場的成長。

預計全球鳥胺酸氨甲醯基轉移酶缺乏症市場將受到患有鳥氨酸轉氨甲酰酶（OTC）缺乏症的患者數量的增加的推動。推動全球鳥胺酸氨甲醯基轉移酶缺乏症市場的關鍵因素是引進針對鳥氨酸轉氨甲酰酶（OTC）缺乏症治療的新療法、針對 OTC 缺乏症治療的優惠償付政策以及多個國際和地區組織提供的各種患者支持計劃。阻礙市場成長的因素之一是缺乏非處方藥缺陷治療的預後和診斷的資料。由於新冠肺炎疫情的影響，許多國家治療非處方藥缺陷所需原料的供應鏈已經中斷。

另外，受疫情影響，藥廠停產，導致醫藥業成長下滑。預計這將對鳥胺酸氨甲醯基轉移酶缺乏症治療市場產生重大影響。全球鳥胺酸氨甲醯基轉移酶缺乏症市場的關鍵參與者收購新興公司以擴大其產品範圍。公司喜歡購買新創企業的研發部門並共同開發突破性技術。這項策略為企業提供了利用製造業和商業化其產品的巨大機會。

市場吸引力

市場吸引力有助於獲取有關領先全球鳥胺酸氨甲醯基轉移酶缺乏症市場的地區的資訊。涵蓋了推動特定區域產業成長的關鍵影響因素。

波特五力模型

波特五力模型是用來理解產業內競爭對手行為和策略定位的架構。波特五力模型可用於評估全球鳥胺酸氨甲醯基轉移酶缺乏症市場的競爭格局，衡量特定產業的吸引力，並評估投資潛力。

目錄

第1章 全球鳥胺酸氨甲醯基轉移酶缺乏症市場簡介

• 市場概況
• 研究範圍
• 先決條件

第2章 執行摘要

第3章 VERIFIED MARKET RESEARCH的研究方法

• 資料探勘
• 驗證
• 主要來源
• 資料來源列表

第4章 全球鳥胺酸氨甲醯基轉移酶缺乏症市場展望

• 概述
• 市場動態
  • 促進因素
  • 抑制因素
  • 機會
• 波特五力模型
• 價值鏈分析

第5章 全球鳥胺酸氨甲醯基轉移酶缺乏症市場（依產品類型）

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

第6章 全球鳥胺酸氨甲醯基轉移酶缺乏症市場（依最終用戶）

• 概述
• 醫院藥房
• 零售藥局
• 網路藥局

第7章 全球鳥胺酸氨甲醯基轉移酶缺乏症市場（依地區）

• 概述
• 北美洲
  • 美國
  • 加拿大
  • 墨西哥
• 歐洲
  • 德國
  • 英國
  • 法國
  • 其他歐洲國家
• 亞太地區
  • 中國
  • 日本
  • 印度
  • 其他亞太地區
• 世界其他地區
  • 拉丁美洲
  • 中東和非洲

第8章 全球鳥胺酸氨甲醯基轉移酶缺乏症市場的競爭格局

• 概述
• 各公司的市場排名
• 主要發展策略

第9章 公司簡介

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

第10章 重大進展

• 產品發布/開發
• 合併和收購
• 業務擴展
• 夥伴關係和合作關係

第11章 附錄

• 相關研究

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