세계의 OTC 결핍증 시장 보고서(2025년)
Ornithine Transcarbamylase Deficiency (OTC Deficiency) Global Market Report 2025
상품코드 : 1730976
리서치사 : The Business Research Company
발행일 : On Demand Report
페이지 정보 : 영문 175 Pages
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한글목차

OTC 결핍증 시장 규모는 향후 몇 년간 강력한 성장이 전망될 예정입니다. 예측 기간의 성장은 연구와 임상시험 증가, 치료에 대한 환자 접근의 개선 등에 기인한다고 생각됩니다. 이 기간의 주요 동향으로는 유전자 치료와 유전자 편집 기술의 발전, 효소 보충 요법의 개발, 공동 연구의 대처나 파트너십, 진단 툴의 기술 혁신, 희소 질병 치료제에 대한 정부의 자금 원조나 우대 조치 등을 들 수 있습니다.

유전자 치료에 대한 수요 증가는 OTC 결핍증 시장의 성장을 가속할 것으로 예측됩니다. 성질환의 유병률 증가와 관련이 있으며, 그 중 대부분은 치료 옵션이 거의 없기 때문에 유전자 치료는 단순히 증상을 완화할 뿐만 아니라 증상의 근본 원인을 다루는 매력적인 접근이 되고 있습니다. 사이클을 저해하는 유전자 변이를 수정함으로써, 대사 기능을 회복시켜, 유해한 암모니아의 축적을 막는 표적 솔루션을 제공해, 유전자 치료의 가능성을 부조로 하고 있습니다. 예를 들어, 2024년 1월 미국 유전자 및 세포 치료학회는 3상 유전자 치료의 수가 2023년 4분기에 10% 증가했으며, 이는 2022년 3분기 이후 처음으로 증가한 것이라고 보고했습니다.

OTC 결핍증 시장에 관여하는 기업은 치료 성과를 개선하고 OTC 결핍증의 유전적 기반을 다루기 위해 mRNA 치료제 등의 혁신적인 치료에 점점 주력하고 있습니다. mRNA 치료제는 메신저 RNA를 사용하여 세포가 유전 질환, 감염, 암을 포함한 질병을 치료할 수 있는 특정 단백질을 생산하도록 지시합니다. 예를 들어, 2023년 6월, 미국에 본사를 둔 생명공학 회사인 아크투러스 테라퓨틱스 홀딩스는 오르니틴 트랜스카바밀라제(OTC) 결핍증 치료를 위한 mRNA 치료제 후보물질인 ARCT-810에 대해 FDA로부터 패스트트랙 지정을 받았습니다. 이 지정은 개발 및 검토 프로세스를 가속화하여 FDA와 더 자주 상호 작용하고 우선 검토 등의 혜택을 제공하는 것을 목표로 합니다.

목차

제1장 주요 요약

제2장 시장 특징

제3장 시장 동향과 전략

제4장 시장 - 거시경제 시나리오 금리, 인플레이션, 지정학, 신형 코로나 바이러스 감염의 영향과 회복이 시장에 미치는 영향을 포함한 거시경제 시나리오

제5장 세계의 성장 분석과 전략 분석 프레임워크

제6장 시장 세분화

제7장 지역별/국가별 분석

제8장 아시아태평양 시장

제9장 중국 시장

제10장 인도 시장

제11장 일본 시장

제12장 호주 시장

제13장 인도네시아 시장

제14장 한국 시장

제15장 서유럽 시장

제16장 영국 시장

제17장 독일 시장

제18장 프랑스 시장

제19장 이탈리아 시장

제20장 스페인 시장

제21장 동유럽 시장

제22장 러시아 시장

제23장 북미 시장

제24장 미국 시장

제25장 캐나다 시장

제26장 남미 시장

제27장 브라질 시장

제28장 중동 시장

제29장 아프리카 시장

제30장 경쟁 구도와 기업 프로파일

제31장 기타 주요 기업 및 혁신 기업

제32장 세계 시장 경쟁 벤치마킹과 대시보드

제33장 주요 인수합병(M&A)

제34장 최근 시장 동향

제35장 시장의 잠재력이 높은 국가, 부문, 전략

제36장 부록

KTH
영문 목차

영문목차

Ornithine transcarbamylase (OTC) deficiency is a rare genetic disorder caused by mutations in the OTC gene, leading to a lack of the ornithine transcarbamylase enzyme, which is crucial for the urea cycle to remove ammonia from the body. This deficiency results in hyperammonemia, which can cause symptoms such as vomiting, lethargy, seizures, and in severe cases, coma, particularly in newborns.

The main product types for treating OTC deficiency include DTX-301, SEL-313, SHP-641, and PRX-OTC. DTX-301 is an investigational AAV8-based gene therapy designed to deliver a functional OTC gene to liver cells for treating OTC deficiency. Treatment options include gene therapy, liver transplant, dietary management, and other therapies. Diagnosis is performed using genetic testing and ammonia level testing. The end users of OTC deficiency treatments include hospitals, specialty clinics, research institutes, and other healthcare providers.

The ornithine transcarbamylase deficiency (OTC deficiency) market research report is one of a series of new reports from The Business Research Company that provides ornithine transcarbamylase deficiency (OTC deficiency) market statistics, including the ornithine transcarbamylase deficiency (OTC deficiency) industry global market size, regional shares, competitors with the ornithine transcarbamylase deficiency (OTC deficiency) market share, detailed ornithine transcarbamylase deficiency (OTC deficiency) market segments, market trends, and opportunities, and any further data you may need to thrive in the ornithine transcarbamylase deficiency (OTC deficiency) industry. This ornithine transcarbamylase deficiency (OTC deficiency) market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The ornithine transcarbamylase deficiency (OTC deficiency) market size has grown strongly in recent years. It will grow from $0.83 billion in 2024 to $0.89 billion in 2025 at a compound annual growth rate (CAGR) of 7.1%. The growth during the historic period can be attributed to increased awareness of rare metabolic disorders, advancements in genetic screening and diagnostics, improvements in neonatal screening programs, the rising prevalence of genetic testing for rare diseases, and enhanced neonatal screening initiatives.

The ornithine transcarbamylase deficiency (OTC deficiency) market size is expected to see strong growth in the next few years. It will grow to $1.16 billion in 2029 at a compound annual growth rate (CAGR) of 6.8%. The growth during the forecast period can be attributed to increasing awareness and diagnosis, the rising prevalence of urea cycle disorders, a growing focus on rare disease treatments, more research and clinical trials, and improved patient access to treatments. Key trends during this period include advancements in genetic therapies and gene editing technologies, the development of enzyme replacement therapies, collaborative research initiatives and partnerships, technological innovations in diagnostic tools, and government funding and incentives for orphan drugs.

The increasing demand for gene therapies is expected to drive the growth of the ornithine transcarbamylase deficiency market. Gene therapy is a medical technique that involves altering a person's genetic material to treat or prevent diseases. The growing demand for these therapies is linked to the rising prevalence of genetic disorders, many of which have few treatment options, making gene therapy an appealing approach to address the root cause of the condition rather than just alleviating symptoms. Ornithine transcarbamylase deficiency highlights the potential of gene therapy by correcting the genetic mutation that disrupts the urea cycle, offering a targeted solution that restores metabolic function and prevents harmful ammonia buildup. For example, in January 2024, the American Society of Gene & Cell Therapy reported that the number of gene therapies in Phase III increased by 10% in Q4 2023, the first increase since Q3 2022. This growth in gene therapy demand is thus contributing to the expansion of the ornithine transcarbamylase deficiency market.

Companies involved in the ornithine transcarbamylase deficiency market are increasingly focusing on innovative therapies such as mRNA therapeutics to improve treatment outcomes and address the genetic basis of OTC deficiency. mRNA therapeutics use messenger RNA to instruct cells to produce specific proteins that can treat diseases, including genetic disorders, infections, and cancers. For instance, in June 2023, Arcturus Therapeutics Holdings Inc., a U.S.-based biotech firm, received Fast Track Designation from the FDA for its mRNA therapeutic candidate, ARCT-810, for treating ornithine transcarbamylase (OTC) deficiency. This designation aims to accelerate the development and review process, providing benefits such as more frequent interactions with the FDA and priority review. ARCT-810 is designed to help OTC deficiency patients produce a functional OTC enzyme in their liver cells, addressing the underlying cause of the condition and potentially improving their quality of life.

In November 2023, Zevra Therapeutics Inc., a U.S.-based pharmaceutical company, acquired Acer Therapeutics Inc. for $91 million. This acquisition strengthens Zevra Therapeutics' position in the rare disease sector, expanding its portfolio and increasing its revenue potential. Acer Therapeutics specializes in developing therapies for ornithine transcarbamylase deficiency, further enhancing Zevra's focus in this therapeutic area.

Major players in the ornithine transcarbamylase deficiency (otc deficiency) market are Thermo Fisher Scientific, Novartis AG, GlaxoSmithKline plc, Roche Holding AG, Eli Lilly and Company, Amgen Inc., Merck & Co. Inc, Biogen Idec, Bausch Health Companies Inc, Alexion Pharmaceuticals, Sobi, BioMarin Pharmaceutical Inc, Glenmark Pharmaceuticals Ltd., Nutricia, Vertex Pharmaceuticals Incorporated, Ultragenyx Pharmaceutical Inc, Arcturus Therapeutics, Regenxbio, OrphanPacific Inc, Ucyclyd Pharma Inc.

North America was the largest region in the ornithine transcarbamylase deficiency (OTC Deficiency) market in 2024. Asia-pacific is expected to be the fastest-growing region in the forecast period. The regions covered in ornithine transcarbamylase deficiency report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the ornithine transcarbamylase deficiency market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The ornithine transcarbamylase deficiency market consists of revenues earned by entities by providing services such as genetic testing & diagnosis services, dietary management & nutrition services, clinical trials & research programs, medical consultation and genetic counseling. The market value includes the value of related goods sold by the service provider or included within the service offering. The ornithine transcarbamylase deficiency (OTC deficiency) market also includes sales of amino acid and protein supplements, gene therapies, nitrogen scavenging drugs, diagnostic and monitoring tests. Values in this market are 'factory gate' values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

Ornithine Transcarbamylase Deficiency (OTC Deficiency) Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.

This report focuses on ornithine transcarbamylase deficiency (otc deficiency) market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.

Reasons to Purchase

Where is the largest and fastest growing market for ornithine transcarbamylase deficiency (otc deficiency) ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The ornithine transcarbamylase deficiency (otc deficiency) market global report from the Business Research Company answers all these questions and many more.

The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.

The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.

Scope

Table of Contents

1. Executive Summary

2. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Characteristics

3. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Trends And Strategies

4. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market - Macro Economic Scenario Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics And Covid And Recovery On The Market

5. Global Ornithine Transcarbamylase Deficiency (OTC Deficiency) Growth Analysis And Strategic Analysis Framework

6. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Segmentation

7. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Regional And Country Analysis

8. Asia-Pacific Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

9. China Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

10. India Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

11. Japan Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

12. Australia Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

13. Indonesia Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

14. South Korea Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

15. Western Europe Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

16. UK Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

17. Germany Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

18. France Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

19. Italy Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

20. Spain Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

21. Eastern Europe Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

22. Russia Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

23. North America Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

24. USA Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

25. Canada Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

26. South America Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

27. Brazil Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

28. Middle East Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

29. Africa Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

30. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Competitive Landscape And Company Profiles

31. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Other Major And Innovative Companies

32. Global Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market Competitive Benchmarking And Dashboard

33. Key Mergers And Acquisitions In The Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

34. Recent Developments In The Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market

35. Ornithine Transcarbamylase Deficiency (OTC Deficiency) Market High Potential Countries, Segments and Strategies

36. Appendix

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