한글목차

약제 요약

아폴리포단백질 CIII(apoCIII) mRNA의 항센스 올리고뉴클레오티드 억제제인 WAYLIVRA(볼라네소센)는 가족성 부분 지방이상증(FPL) 치료제로 Ionis Pharmaceuticals가 자회사인 Akcea Therapeutics를 통해 개발 중입니다. 가족성 부분 지방이상증(FPL)은 신체의 여러 부위에서 체지방(지방조직)이 선택적이고 점진적으로 감소하는 희귀 유전질환으로, FPL 환자는 팔과 다리의 피하지방이 감소하는 경우가 많으며, 머리와 몸통의 지방이 감소할 수도 있고 그렇지 않을 수도 있습니다. 그렇지 않을 수도 있습니다. 이 약은 브라질에서 지방이상증 치료제로 시판 허가를 받았으며, 미국, 유럽, 일본에서 임상 개발 중입니다.

보라네소센은 아포 C-III mRNA에 결합하여 그 분해를 유도하고 아포 C-III 단백질의 번역을 억제하는 항센스 올리고뉴클레오타이드입니다. 일반적으로 아포 C-III는 트리글리세리드 대사와 카이로미크론의 간 클리어런스를 억제합니다. 아포 C-III의 번역을 차단함으로써 이러한 트리글리세라이드와 카이로미크론의 대사와 분해를 가능하게 합니다.

주요 7 시장(미국·독일·프랑스·이탈리아·스페인·영국·일본)의 지방이상증용 WAYLIVRA에 대해 조사분석했으며, 작용기서, 용법과 용량, 연구개발 활동에 관한 인사이트, 매출의 예측 등을 제공하고 있습니다.

목차

제1장 리포트의 서론

제2장 지방이상증에서의 WAYLIVRA 개요

• 제품의 상세
• 임상 개발
  • 임상 연구
  • 임상시험 정보
  • 안전성과 유효성
• 기타 개발 활동
• 제품 개요

제3장 경쟁 구도(출시 치료법)

제4장 경쟁 구도(후기 신치료법)

제5장 WAYLIVRA 시장 평가

• 지방이상증에서 WAYLIVRA의 시장 전망
• 주요 7 시장 분석
  • 주요 7 시장 지방이상증용 WAYLIVRA의 시장 규모
• 시장 분석 : 국가별
  • 미국의 지방이상증용 WAYLIVRA의 시장 규모
  • 독일의 지방이상증용 WAYLIVRA의 시장 규모
  • 영국의 지방이상증용 WAYLIVRA의 시장 규모

제6장 SWOT 분석

제7장 애널리스트의 견해

제8장 부록

제9장 DelveInsight의 서비스 내용

제10장 면책사항

제11장 DelveInsight 소개

제12장 리포트 구입 옵션

영문목차

"WAYLIVRA Market Size, Forecast, and Emerging Insight - 2032" report provides comprehensive insights about WAYLIVRA for lipodystrophy in the seven major markets. A detailed picture of the WAYLIVRA for lipodystrophy in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan for the study period 2019 -2032 is provided in this report along with a detailed description of the WAYLIVRA for lipodystrophy. The report provides insights about mechanism of action, dosage and administration, as well as research and development including regulatory milestones, along with other developmental activities. Further, it also consists of future market assessments inclusive of the WAYLIVRA market forecast analysis for lipodystrophy in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in lipodystrophy.

Drug Summary:

WAYLIVRA (volanesorsen), an antisense oligonucleotide inhibitor of apolipoprotein CIII (apoCIII) mRNA, is being developed by Ionis Pharmaceuticals through its subsidiary company, Akcea Therapeutics to treat familial partial lipodystrophy (FPL). Familial partial lipodystrophy (FPL) is a rare genetic disorder characterized by selective, progressive body fat loss (adipose tissue) from various body areas. Individuals with FPL often have reduced subcutaneous fat in the arms and legs, and the head and trunk regions may or may not have fat loss. The drug received marketing approval in Brazil for the treatment of lipodystrophy and is investigated under clinical development in the US, Europe and Japan.

Volanesorsen is an antisense oligonucleotide that binds to apoC-III mRNA, leading to its degradation and preventing the translation of apoC-III protein. Normally, apoC-III would inhibit triglyceride metabolism and hepatic clearance of chylomicrons. Preventing translation of apoC-III allows for the metabolism and breakdown of these triglycerides and chylomicrons.

Scope of the Report:

The report provides insights into:

• A comprehensive product overview including the WAYLIVRA description, mechanism of action, dosage and administration, research and development activities in lipodystrophy.
• Elaborated details on WAYLIVRA regulatory milestones and other development activities have been provided in this report.
• The report also highlights the WAYLIVRA research and development activities in lipodystrophy across the United States, Europe and Japan.
• The report also covers the patents information with expiry timeline around WAYLIVRA.
• The report contains forecasted sales of WAYLIVRA for lipodystrophy till 2032.
• Comprehensive coverage of the late-stage emerging therapies for lipodystrophy.
• The report also features the SWOT analysis with analyst views for WAYLIVRA in lipodystrophy.

Methodology:

The report is built using data and information sourced primarily from internal databases, primary and secondary research and in-house analysis by DelveInsight's team of industry experts. Information and data from the secondary sources have been obtained from various printable and nonprintable sources like search engines, news websites, global regulatory authorities websites, trade journals, white papers, magazines, books, trade associations, industry associations, industry portals and access to available databases.

WAYLIVRA Analytical Perspective by DelveInsight

• In-depth WAYLIVRA Market Assessment

This report provides a detailed market assessment of WAYLIVRA for lipodystrophy in the seven major markets, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. This segment of the report provides forecasted sales data from 2024 to 2032.

• WAYLIVRA Clinical Assessment

The report provides the clinical trials information of WAYLIVRA for lipodystrophy covering trial interventions, trial conditions, trial status, start and completion dates.

Report Highlights:

• In the coming years, the market scenario for lipodystrophy is set to change due to the extensive research and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies are developing therapies that focus on novel approaches to treat/improve the disease condition, assess challenges, and seek opportunities that could influence WAYLIVRA dominance.
• Other emerging products for lipodystrophy are expected to give tough market competition to WAYLIVRA and launch of late-stage emerging therapies in the near future will significantly impact the market.
• A detailed description of regulatory milestones, and developmental activities, provide the current development scenario of WAYLIVRA in lipodystrophy.
• Our in-depth analysis of the forecasted sales data of WAYLIVRA from 2024 to 2032 will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the WAYLIVRA in lipodystrophy.

Key Questions:

• What is the product type, route of administration and mechanism of action of WAYLIVRA?
• What is the clinical trial status of the study related to WAYLIVRA in lipodystrophy and study completion date?
• What are the key collaborations, mergers and acquisitions, licensing and other activities related to the WAYLIVRA development?
• What are the key designations that have been granted to WAYLIVRA for lipodystrophy?
• What is the forecasted market scenario of WAYLIVRA for lipodystrophy?
• What are the forecasted sales of WAYLIVRA in the seven major countries, including the United States, Europe (Germany, France, Italy, Spain, and the United Kingdom), and Japan?
• What are the other emerging products available and how are these giving competition to WAYLIVRA for lipodystrophy?
• Which are the late-stage emerging therapies under development for the treatment of lipodystrophy?

Table of Contents

1. Report Introduction

2. WAYLIVRA Overview in lipodystrophy

• 2.1. Product Detail
• 2.2. Clinical Development
  • 2.2.1. Clinical studies
  • 2.2.2. Clinical trials information
  • 2.2.3. Safety and efficacy
• 2.3. Other Developmental Activities
• 2.4. Product Profile

3. Competitive Landscape (Marketed Therapies)

4. Competitive Landscape (Late-stage Emerging Therapies)

5. WAYLIVRA Market Assessment

• 5.1. Market Outlook of WAYLIVRA in lipodystrophy
• 5.2. 7MM Analysis
  • 5.2.1. Market Size of WAYLIVRA in the 7MM for lipodystrophy
• 5.3. Country-wise Market Analysis
  • 5.3.1. Market Size of WAYLIVRA in the United States for lipodystrophy
  • 5.3.2. Market Size of WAYLIVRA in Germany for lipodystrophy
  • 5.3.3. Market Size of WAYLIVRA in France for lipodystrophy
  • 5.3.4. Market Size of WAYLIVRA in Italy for lipodystrophy
  • 5.3.5. Market Size of WAYLIVRA in Spain for lipodystrophy
  • 5.3.6. Market Size of WAYLIVRA in the United Kingdom for lipodystrophy
  • 5.3.7. Market Size of WAYLIVRA in Japan for lipodystrophy

6. SWOT Analysis

7. Analysts' Views

8. Appendix

• 8.1. Bibliography
• 8.2. Report Methodology

9. DelveInsight Capabilities

10. Disclaimer

11. About DelveInsight

12. Report Purchase Options