듀셴형 근이영양증 : 기회 평가와 예측(-2030년)

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미국과 일본의 듀셴형 근이영양증(DMD) 시장 규모는 2020년에 6억 1,900만 달러를 기록하고, 12.0%의 CAGR로 성장하여 2030년에는 19억 달러가 될 것으로 예측됩니다. DMD 영역의 최대 미충족 수요는 질환 진행률, 특정 환자층에 대한 치료법 액세스, 치료비에 관한 것입니다. 2020-2030년간 DMD 유병자수는 미국에서 0.14% 증가, 일본에서 1.27% 감소할 것으로 예측됩니다. 미국과 일본의 현재 치료법은 엑손 스키핑 요법과 스테로이드제입니다.

듀셴형 근이영양증(DMD : Duchenne Muscular Dystrophy) 시장을 조사했으며, 질병 개요, SWOT 분석, 역학, 현재 치료 옵션, 미충족 수요와 기회, 파이프라인 평가, 연구개발 전략, 시장 전망 등 체계적인 정보를 제공합니다.

목차

목차

제1장 주요 요약

• 주요 요약

제2장 질병 개요

• 호산구성 식도염 개요
• DMD 병태생리학
• 진단 패러다임
• DMD SWOT 분석

제3장 역학

• DMD 진단된 유병률, 남아/남성, 0세-39세(2020-2030년)
• 돌연변이별 DMD 진단된 일반적인 증례, 남아/남성, 0세-39세(2020년)
• DMD 진단된 유병률의 정보 출처와 조사 방법
• 정보 출처와 조사 방법

제4장 현재 치료 옵션

• 치료 패러다임
• 시판 제품
• 제품 프로파일 : Exondys 51(eteplirsen)
• 제품 프로파일 : Amondys 45(casimersen)
• 제품 프로파일 : Vyondys 53(golodirsen)
• 제품 프로파일 : Viltepso(viltolarsen)
• 제품 프로파일 : Emflaza(deflazacort)
• 제품 프로파일 : prednisolone

제5장 미충족 수요와 기회

• DMD의 미충족 수요
• 질환 퇴행의 지연 또는 역행
• 비외래 환자의 치료로의 액세스
• 치료비

제6장 파이프라인 평가

• DMD 파이프라인 개요
• 후기 DMD 파이프라인 의약품의 임상시험 매핑
• 후기 파이프라인 에이전트
• 제품 프로파일 : PTC Therapeutics의 Translarna(ataluren)
• 제품 프로파일 : Pfizer의 fordadistrogene movaparvovec
• 제품 프로파일 : Italfarmaco의 givinostat
• 제품 프로파일 : FibroGen의 pamrevlumab
• 제품 프로파일 : Taiho Pharmaceutical의 pizuglanstat
• 제품 프로파일 : Sarepta Therapeutics의 delandistrogenemoxeparvovec
• 제품 프로파일 : Santhera Pharmaceuticals의 vamorolone
• 제품 프로파일 : Capricor Therapeutics의 CAP-1002

제7장 연구개발 전략

• DMD의 임상시험 설계 동향
• DMD의 거래 동향
• DMD의 임상시험 설계 동향

제8장 시장 전망

• DMD 질환 시장 예측
• 시장 성장 촉진요인과 장벽

제9장 부록

• 참고문헌
• 1차 조사 : KOL 정보
• 저자 소개

제10장 문의

Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin. Dystrophin is part of a group of proteins, which together form the dystrophin-associated protein complex (DPC). These proteins form a transmembrane scaffold that are crucial for the structure of the muscle tissue, protect muscle fibers from injury during muscle contraction and relaxation, and also serve integral functions in cell signalling. DMD is one of four conditions known as dystrophinopathies, and it is classified as the most severe end of the spectrum, with a lack of functional dystrophin. The disease is an X-linked recessive disorder mainly affecting males, with female carriers. While females are generally unaffected, some may show a milder phenotype ("manifesting carriers") such as cardiac changes, mild muscle weakness, and muscle cramping.

Lack of dystrophin in muscle cells causes them to be fragile and easily damaged. DMD is progressive and most affected individuals require a wheelchair by adolescence. Serious life-threatening conditions including cardiomyopathy and respiratory difficulties ultimately develop and lead to mortality of DMD patients between 19 and 25 years of age; however, GlobalData's primary research revealed that survival has increased in recent years to the 3rd decade of life.

Key Highlights

• Diagnosed prevalent cases of DMD are expected to increase by 0.14% in the US and decrease by 1.27% in Japan between 2020 and 2030.
• Current treatments in the US and Japan consist of exon-skipping therapies and steroids.
• Greatest unmet needs in the DMD space pertain to disease progression rate, access to treatment for specific patient segments, and cost of therapies.
• The DMD pipeline consists of 23 drugs across all stages of development in the US and Japan, with 8 products in late-stage development.
• The DMD market in the US and Japan was valued at $619.0M in 2020. The market is projected to grow at a Compound Annual Growth Rate (CAGR) of 12.0%, reaching $1.9B by 2030.

KEY QUESTIONS ANSWERED

• Which unmet needs are currently limiting DMD treatment in the US and Japan?
• What are the prospects of late-stage drugs anticipated to launch within the next decade?
• How are market dynamics expected to shift as a result of new treatment options?
• What are the main R&D trends in the DMD market and what is the outlook of clinical trials in the US and Japanese markets?
• What are the most important deal types and how have primary endpoints changed in clinical trial design?

Scope

• Overview of DMD including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
• Topline DMD market revenue, annual cost of therapy, and major pipeline product sales in the forecast period.
• Key topics covered include current treatment and pipeline therapies, unmet needs and opportunities, and the drivers and barriers affecting DMD therapeutics sales in the US and Japan.
• Pipeline analysis: Comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
• Analysis of the current and future market competition in the US and Japanese DMD market. Insightful review of key industry drivers, restraints and challenges.

Reasons to Buy

The report will enable you to -

• Develop and design your in-licensing and out-licensing strategies, using a detailed overview of current deals and technologies to identify companies with the highest potential.
• Develop business strategies by understanding the trends shaping and driving the DMD pharma market.
• Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact DMD pharma market in the future.
• Design your development strategy through a review of potential novel targets or combinations in the DMD space.
• Understand the challenges and strategies impacting the development of therapy agents in DMD preclinical studies and clinical trials.
• Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
• Organize your sales and marketing efforts by identifying the classes of therapies and indications that present maximum opportunities for consolidations, investments and strategic partnerships in the DMD space.

Table of Contents

Table of Contents

1 Executive Summary

• 1.1 Executive Summary

2 Disease Overview

• 2.1 Overview of Eosinophilic Esophagitis
• 2.2 Pathophysiology of DMD
• 2.3 Diagnostic Paradigm

2.4 DMD SWOT Analysis

3 Epidemiology

• 3.1 Diagnosed Prevalent Cases of DMD, Boys/Men, Ages 0-39 Years, 2020-2030
• 3.2 Diagnosed Prevalent Cases of DMD by Mutation, Boys/Men, Ages 0-39 Years, 2020
• 3.3 Sources and Methodology for Diagnosed Prevalence of DMD
• 3.4 Sources and Methodology

4 Current Treatment Options

• 4.1 Treatment Paradigm
• 4.2 Marketed Products
• 4.3 Product Profile: Exondys 51 (eteplirsen)
• 4.4 Product Profile: Amondys 45 (casimersen)
• 4.5 Product Profile: Vyondys 53 (golodirsen)
• 4.6 Product Profile: Viltepso (viltolarsen)
• 4.7 Product Profile: Emflaza (deflazacort)
• 4.8 Product Profile: prednisolone

5 Unmet Needs and Opportunities

• 5.1 Unmet Needs in DMD
• 5.1 Delay or Reverse Disease Regression
• 5.2 Access to Treatments for Non-ambulatory Patients
• 5.3 Cost of Therapies

6 Pipeline Assessment

• 6.1 DMD Pipeline Overview
• 6.2 Clinical Trial Mapping of Late-Stage DMD Pipeline Drugs
• 6.3 Late-Stage Pipeline Agents
• 6.4 Product Profile: PTC Therapeutics' Translarna (ataluren)
• 6.5 Product Profile: Pfizer's fordadistrogene movaparvovec
• 6.6 Product Profile: Italfarmaco's givinostat
• 6.7 Product Profile: FibroGen's pamrevlumab
• 6.8 Product Profile: Taiho Pharmaceutical's pizuglanstat
• 6.9 Product Profile: Sarepta Therapeutics' delandistrogene moxeparvovec
• 6.10 Product Profile: Santhera Pharmaceuticals' vamorolone
• 6.11 Product Profile: Capricor Therapeutics' CAP-1002

7 R&D Strategies

• 7.1 Trends in Clinical Trial Design in DMD
• 7.2 Trends in Deal-Making in DMD
• 7.3 Trends in Clinical Trial Design in DMD

8 Market Outlook

• 8.1 DMD Disease Market Forecast
• 8.2 Market Drivers and Barriers

9 Appendix

• 9.1 Bibliography
• 9.2 Primary Research: KOL Information
• 9.3 About the Authors

10 Contact Us