듀셴형 근이영양증(DMD) 치료의 최신 진보란 무엇인가? Agamree, Elevidys, Duvyzat와 같은 새로운 치료법은 DMD 치료의 미래를 어떻게 좌우하는가? 듀셴형 근이영양증 치료제의 최신 동향 및 전망에 대한 주요 KOL 인사이트을 정리하고, 주요 치료제의 유효성·안전성·가능성, 최근 DMD 치료 환경의 변동 등에 관한 인사이트을 제공합니다.
이 보고서의 조사 대상 주요 브랜드
- Emflaza(deflazacort)
- Agamree(vamorolone)
- Exondys 51(eteplirsen)
- Vyondys 53(golodirsen)
- Viltepso(viltolarsen)
- Amondys 45(casimersen)
- Translarna(ataluren)
- Elevidys(SRP-9001/delandistrogene moxeparvovec)
- fordadistrogene movaparvovec(PF-06939926)
- RGX-202
- GNT0004
- SGT-003
|
- deramiocel(CAP-1002)
- Duvyzat(givinostat)
- sevasemten(EDG-5506)
- vesleteplirsen(SRP-5051)
- DYNE-251
- PNG-EDO51
- BMN 351
- WVE-N531
- AOC 1044
- brogidersen
- ATL1102
|
기업
Roche, Pfizer, BioMarin Pharmaceutical, Capricor, PTC Therapeutics, Sarepta, Nippon Shinyaku, Solid Biosciences, Genethon, Italfarmaco, Santhera Pharmaceuticals, Wave Life Sciences, Edgewise Therapeutics, Regenxbio, Dyne Therapeutics, Avidity Biosciences, Catalyst Pharmaceuticals, NS Pharma, ITF Therapeutics, PepGen, Percheron Therapeutics.
주요 질문에 답변합니다. :
- 현재 의사는 DMD를 어떻게 치료하고 있는가?
- KOL은 현재 이용 가능한 치료 옵션이 만족스럽다고 생각하고 있는가?
- DMD 치료법은 환자 유형(연령, 병기 등)에 따라 어떻게 다른가?
- DMD에 관한 현재 주요 미충족 요구란 무엇인가? 또한 이용 가능한/향후 치료법은 환자 유형에 따라 어떻게 다른지?
- DMD 치료법에 관하여 지불자에게 어떤 과제가 있는가?
- 유전자 치료 및 엑손 스키핑 치료는 현재 치료를 대신하는가?
- 새로운 치료법의 장기적 과제는 무엇인가?
- 차세대 치료는 현재 DMD 치료를 대폭 개선하는가?
- 병용 전략은 DMD 향후 치료 전망을 어떻게 좌우하는가?
목차
개요
치료 알고리즘
조사 목적
출시 치료제
- 스테로이드 치료제
- Emflaza(deflazacort, PTC Therapeutics)
- 차세대 스테로이드 치료제
- Agamree(vamorolone, Santhera Pharmaceuticals/Catalyst Pharmaceuticals)
- 기타 경구 치료제
- Translarna(ataluren, PTC Therapeutics)
- 엑손 스킵 치료제
- Exondys 51(eteplirsen, Sarepta Therapeutics)
- Vyondys 53(golodirsen, Sarepta Therapeutics)
- Viltepso(viltolarsen, Nippon Shinyaku/NS Pharma)
- Amondys 45(카시메르센, Sarepta Therapeutics)
- 유전자 치료
- Elevidys(SRP-9001/delandistrogene moxeparvovec, Sarepta Therapeutics/Roche)
파이프라인 제품(제III상· 제II상)
- 유전자 치료제
- Fordadistrogene movaparvovec(PF-06939926, Pfizer)
- RGX-202(REGENXBIO)
- GNT0004(Genethon/Sarepta Therapeutics)
- SGT-003(Solid Biosciences)
- 경구소분자
- Duvyzat(givinostat, Italfarmaco/ITF Therapeutics)
- Sevasemten(EDG-5506, Edgewise Therapeutics)
- 세포치료
- Deramiocel(CAP-1002; Capricor Therapeutics)
- 차세대 엑손 스키핑 요법
- Vesleteplirsen(SRP-5051, Sarepta Therapeutics)
- DYNE-251(Dyne Therapeutics)
- PGN-EDO51(PepGen)
- BMN 351(BioMarin Pharmaceutical)
- WVE-N531(Wave Life Sciences)
- AOC 1044(Avidity Biosciences)
- Brogidirsen(NS-089/NCNP-02, Nippon Shinyaku/NS Pharma)
- 기타 올리고뉴클레오티드
- ATL1102(Percheron Therapeutics)
DMD의 향후 치료 동향
- 주요 인사이트 요약
- 유전자 치료의 개선은(그리고 아마 유전자 편집도) 향후 3-5년에 DMD 치료의 매우 중요한 요소가 되며, 병용요법도 큰 역할을 담당하게 된다.
KOL 상세
KSA
What are the latest advancements in Duchenne Muscular Dystrophy (DMD) treatments? How are new therapies like Agamree, Elevidys and Duvyzat shaping the future of DMD care? Insights from leading KOLs provide a detailed analysis of current and emerging treatments, featuring expert opinions from leading KOLs. Gain insights into the efficacy, safety, and potential of these therapies, and understand the evolving treatment landscape for DMD.
Key brands covered in this report:
- Emflaza (deflazacort)
- Agamree (vamorolone)
- Exondys 51 (eteplirsen)
- Vyondys 53 (golodirsen)
- Viltepso (viltolarsen)
- Amondys 45 (casimersen)
- Translarna (ataluren)
- Elevidys (SRP-9001/delandistrogene moxeparvovec)
- fordadistrogene movaparvovec (PF-06939926)
- RGX-202
- GNT0004
- SGT-003
|
- deramiocel (CAP-1002)
- Duvyzat (givinostat)
- sevasemten (EDG-5506)
- vesleteplirsen (SRP-5051)
- DYNE-251
- PNG-EDO51
- BMN 351
- WVE-N531
- AOC 1044
- brogidersen
- ATL1102
|
Companies:
Roche, Pfizer, BioMarin Pharmaceutical, Capricor, PTC Therapeutics, Sarepta, Nippon Shinyaku, Solid Biosciences, Genethon, Italfarmaco, Santhera Pharmaceuticals, Wave Life Sciences, Edgewise Therapeutics, Regenxbio, Dyne Therapeutics, Avidity Biosciences, Catalyst Pharmaceuticals, NS Pharma, ITF Therapeutics, PepGen, Percheron Therapeutics.
Key questions answered:
- How do physicians currently treat DMD?
- Do KOLs think the currently available treatment options are satisfactory?
- How does DMD treatment vary by patient type (age, disease stage, etc.)?
- What are the primary unmet needs in DMD today, and how will the various available and future treatments align with different patient types?
- What payer challenges exist in treating DMD?
- Will gene or exon-skipping therapies displace current treatments?
- What are the long-term challenges of emerging treatments?
- Will next-generation therapies significantly improve current DMD treatments?
- How might combination strategies shape the future treatment landscape for DMD?
Table of Contents
Executive summary
Treatment algorithm
Research objectives
Marketed therapies
- Steroid treatments
- Emflaza (deflazacort; PTC Therapeutics)
- Next-generation steroid treatments
- Agamree (vamorolone; Santhera Pharmaceuticals/Catalyst Pharmaceuticals)
- Other oral treatments
- Translarna (ataluren; PTC Therapeutics)
- Exon-skipping treatments
- Exondys 51 (eteplirsen; Sarepta Therapeutics)
- Vyondys 53 (golodirsen; Sarepta Therapeutics)
- Viltepso (viltolarsen; Nippon Shinyaku/NS Pharma)
- Amondys 45 (casimersen; Sarepta Therapeutics)
- Gene therapies
- Elevidys (SRP-9001/delandistrogene moxeparvovec; Sarepta Therapeutics/Roche)
Pipeline products (Phase III and Phase II)
- Gene therapies
- Fordadistrogene movaparvovec (PF-06939926; Pfizer)
- RGX-202 (REGENXBIO)
- GNT0004 (Genethon/Sarepta Therapeutics)
- SGT-003 (Solid Biosciences)
- Small oral molecules
- Duvyzat (givinostat; Italfarmaco/ITF Therapeutics)
- Sevasemten (EDG-5506; Edgewise Therapeutics)
- Cell therapies
- Deramiocel (CAP-1002; Capricor Therapeutics)
- Next-generation exon-skipping therapies
- Vesleteplirsen (SRP-5051; Sarepta Therapeutics)
- DYNE-251 (Dyne Therapeutics)
- PGN-EDO51 (PepGen)
- BMN 351 (BioMarin Pharmaceutical)
- WVE-N531 (Wave Life Sciences)
- AOC 1044 (Avidity Biosciences)
- Brogidirsen (NS-089/NCNP-02; Nippon Shinyaku/NS Pharma)
- Other oligonucleotides
- ATL1102 (Percheron Therapeutics)
Future treatment trends in DMD
- Key insights summary
- Improved gene therapies, and perhaps even gene editing, will be pivotal to DMD treatment over the next three to five years, with a larger role also for combination approaches
KOL details
- KOLs from the USA
- KOLs from Europe
