Duchenne Muscular Dystrophy

Description

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?

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

Duchenne Muscular Dystrophy - KOL Insight