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ELEVIDYS (delandistrogene moxeparvovec-rokl)

Duchenne Muscular Dystrophy (DMD)

Marketed / Phase 3FDA Accelerated Approval; Confirmatory trial ongoing

Key Facts

Indication

Duchenne Muscular Dystrophy (DMD)

Phase

Marketed / Phase 3

Status

FDA Accelerated Approval; Confirmatory trial ongoing

Company

Sarepta Therapeutics

About Sarepta Therapeutics

Sarepta Therapeutics is a commercial-stage biotech leader with an urgent mission to engineer and deliver precision genetic medicines for rare diseases. The company has achieved significant commercial success with multiple FDA-approved RNA-based exon-skipping therapies for Duchenne muscular dystrophy (DMD) and the landmark approval of ELEVIDYS, a gene therapy for DMD. Its strategy leverages a deep, multi-modal pipeline, a hybrid manufacturing model for scale, and a global commercial footprint to solidify its position as a dominant force in rare disease therapeutics.

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Therapeutic Areas

Rare Neuromuscular Diseases Central Nervous System Diseases

Other Duchenne Muscular Dystrophy (DMD) Drugs

Drug	Company	Phase	Status
DT-DEC01	Dystrogen Therapeutics	Phase 1	Active (Dose Escalation)
Skeletal Muscle Regeneration Program	IPS HEART	Pre-clinical	Active
KYMBEE (deflazacort)	Upsher-Smith	Approved	Active
ALY688ER	Allysta Pharmaceuticals	Phase 1	Active
AVGN7 + AVGND	AAVogen	Pre-clinical	Active
Myomatrix Stabilization Program	Strykagen	Preclinical	Active
RIPPLE™ (implied)	SonoThera	Preclinical	Active
Vamorolone (AGAMREE)	ReveraGen BioPharma	Approved	Active
AGAMREE (vamorolone)	Catalyst Pharmaceuticals	Marketed	Commercial; Long-term safety monitored via SUMMIT Registry
EDG-5506	Edgewise Therapeutics	Phase 2	Active
SRP-5051 (vesleteplirsen)	Sarepta Therapeutics	Phase 2	Active (Next-gen PPMO)
EXONDYS 51 (eteplirsen)	Sarepta Therapeutics	Marketed	FDA Approved