TheTechno Optimist.

ElevidysSarepta Therapeutics—FDA adds boxed warning to Elevidys for acute liver injury; non-ambulatory indication removedElevidysSarepta Therapeutics—Three-year Phase 3 EMBARK data shows Elevidys slows disease progression by 70-73% in ambulatory Duchenne patientsSarepta Therapeutics—Michael Severino appointed CEO of Sarepta Therapeutics

Elevidys

by Sarepta TherapeuticsUS

AAV gene therapy delivering a shortened dystrophin gene to muscle cells. Approved for ambulatory Duchenne children 4+ with confirmed mutations.

DeployStage 4 of 5

FDA approved for ambulatory Duchenne; slows progression 70%. Non-ambulatory indication removed.

Updated Jan 2026Checked 10 Oct0 updates this week

Milestones

Next · Commercial scale-up and patient access
  1. FDA approval for ambulatory Duchenne (Elevidys)Jun 2024Complete.
  2. FDA removes non-ambulatory indication after liver safety eventsNov 2025Complete.
  3. Three-year Phase 3 EMBARK data shows 70% disease progression slowingJan 2026Complete.
  4. Commercial scale-up and patient accessTarget 2026Current milestone.

Most important updates

Upcoming

  1. 2026Commercial scale-up and patient access (next)
  2. 2027Long-term Phase 3 efficacy and safety data
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