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A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4

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Trial Profile

A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4

Status: Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 21 Apr 2025

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At a glance

  • Drugs Bidridistrogene xeboparvovec (Primary) ; Glucocorticoids
  • Indications Limb girdle muscular dystrophies
  • Focus Registrational; Therapeutic Use
  • Acronyms EMERGENE
  • Sponsors Sarepta Therapeutics

    • Most Recent Events

    • 15 Apr 2025 According to Sarepta Therapeutics media release, the company to submit a BLA to the U.S. FDA in the second half of 2025. Data expected in mid 2025.
    • 04 Mar 2025 Planned primary completion date changed from 31 Jan 2025 to 4 Mar 2025.
    • 18 Dec 2024 According to Sarepta Therapeutics media release, Data from EMERGENE are expected in the first half of 2025.Assuming a positive pre-Biologics License Application (BLA) meeting and supportive data from EMERGENE, Sarepta anticipates submitting a BLA to the U.S. Food and Drug Administration seeking accelerated approval for SRP-9003 in 2025.

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