U.S. flag An official website of the United States government

On Oct. 1, 2024, the FDA began implementing a reorganization impacting many parts of the agency. We are in the process of updating FDA.gov content to reflect these changes.

U.S. Food and Drug Administration
  1. Home
  2. Vaccines, Blood & Biologics
  3. Tissue & Tissue Products
  4. ELEVIDYS
  1. Tissue & Tissue Products

ELEVIDYS

STN: 125781
Proper Name: delandistrogene moxeparvovec-rokl
Tradename: ELEVIDYS
Manufacturer: Sarepta Therapeutics, Inc.
Indication:

ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated in individuals at least 4 years of age:

  • For the treatment of Duchenne muscular dystrophy (DMD) in patients who are ambulatory and have a confirmed mutation in the DMD gene.
  • For the treatment of DMD in patients who are non-ambulatory and have a confirmed mutation in the DMD gene (1, 12.2) The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Product Information

  • Package Insert - ELEVIDYS
  • Demographic Subgroup Information – delandistrogene moxeparvovec-rokl (ELEVIDYS)
    Refer to Section 1.1 of the Clinical Review Memo for information about participation in the clinical trials and any analysis of demographic subgroup outcomes that is notable.

Supporting Documents

 

Back to Top