FDA Update on the Accelerated Approval of Sarepta’s Gene Therapy   


UPDATED May 24, 2023

As you may have read in the news, Sarepta Therapeutics today announced an update from the FDA on the review of SRP-9001, Sarepta’s experimental gene therapy for Duchenne muscular dystrophy. They shared the following information: 

  • The FDA has pushed back the decision deadline to June 22, 2023 (from the original date of May 29th).   
  • The FDA has indicated by that time they may potentially grant accelerated approval for SRP-9001 in 4-5 year-olds living with Duchenne.   
  • The FDA also indicated that if the confirmatory study meets its objectives, the label may possibly be expanded to other ages.  Results of that study—called the EMBARK study–are expected at the end of this year.    

CureDuchenne is pleased that the FDA may potentially grant Accelerated Approval for SRP-9001 in 4-5 year-olds living with Duchenne. We urge the FDA to grant Accelerated Approval of this therapy, as it is a critical step in setting a path forward toward truly transformative treatments for those living with this devastating disease. We look forward to the full data readout from the EMBARK trial and expect this confirmatory study will generate important data to further inform the best clinical use of this therapy.     

We understand that this news may be disappointing, particularly if your child would be excluded from treatment because of this narrow label. We encourage you to keep hope, as there are ongoing active clinical trials that may provide treatment options for boys that are not included in this label, and we are hopeful that eligibility for this and other therapies will expand as the research progresses.

​We encourage you to contact CureDuchenne to set up a 1:1 meeting to understand the landscape of options for your family.    

CureDuchenne remains committed to finding and funding transformative treatments for all individuals with Duchenne, and we will not rest until we do.   

Read Sarepta’s Letter to the Community HERE 

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