Sarepta Therapeutics Update

Sarepta Therapeutics hosted an Earnings Call on February 26, 2025 where they announced that they expect to file an Investigational New Drug Application (IND) for their LGMD2A/R1 gene therapy program in 2025.  

An IND is submitted to the FDA during a new drug’s early development. The primary goal is to determine if the product is reasonably safe for initial use in humans, and if the gene therapy investigational treatment justifies potential further development. When an investigational product is identified as a viable candidate for further development, the sponsor (in this case, Sarepta) then focuses on collecting the data and information necessary to establish that the product will not expose humans to unreasonable risks when used in limited, early-stage clinical studies. Further information on what an IND is may be found on the FDA’s website here: https://www.fda.gov/drugs/types-applications/investigational-new-drug-ind-application

Sarepta plans to initiate studying their gene therapy in humans and the IND is an important step in continuing to advance the program. When there is more information to share with the patient community about the first clinical study and milestones in the program, the community will be updated. Coalition to Cure Calpain 3 is committed to sharing news and updates on this and other LGMD2A/R1 drug development with the LGMD2A/R1 community.

Sarepta’s current focus is enrolling in their LGMD2A/R1 natural history study cohort while simultaneously advancing their preclinical (animal/cell) work. For further information on the natural history study, go to https://clinicaltrials.sarepta.com/journeyLGMD

Questions for the Sarepta team can be directed to advocacy@sarepta.com