Sarepta wins first FDA platform technology designation for gene therapy

Sarepta Therapeutics announced that the rAAVrh74 viral vector used in its investigational gene therapy, SRP-9003, for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the U.S. FDA.

The newly-rolled out FDA program is designed to help support rare disease therapies by streamlining drug development, manufacturing, and review processes for drug product applications by incorporating data from designated technologies. To be eligible for the designation, the technology must be a well-understood and reproducible technology — such as a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of any such technologies — and can support the development of multiple drugs or biologics.

Sarepta uses the AAVrh74 vector in multiple clinical programs, including Elevidys, which was approved to treat Duchenne muscular dystrophy in June 2023.

Once granted, the designation enables sponsors to leverage prior data from the platform to support INDs, NDAs or BLAs.

Sarepta’s SRP-9003 is an investigational gene therapy designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat neuromuscular diseases. The therapy is intended to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to robustly express in the heart which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.