CAMBRIDGE, Mass.– Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted platform technology designation to the rAAVrh74 viral vector used in its investigational gene therapy, SRP-9003 (bidridistrogene xeboparvovec), developed to treat limb-girdle muscular dystrophy type 2E/R4 (LGMD2E or LGMDR4).
This designation marks a significant regulatory milestone and highlights the FDA’s recognition of the viral vector’s reproducibility and adaptability across multiple therapeutic applications. Sarepta’s rAAVrh74 vector has demonstrated consistent clinical results across various programs, reinforcing its utility in treating rare genetic disorders.
“This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs,” said Dr. Louise Rodino-Klapac, Sarepta’s Chief Scientific Officer and Head of R&D. “It underscores the strength of our data and our commitment to accelerating transformative treatments for rare genetic diseases.”
The FDA’s platform technology designation program aims to streamline the development and regulatory review of rare disease therapies by allowing companies to leverage existing data across multiple drug development programs. Technologies considered must be well-characterized and capable of supporting multiple therapeutics without compromising safety, quality, or manufacturing integrity. The designation allows sponsors to use previously submitted data to support new investigational new drug (IND) applications, new drug applications (NDAs), or biologics license applications (BLAs).
SRP-9003 is designed to systemically deliver a full-length beta-sarcoglycan transgene using the MHCK7 promoter, which enables strong expression in skeletal, diaphragm, and cardiac muscle. The therapy targets LGMD2E, a severe neuromuscular disorder that often leads to life-threatening cardiac or pulmonary complications. By focusing on muscle-specific delivery and gene expression, Sarepta aims to address the underlying cause of the disease and improve long-term outcomes for patients.
Sarepta, a leader in precision genetic medicine, continues to advance its platform-based approach to gene therapy development, leveraging technologies like rAAVrh74 to expand treatment options across a range of rare neuromuscular disorders.
